BioSpace

It’s officially holiday shopping season, and biopharma is getting into the spirit. Novartis expanded its presence in Huntington’s, paying up to $2.9 billion to advance PTC Therapeutics’ Phase II candidate, while as Gilead committed up to $415 million to Tubulis to develop novel ADCs for solid tumors and Roche’s Genentech inked an autoimmune development deal with COUR Pharmaceuticals potentially worth more than $900 million. Finally, Takeda put up to $1.3 billion on the line for Keros’ blood cancer anemia drug, a potential competitor to Bristol Myers Squibb’s Reblozyl. On the regulatory front, Applied Therapeutics’ stock crashed more than 80% after the FDA last week denied approval of govorestat in the rare metabolic disorder classic galactosemia. Also last week, the FDA announced it is looking into the safety of bluebird bio’s gene therapy Skysona after new reports of secondary blood cancers in treated patients. On a more positive note this week, Intra-Cellular Therapies submitted an application for Caplyta in major depressive disorder, potentially opening up an additional $1 billion in revenue. Finally, R&D investment in glioblastoma is ticking up as Merck, Kazia Therapeutics and Black Diamond Therapeutics look to advance treatments for the rare but devastating brain tumor.

Biopharma had its collective deal-making hat on heading into the Thanksgiving holiday, with Roche buying Poseida for up to $1.5 billion, and Sarepta and Arrowhead tying up in a collaboration potentially worth up to $10 billion. These announcements followed last week’s news from Novartis, which snatched up Kate Therapeutics for a little over $1 billion while promising more acquisitions below $5 billion. On the regulatory front, the FDA gave BridgeBio—and transthyretin amyloid cardiomyopathy (ATTR-CM) patients—something to be thankful for late last week with the approval of Attruby for the rare, cardiovascular disease. The nod sets up a potential three-way race with Pfizer’s tafamidis and Alnylam’s Amvuttra, the latter of which was accepted for FDA review in ATTR-CM on Monday. On the opposite end of the clinical development spectrum, Cassava Sciences’ controversial Alzheimer’s drug failed to reduce cognitive or functional decline in a Phase III trial. And investors were unimpressed by the 20% weight loss generated by Amgen’s MariTide in a much-anticipated Phase II trial, as the company’s stock tumbled 11% Tuesday. Drawing much attention from the biopharma industry, President-elect Donald Trump continued with his nominees for top healthcare posts, announcing COVID-19 critic Marty Makary to lead the FDA and well-known vaccine skeptic Dave Weldon for CDC director. Meanwhile, the FDA is grappling with the loss of Chevron Deference pertaining to a legal challenge over Eli Lilly’s GLP-1 shortages. Finally, the next generation of antibody-drug conjugates remains hot, with Danish biotech Adcendo reeling in $135M in a Series B financing round.

Women represent 51% of the population; not investing is a poor investment strategy. Incorporating women into leadership positions, into board rooms, is the only way to tap this market. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠Konstantina Katcheves⁠, Senior Vice President, Innovative Medicines Global Business Development and Acquisitions, Teva Pharmaceuticals ⁠Sanskriti Thakur⁠, Chairwoman, TOWER Capital Group

President-elect Donald Trump announced last week that he would nominate prominent anti-vaxxer Robert F. Kennedy Jr. to lead the HHS. While this news caused a dip in biotech stocks, one analyst said the selloff was an overreaction. Meanwhile, fall conference season continues with the American College of Rheumatology Convergence (ACR) and American Heart Association’s 2024 Scientific Sessions (AHA). Lupus was the indication du jour at ACR, where BMS, Kyverna, Fate Therapeutics and Cabaletta Bio presented data from early-stage trials of their CAR T candidates, and Biogen and UCB detailed data behind their unexpected late-stage victory for dapirolizumab pegol. This was a much-needed win for Biogen, which has seen its shares decline 36% this year. Neurogene’s stock, meanwhile, tumbled 36% on Monday alone, following the release of details about an adverse event in a trial for its Rett syndrome gene therapy. This followed a 35% fall last week when the adverse event was first announced. And the company decided to drop its gene therapy in Batten disease after the FDA declined to grant Regenerative Medicine Advance Therapy designation to support the program. We also took a look at bluebird bio’s cash problem, with the company slated to exhaust its runway to reach a breakeven point before the end of next year. On a more positive note, RegenxBio announced this week it has aligned with the FDA on path to possible accelerated approval for its investigational gene therapy for Duchenne muscular dystrophy, following behind Sarepta’s Elevydis, which faced controversy after it received accelerated approval in June 2023, only to miss the primary functional endpoint in its confirmatory trial four months later. And speaking of accelerated approval, we released a special edition of ClinicaSpace Monday focused on this very topic. Sign up to receive it here.

Possibly the biggest news this week comes from the schizophrenia space, where AbbVie’s emraclidine failed two mid-stage trials. Acquired by AbbVie in its $8.7 billion Cerevel Therapeutics buy, this result is in stark contrast to that of Bristol Myers Squibb’s $14 billion acquisition of Karuna Therapeutics, which yielded Cobenfy—the first novel schizophrenia drug approved in 35 years. Also having a tough month is AstraZeneca, which despite reporting strong Q3 sales Tuesday has been dealing with drama at its China headquarters as a top executive there is being investigated for alleged medical insurance fraud. Meanwhile, Bayer had less good fortune in Q3 as CEO Bill Anderson acknowledged that the company’s earnings were “not pretty.” And while much of the attention during earnings season focuses on Big Pharma, several biotech companies, including Nkarta, Intellia and Cassava Sciences, also announced their earnings last week. As we head into the holiday season, some employees at Sana Biotechnology and genetic testing firm 23andMe aren’t feeling very fesitve as those companies announced layoffs. Finally, BioSpace takes a deep dive into one of the most intractable neurodegenerative diseases, Huntington’s, where the first disease-modifying drug just might be on the horizon.

No discussion regarding the future is complete unless AI is incorporated. In this episode, Konstantina Katcheves of Teva Pharmaceuticals and Sanskriti Thakur of TOWER Capital Group provide their insights on the impact of not only the benefits of the technology but the regulatory challenges and uncertainty surrounding AI. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests Konstantina Katcheves, Senior Vice President, Innovative Medicines Global Business Development and Acquisitions, Teva Pharmaceuticals Sanskriti Thakur, Chairwoman, TOWER Capital

Last week, Eli Lilly suffered a rare third-quarter earnings miss as diabetes and obesity drugs Mounjaro and Zepbound fell short of Wall Street expectations, in part due to wholesalers’ stocking decisions. On the flip side of the GLP-1 race, Novo Nordisk’s Wegovy aced part 1 of a pivotal Phase III trial in metabolic dysfunction–associated steatohepatitis (MASH), with results comparable to Madrigal’s Rezdiffra, according to analysts. In ClinicaSpace this week, BioSpace explores the shift in the CAR T cell therapy space from cancer to autoimmune disease as early data spark excitement and companies recruit autoimmune experts to fill in knowledge gaps. In the neurodegenerative space, we take a deep dive into the Alzheimer’s and Parkinson’s pipelines after recent news of a few terminated programs and returned assets. Finally, we look at the lawsuits filed by Henrietta Lacks’ estate against life sciences companies and the history of the cells that bear her name.

Earnings heat up as Pfizer got a much-needed Q3 beat amidst criticism from activist investor Starboard Value. Novartis and Sanofi are among others that have outpaced Wall Street expectations this quarter, as Eli Lilly, Merck, AbbVie, Amgen, Biogen, GSK, Bristol Myers Squibb and Takeda are all reporting today and tomorrow. The past week has also seen a pack of deals, with AbbVie’s $1.4 billion buy of Aliada Therapeutics, Roche’s potential $1 billion deal with Dyno Therapeutics and Novartis’ up to $2.1 billion commitment to Monte Rosa’s molecular glue degraders. With less than a week until Election Day, we unpack what biopharma might expect under a Trump or Harris administration. We also take a look back at 10 years of BioSpace’s NextGen list of top up-and-coming startups. A lot have been bought out by bigger companies—some for big money—while some, such as CRISPR Therapeutics, continue to operate independently. Finally, we took a close look at questions stemming from Sarepta’s new data for Duchenne muscular dystrophy gene therapy Elevidys, and separately but not unrelatedly, at the FDA’s accelerated approval pathway.

In this episode, we’re talking money. The guests in this discussion recognize that the economic climate has been different (and more chaotic) in last three to four months, but expect a steady but slow improvement as we move into the new year. This episode reviews 2024’s investment landscape and the policies influencing investment going into 2025. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests Konstantina Katcheves, Senior Vice President, Innovative Medicines Global Business Development and Acquisitions, Teva Pharmaceuticals Sanskriti Thakur, Chairwoman, TOWER Capital

Last week, Gilead withdrew Trodelvy in bladder cancer after the antibody-drug conjugate failed to meet the primary endpoint in a confirmatory study. This follows Pfizer’s recent withdrawal of another therapy that had earned FDA accelerated approval, Oxbryta for sickle cell disease. With few other options available to patients, BioSpace took a look at 5 sickle-cell candidates currently in clinical trials. Following a disappointing Alzheimer’s readout, the company’s third neuro stumble in six months, Sage Therapeutics will lay off over 165 employees—about 33% of its workforce. The company is reporting Q3 earnings on Oct. 29. On a more positive note, Vertex reported full Phase III data this week for its non-opioid pain treatment, su-zetri-gine. If approved, suzetrigine, which has a PDUFA date of Jan. 30, 2025, would be the first new class of acute pain medicine in more than two decades. And on Monday, a company that never leaves the news, Novo Nordisk, announced positive results from a cardiovascular study with its oral version of semaglutide, Rybelsus. In other Novo news, scrutiny around Novo Holdings’ acquisition of Catalent is heating up with a coalition of unions, consumer groups and public interest organizations last week expressing their concerns about the buyout. This prompted BioSpace to unpack the unique structure of the collection of organizations that is Novo. Finally, Sanofi is having a busy month, securing the sale of its healthcare unit Opella and separately paying approximately $326 million to obtain a 16% stake in European radiopharma biotech Orano Med. Radiopharma is skyrocketing in popularity, and some companies are even trying to marry it with another hot therapeutic spaces: antibody-drug conjugates. Could radiolabeled ADCs overcome some of the side effects of radiation treatments, speed up treatment times and enable lower doses than traditional therapies?

It’s that time again: earnings season. Q3 calls started out with a bit of a snore from J&J, which did beat analyst expectations but announced no big shakeups or surprises. As expected, the company saw shrinking revenue for its blockbuster Stelara, which just lost to Lilly’s Omvoh in a head-to-head clinical trial in Crohn’s disease. Last week, a concerning study was published regarding seven children who developed blood cancers after being treated with bluebird bio’s gene therapy Skysona. This publication comes at a time when the company is struggling to regain its share price, which has dropped below the Nasdaq minimum bid price requirement. The bad news about Skysona also fits with another trend BioSpace covered this week: accelerated approvals gone wrong. In other news, Sanofi is following some of its pharma peers and looking to sell its consumer healthcare unit. This follows similar moves from J&J, which spun off Kenvue last year, as well as GSK, Pfizer and others. And Senator Elizabeth Warren (Mass.-D) is upping the pressure on Novo in relation to its acquisition of Catalent, penning a letter to the Federal Trade Commission voicing concerns about the potential effects of such a deal, should it go through. Finally, bispecific antibodies make a comeback in oncology.

Designating something "safe" in this industry means acceptable risk. AI can help us improve acceptable risks if it is used appropriately. However, industry experts must be thoughtful and responsible in their use of AI within clinical trials. In the third episode of our mini series on artificial intelligence, our guests explore the challenges and benefits of integrating technology into the lives of patients. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Mike King⁠⁠⁠, Senior Director of Product and Strategy, IQVIA ⁠⁠⁠⁠Moritz von Stosch⁠⁠⁠⁠ Chief Innovation Officer, DataHow ⁠⁠⁠Nindhana Paranthaman⁠⁠⁠, Executive Medical Director, Clinical Development, Summit Therapeutics ⁠⁠⁠⁠Paul Agapow⁠⁠⁠⁠, Head of Data Science, BioNTech

The cell and gene therapy sector may be on the road to recovery after being met with investment headwinds following the highs seen during the pandemic, according to data presented Monday at the 2024 Cell & Gene Meeting on the Mesa hosted by the Alliance for Regenerative Medicine. BioSpace News Editor Greg Slabodkin reports from Phoenix. Last week, news broke that WuXi AppTec and WuXi Biologics, two companies named in the BIOSECURE Act, are looking to unload facilities in the U.S. and abroad as uncertainty looms over their U.S. business prospects. As Eli Lilly resolves shortages of its GLP-1 blockbusters, the company remains confident in its massive lead, along with competitor Novo Nordisk, over other companies with weight loss drug candidates—GLP-1s or other modalities such as next-gen CB1 inhibitors—looking to compete in the lucrative space. Not only are both companies making deals to expand their pipelines beyond GLP-1s, Lilly and Novo are actively pursuing broader markets for their current diabetes and weight loss blockbusters. Meanwhile, Big Pharma’s layoffs continue with announcements last week from Bayer, J&J and Pfizer.

Bristol Myers Squibb notched one of this year’s biggest approvals as the FDA greenlit Cobenfy, formerly KarXT, as the first novel treatment for schizophrenia in 35 years. Cobenfy’s origins go nearly as far back, beginning as an Alzheimer’s hopeful developed by current obesity rivals Eli Lilly and Novo Nordisk. On a lower note, Pfizer withdrew sickle cell medicine Oxbryta from the market, sending patients and advocates scrambling for more information. In other news, Roche hosted a Pharma Day event where it touted the $850 million acquisition of a portfolio of CDK inhibitors from Regor Pharmaceuticals and projected $3.6 billion in annual sales from three Carmot Therapeutics-acquired obesity and diabetes drug candidates. For those companies not bringing in billions of dollars, royalty financing provides an attractive option. And news editor Greg Slabodkin gives a preview of the upcoming Meeting on the Mesa. Plus, BioSpace looks at how the new abortion and IVF laws could impact women’s health research. With nearly a dozen abortion-related measures on state ballots this year, this will be an area to watch during next month’s elections.

Globalization, return on investment, diverse data sets undiscovered: this episode continues the exploration of AI. As we see the industry level out AI adoption, the future is still exciting and yet uncertain. With quantum around the corner, there are still challenges with AI at every turn. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Mike King⁠⁠⁠, Senior Director of Product and Strategy, IQVIA ⁠⁠⁠⁠Moritz von Stosch⁠⁠⁠⁠ Chief Innovation Officer, DataHow ⁠⁠⁠Nindhana Paranthaman⁠⁠⁠, Executive Medical Director, Clinical Development, Summit Therapeutics ⁠⁠⁠⁠Paul Agapow⁠⁠⁠⁠, Head of Data Science, BioNTech

Novo Nordisk CEO Lars Fruergaard Jørgensen testified Tuesday in front of Sen. Bernie Sanders’ Senate health committee regarding the prices of Ozempic and Wegovy—with some interesting takeaways. Meanwhile, PhRMA scored a rare win as the Fifth Circuit Court of Appeals ruled in the trade group’s favor, sending its complaint against the Inflation Reduction Act (IRA) back to a lower Texas court. Looking ahead, 15 more drugs are expected to be negotiated under the IRA next year, including Novo’s Ozempic. Last week, the Federal Reserve cut the federal interest rate by half a percentage point—a move that was expected and met with a collective shrug from the biotech market, with experts skeptical of its impact. Elsewhere, the schizophrenia space is gearing up for an FDA decision on BMS’ KarXT. If approved, KarXT would be the first novel drug for the neuropsychiatric disease in decades. And on the obesity front, Novo’s oral cannabinoid receptor 1 drug elicited impressive weight loss in a Phase IIa trial, with some adverse events, and young startup Metsera touted robust Phase I data for its injectable GLP-1 candidate.

After their groundbreaking approval last year, infusions of Vertex and CRISPR Therapeutics’ and bluebird bio’s sickle cell gene therapies have begun, bringing hope to patients and the companies closer to realizing revenue. Meanwhile, bispecifics and anti-TIGIT therapies were all the rage at ESMO 2024 as BioNTech, GSK and iTeos, BMS and more reported positive results across multiple cancers. Last week, Moderna announced it would slash its R&D budget by $4 billion as it targets 10 new approvals through 2027. Possibly boding well for this target, the biotech features prominently on our list of 5 late-stage mRNA vaccines to watch. Meanwhile, the oral obesity drug race continues to heat up, with Terns Pharmaceuticals, Roche and Novo Nordisk all reporting new data from their respective trials. And in the equally hot radiopharmaceuticals space, Sanofi inked a $110 million licensing deal with RadioMedix to develop a neuroendocrine tumor candidate. Finally, BioSpace takes a deep dive into the HEALEY ALS Platform Trial, which has so far seen quick failures and small victories as leaders and early participants remain hopeful.

As 2024 closes, it is only right to discuss where we are when it comes to AI and where we will be in the future. As technology continues to evolve and blend into science in 2025 and beyond, a practical approach to what it can and cannot do must be explored. Additionally, how overpromising while underdelivering has affected investors' and the industry’s confidence in AI. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Mike King⁠⁠⁠, Senior Director of Product and Strategy, IQVIA ⁠⁠⁠⁠Moritz von Stosch⁠⁠⁠⁠ Chief Innovation Officer, DataHow ⁠⁠⁠Nindhana Paranthaman⁠⁠⁠, Executive Medical Director, Clinical Development, Summit Therapeutics ⁠⁠⁠⁠Paul Agapow⁠⁠⁠⁠, Head of Data Science, BioNTech

Summit Therapeutics made headlines this weekend at the 2024 World Conference on Lung Cancer, revealing that its bispecific antibody outperformed Keytruda in first-line non-small cell lung cancer. Meanwhile, bispecifics are one of several therapeutic classes on the agenda at the 2024 European Society for Medical Oncology (ESMO) Congress in Barcelona later this week. Last week, BioMarin held a public address intended to calm anxious investors after rounds of layoffs and pipeline changes—but many were left wanting. Also undergoing major upheaval is Lykos Therapeutics, which laid off 75% of its workforce after failing to secure approval for its MDMA-based post-traumatic stress disorder therapy and announced last week that CEO Amy Emerson is stepping down from her role. In Washington, D.C., Congress is back in session this week and wasted no time in reviewing the BIOSECURE Act, which passed a House vote on Monday. And on the weight loss front, Terns Pharmaceuticals is moving ahead to Phase II after its investigational GLP-1 pill elicited positive results in Phase I, and Amgen is pushing MariTide into a broad late-stage development program that will test the obesity treatment in other weight-related conditions, such as heart, kidney and liver diseases.

Eli Lilly shook up the weight loss market again last week, announcing plans to sell single-dose vials of its blockbuster GLP-1 drug Zepbound directly to consumers. Meanwhile, Novo Nordisk said Monday that it expects the shortage for the lower doses of its own GLP-1 therapy Ozempic to persist into the fourth quarter of 2024. This week, we take a closer look at eyes, where gene therapy is breaking through against wet AMD, a common cause of blindness—potentially significantly minimizing the number of treatments required by patients—and cell therapy is making strides against another common foe: dry eye disease. Lastly, an unfortunate trend—layoffs—continues to play out, with BioMarin, Genentech and Astellas Gene Therapies all parting with staff members.

As the legal landscape continues to evolve, the uncertainty surrounding diversity, equity and inclusion (DEI) initiatives increases though the life sciences industry continues to move forward with DEI initiatives to include all patients in clinical trials. In this episode, our guests discuss framing strategies designed to protect DEI initiatives from legal challenges. Additionally, the guests acknowledge the importance of clinical trial sites in gaining patient trust. We also address the increased burden technology is putting on the sites as well as patients, suggesting potential ways to reduce these burdens. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Otis Johnson, PhD, MPA⁠⁠, Principal Consultant and Co-founder, Trial Equity ⁠⁠Pamela Tenaerts, MD, MBA⁠⁠, Chief Science Officer, Medable ⁠⁠Chris Hart,⁠⁠ Partner, Co-Chair, Privacy and Data Security Group, Foley Hoag LLP ⁠⁠Patrick Floody⁠⁠, Executive Director, Global Clinical Trial Services, Regeneron ⁠⁠Ken Getz⁠⁠, Executive Director, Tufts Center for the Study of Drug Development; Professor, Tufts University School of Medicine Disclaimer:The views expressed in this discussion by Patrick Floody are his own and do not represent those of Regeneron.

Gene editing startup Tome Biosciences is laying off nearly its entire workforce, as biopharma companies continue to face challenges in the current market. Meanwhile, Bayer, which cut 1,500 jobs in May, is parting with another 150 in Basel, Switzerland. On the other side of the spectrum, two new companies debuted last week. Novartis and Versant Ventures launched Borealis Biosciences to develop RNA medicines for kidney diseases, and BridgeBio spawned GondolaBio to focus on therapies for genetic and rare diseases. In Q2, biopharma VC funding reached its highest quarterly level since the same quarter in 2022, according to PitchBook. Separately, U.S.-China biopharma relations are making headlines again, with a House committe writing to the FDA comissioner about U.S. companies working with the Chinese military on potentially unethical clinical trials. And in case you missed it, a special edition of ClinicaSpace this week focuses on the obesity and diabetes space—a combined market that is expected to exceed $200 billion within the next decade.

With the Democratic National Convention underway, the Biden Administration’s announcing the first 10 drug prices negotiated under the under the Inflation Reduction Act seems likely to be a political move. But it’s still unclear how much the government is really saving—and what sorts of discounts patients will see. Separately, the biopharma industry saw a flood of layoffs. Lykos Therapeutics slashed around 75% of its staff following the FDA’s rejection of its MDMA-assisted therapy for PTSD. Meanwhile, diagnostic company GRAIL parted with about a quarter of its workforce, and German biotech Evotec could cut 400 roles globally. And as vaccine makers fall off the COVID-19 cliff, some are turning to combination COVID-flu vaccines, with Pfizer and BioNTech last week announcing mixed data following Moderna’s positive results earlier this summer.

In the second part of the discussion, our guests address clinical trial design which if done without careful consideration of the patient population can exclude patients from clinical trials instead of being inclusive. Using specific examples, our guests highlight the progress the industry has made to design inclusive clinical trials but stress the continuing need for improvement. In parallel, our guests discuss the continual judicial actives at the federal and state level surrounding women's health issues. As states begin to challenge other states for access to women's data, the future participation of women patients in clinical trials is becoming uncertain. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠Otis Johnson, PhD, MPA⁠, Principal Consultant and Co-founder, Trial Equity ⁠Pamela Tenaerts, MD, MBA⁠, Chief Science Officer, Medable ⁠Chris Hart,⁠ Partner, Co-Chair, Privacy and Data Security Group, Foley Hoag LLP ⁠Patrick Floody⁠, Executive Director, Global Clinical Trial Services, Regeneron ⁠Ken Getz⁠, Executive Director, Tufts Center for the Study of Drug Development; Professor, Tufts University School of Medicine Disclaimer:The views expressed in this discussion by Patrick Floody are his own and do not represent those of Regeneron.

In one of the year’s most highly anticipated decisions, the FDA rejected Lykos Therapeutics’ MDMA-assisted therapy for post-traumatic stress disorder (PTSD). Reaction from Lykos was swift, with the company stating its intention to “ask for reconsideration of the decision.” Meanwhile, against the backdrop of the CDC’s recent RSV guidelines, Pfizer scored a big Phase III win for its shot in immunocompromised adults. Separately, Merck halted a Phase III trial of its Keytruda, anti-TIGIT, chemo combo in small cell lung cancer but made a splash with the potential $1.3 billion acquisition of Curon’s B cell depletion therapy. And AI-focused biotechs Recursion and Exscientia are merging to create a new company that will take Recursion’s name. Plus, as we reflect on Q2 earnings, it’s becoming clear that Eli Lilly is catching up to Novo Nordisk in the weight loss sphere, while others faced challenges in the vaccine space and the continued COVID cliff. Finally, BioSpace highlights five obesity data readouts to watch in the second half of 2024.

This week, Kamala Harris selected Minnesota Governor Tim Walz as her running mate. In July, Harris’ campaign netted a whopping $310 million—so where do pharmaceutical-connected contributions stand? Meanwhile, Q2 earnings continue to roll in, and the picture isn’t looking so bright for some companies particularly vaccine makers like BioNTech, Moderna and Pfizer, who have been hit hard by the COVID-19 cliff and new CDC guidelines around RSV vaccines. The FDA has also been busy of late, approving the first engineered cell therapy for solid tumors in Adaptimmune’s Tecelra. The regulator has another potential first on the docket this week in the form of Lykos Therapeutics’ MDMA-assisted treatment for PTSD—but Lykos will need to overcome a decidedly negative advisory committee vote and concerns surrounding its Phase III trial design. If approved, it would be the first classic psychedelic therapy authorized in the U.S.

The 2023 New Benchmarks on Demographic Disparities in Pivotal Trials study indicates that as Black representation increases, clinical trial enrollment time decreases. This is contradictory to what has been reported in the past. However, the highest disparity in clinical trial enrollment remains to be Black or African descent patients, with only a third being enrolled in clinical trials. Understanding not just the benefits on patient lives, but also the cost-effectiveness of adequate representation, pharma companies are continuing to lean into DEI practices for their clinical trials. In parallel, the legal environment for DEI initiatives has become unstable. With organizations actively pursuing legal cases against DEI initiatives, pharmaceutical and biotech companies must keep a watchful eye on judicial and political activities surrounding DEI and how these will shape the future of the clinical trial space in the United States. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests Otis Johnson, PhD, MPA, Principal Consultant and Co-founder, Trial Equity Pamela Tenaerts, MD, MBA, Chief Science Officer, Medable Chris Hart, Partner, Co-Chair, Privacy and Data Security Group, Foley Hoag LLP Patrick Floody, Executive Director, Global Clinical Trial Services, Regeneron Ken Getz, Executive Director, Tufts Center for the Study of Drug Development; Professor, Tufts University School of Medicine Disclaimer:The views expressed in this discussion by Patrick Floody are his own and do not represent those of Regeneron.

Second-quarter earnings season continues with Big Pharma beating Wall Street expectations, the author of an encrypted email sent to BioSpace has a proposal for Moderna and Merck, Roche and Viking seek quicker entry to the obesity market, and AAIC is in full swing. As July comes to a close, biopharma second-quarter earnings continue to roll in with Pfizer, Merck, AbbVie, AstraZeneca and many more reporting. So far, everything is coming up roses with most major companies beating Wall Street expectations. But every rose has its thorn and for biopharma executives this has long been drug price negotiations under the Inflation Reduction Act. Recently, however, drugmakers have expressed confidence that the IRA will not greatly impact their bottom lines. Speaking of bottom lines, in an anonymous email sent to the CFOs of Merck and Moderna and shared with BioSpace, a shareholder made the case that Merck should buy Moderna out of its 50/50 partnership involving Keytruda and their shared investigational personalized cancer vaccine. Meanwhile, M&A activity picked up in the second quarter of 2024 with nearly $18 billion changing hands, according to J.P. Morgan. Q3 has been no stranger to deals so far, either, with Boehringer Ingelheim acquiring Nerio Therapeutics for up to $1.3 billion to boost its immuno-oncology pipeline, and GSK and Flagship Pioneering striking a potential $7B deal to develop a portfolio of novel vaccines and medicines starting with immunology and respiratory treatments. Pfizer raised its full-year outlook after reporting positive Q2 results and announced layoffs this week at two North Carolina sites, with a total of 210 workers losing their jobs. Cuts at the Sanford, N.C. gene therapy manufacturing site come on the heels of Pfizer’s Duchenne muscular dystrophy gene therapy failed a Phase III trial. On the obesity front, both Viking Therapeutics and Roche indicated that they will seek entry to the market more quickly than originally anticipated, and the FDA issued a warning on Monday about possible safety risks associated with compounded versions of Novo Nordisk’s semaglutide. And the Alzheimer’s Association International Conference (AAIC) this week revealed some interesting nuggets, including long-term data regarding Eisai and Biogen’s Leqembi, and a small Phase IIb study showing that Novo’s GLP-1 liraglutide slowed cognitive decline in Alzheimer’s patients by up to 18%.

While the biopharma industry has seen glimmers of economic optimism, there were still more than 14,000 employees laid off in the first half of 2024. And BioSpace’s readers are among them. In this week’s Job Market Trends, H2 Update, 43% of respondents indicated they are currently unemployed, the highest-ever rate since BioSpace started conducting surveys. One large company that intends to eliminate around 680 jobs in its project development sector over the next few years is Novartis. Despite this, the Swiss pharma announced positive Q2 results last week, exceeding revenue expectations thanks to the continued revenue generated by its blockbuster drugs. Johnson & Johnson, which released its second quarter results on Wednesday, also beat analyst estimates. Interestingly, Novartis CEO Vas Narasimhan said in a statement that coming into the market late with another GLP-1 drug would not be a “prudent approach” for the company. Meanwhile, several companies, including start-ups, are firmly committed to the weight loss space. Recently launched companies like Metsera, ProFound Therapeutics and Metaphore are developing GLP-1s or leveraging other mechanisms to target obesity, while market leaders Novo Nordisk and Eli Lilly are expanding their GLP-1 pipelines beyond weight loss. Novo and Lilly are also now marketing their GLP-1 medicines, semaglutide and tirzepatide, for weight loss in China, where GLP-1 biosimilars will soon hit the market. And finally, the Cassava Sciences saga continued, with CEO Remi Barbier and SVP of Neuroscience Lindsay Burns both resigning their posts. Cassava has faced allegations of data manipulation relating to its Alzheimer’s drug simufilam. The company has denied any wrongdoing.

This is the third episode of Denatured's discussion on diversity, equity and inclusion (DE&I). AI represents unlimited opportunities to increase efficiency, but as our guests note, it can also do incredible harm to patients and society. It largely comes down to training of the professionals using AI, as well as understanding how the AI model was grounded or trained. The data quality must be accurate and clean, but as they noted, DE&I is an issue the industry has been struggling with for decades. The solution lies in humans identifying problems, such as the lack of patient diversity in data, as well as studies in certain disease states, such as HPV-derived cancers. Going further, it is up to the industry to collaborate with each stake holder within the life sciences and healthcare spaces to use AI in ways that are patient focused. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests Dr. ⁠⁠⁠Ali Pashazadeh⁠⁠⁠, CEO,Treehill Partners Dr. ⁠Charlotte Jones-Burton⁠, Board Member, bluebird bio; Founder & President, Women of Color in Pharma⁠⁠⁠⁠⁠ ⁠Chia Chia Sun⁠⁠⁠⁠, Chief Commercial Officer, ⁠⁠⁠Fab Biopharma; CEO, Damiva ⁠Phyllis Greenberger⁠, Senior Vice President, Policy & Regulatory, Healthy Women Dr. ⁠Todd Rudo⁠, Chief Medical Officer, Clario

Pfizer stole the show late last week with the announcement that it will move forward with its oral GLP-1 analog danuglipron. While Pfizer may hope to advance in the already crowded race to bring an oral GLP-1 to market, some analysts reacted to the news with caution, mainly due to lack of data. Meanwhile, in the injectable GLP-1 space, it’s primarily a two-horse race between Novo and Lilly. While Novo had a two-year head start, Lilly has been picking up ground since the November 2023 approval of Zepbound (tirzepatide). A recent observational study saw tirzepatide outperform Novo’s semaglutide in helping patients to lose weight, and some analysts predict Lilly will indeed gain majority market share within a few years in part due to this potential efficacy advantage, though manufacturing is a complicating factor that currently favors Novo. Separately, the drug pricing debates heat up after the Federal Trade Commission released a scathing report last week about the business practices of pharmacy benefit managers (PBMs), noting how they charge payers higher prices than what they pay to pharmacies, profiting at the expense of patients and pharmacies. But biopharma firms must share the blame, and the Senate last week unanimously passed a bipartisan bill that would limit their use of patent thickets to maintain exclusivity, thereby making it easier for generic competitors to enter the market. Finally, several neurology-focused companies are hoping to bring new drugs to the market. BioSpace highlights five key neuro readouts to watch in the second half of 2024 in the Alzheimer’s, schizophrenia, depression and rare disease spaces.

After securing approval last week for Alzheimer’s drug Kisunla, Eli Lilly was back in the news Monday with the $3.2 billion acquisition of immunology biotech Morphic Holding. This represents another deal in the lucrative immunology and inflammation (I&I) space, which saw $12.3 billion in M&A activity last year, with analysts predicting this momentum would carry over into 2024. Investment continues to pour into another hot therapeutic space: antibody-drug conjugates (ADCs). British biopharma company Myricx Bio netted a £90 million ($115.5 million) Series A round to expand its ADC platform and advance its pipeline into the clinic. Several other pharmaceutical companies are looking to capitalize on the potential of the ADC market—which is estimated to reach nearly $30 billion by 2028—including Johnson & Johnson, Genmab and Ipsen, who have all struck M&A deals in the space this year. Still others, including Sutro Biopharma and Mersana Therapeutics, are hoping to supercharge their potential via the immune system with immunostimulatory ADCs. And GLP-1s continue to make headlines, with one study linking semaglutide to an optic nerve condition that can cause sudden vision loss, and another showing the drug class could significantly lower the risk of 10 obesity-related cancers in diabetes patients. Novo Nordisk’s Wegovy has successfully expanded into China, where its patent will expire in less than two years, paving the way for biosimilar competition, and Lilly’s Mounjaro elicited greater weight loss than Novo’s Ozempic in a new observational study.

The biggest news of the week was the FDA approval of Eli Lilly’s Kisunla (donanemab) on Tuesday. While not unexpected, it was one of the year’s most highly anticipated decisions. And last week saw another big approval from the FDA—that of Verona’s novel COPD drug. But the regulator has also dropped three Complete Response Letters on drugmakers in the last seven days. Two of these were directly related to issues with third-party manufacturers. Though it’s unclear if those contract development and manufacturing organizations (CDMOs) were overseas, the FDA has flagged several concerns regarding manufacturers in India and China that have contributed to extreme drug shortages in the U.S. This will be particularly important in the face of a potential decoupling from Chinese CDMOs should the BIOSECURE Act become law, as India has been one country eyeing the opportunity. Meanwhile, Korean company Samsung Bio struck a $1 billion manufacturing contract with an undisclosed U.S. biopharma company. Among the many products the manufacturer makes are the piping hot antibody-drug conjugates, or ADCs. The global market has already exceeded $10 billion and is estimated to grow to nearly $30 billion by 2028. As evidence of the excitement surrounding ADCs are five major deals struck by biopharma companies this year. It’s not all good news for the ADC space, though, as one of those three FDA rejections was handed to Merck and partner Daiichi Sankyo’s investigational ADC for the treatment of certain non-small cell lung cancers. Meanwhile, BMS backed out of a collaboration with Eisai to develop an ADC being investigated in ovarian, peritoneal and fallopian tube cancers, as well as non-small cell lung cancer. We will continue to watch the industry’s strategies unfold as biopharma firms compete for a piece of that exploding ADC market.

This is the second episode of Denatured's discussion on diversity, equity and inclusion (DE&I). Data is the essential piece for both science and technology. The lack of DE&I data is becoming even more imperative as we move towards personalized medicines. Without understanding why there are differences in outcomes, the industry will continue to see worsening outcomes. However, DE&I is still not at the forefront of the mind during clinical trial designs, which influences not only the training but the application of AI models. Additionally, what this means not only for life sciences but also for healthcare providers as more of the healthcare industry adopts AI to assist with patient care. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests Dr. ⁠⁠Ali Pashazadeh⁠⁠, CEO,Treehill Partners Dr. Charlotte Jones-Burton, Board Member, bluebird bio; Founder & President, Women of Color in Pharma⁠⁠⁠ Chia Chia Sun⁠⁠⁠, Chief Commercial Officer, ⁠⁠⁠Fab Biopharma; CEO, Damiva Phyllis Greenberger, Senior Vice President, Policy & Regulatory, Healthy Women Dr. Todd Rudo, Chief Medical Officer, Clario

The biggest news of the last week was easily the expanded approval of Sarepta’s Duchenne muscular dystrophy gene therapy, Elevidys. CBER Director Peter Marks again overruled FDA staff members and review teams to grant the therapy full approval and a broad label expansion despite its missing the primary endpoint in a Phase III confirmatory study. This approval has us thinking about other big FDA decisions to watch this year, first and foremost, Eli Lilly’s anti-amyloid antibody donanemab. If given the green light, donanemab will be a direct competitor to Biogen and Eisai’s Leqembi, also a disease-modifying anti-amyloid antibody. Beyond that, Verona Pharma is expecting a decision Wednesday on ensifentrine, which could be the first novel mechanism for chronic obstructive pulmonary disease in over a decade, and in August, the FDA is set to decide on Lykos’ MDMA-assisted PTSD therapy, which an advisory committee voted against earlier this month. Then this week, Alnylam scored big with a Phase III win in transthyretin amyloidosis with cardiomyopathy (ATTR-CM). In what Alnylam CMO Pushkal Garg called “overwhelmingly positive data,” Amuvttra significantly lowered the risk of death and recurrent cardiovascular events in patients with ATTR-CM. Meanwhile, biopharma conference season continues with the American Diabetes Association’s annual conference held this past weekend. Eli Lilly’s blockbuster drug Zepbound “significantly improved” disease severity in patients with obesity and sleep apnea, and Altimmune racked up a Phase II victory for pemvidutide, which was effective at helping patients lose weight while retaining lean muscle.

BioSpace's ⁠Lori Ellis⁠ and ⁠Chantal Dresner⁠ are bringing live updates from ⁠#DIA2024⁠ in San Diego this week where we've been attending sessions on trial design, digital twins, cell and gene therapy regulation, clinical research workforce trends, professional development and many more. We discuss some of our key takeaways and event highlights, including Lori's exclusive interview with CBER director Dr. Peter Marks. Hosts ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠⁠⁠BioSpace⁠⁠⁠ Chantal Dresner, VP of Marketing, BioSpace⁠⁠⁠

Diversity, equity and inclusion (DEI) continues to be a challenge for drug and medical device developers. While there has been some improvement in clinical trials, our guests note that unless there is a commercial or regulatory need, DEI is still an afterthought for many developers when designing clinical trials. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Ali Pashazadeh⁠⁠, CEO,Treehill Partners Charlotte Jones-Burton, Board Member, bluebird bio; Founder & President, Women of Color in Pharma⁠⁠⁠ Chia Chia Sun⁠⁠⁠, Chief Commercial Officer, ⁠⁠⁠Fab Biopharma; CEO, Damiva Phyllis Greenberger, Senior Vice President, Policy & Regulatory, Healthy Women Todd Rudo, Chief Medical Officer, Clario

We’re off to a rocky start this week on the clinical side, with some safety issues. BioNTech and MediLink’s ADC was placed on partial hold after multiple patient deaths, and some safety concerns were also revealed for Gilead’s blood cancer therapy magrolimab. But on a more upbeat note, DIA in San Diego is filled with hope of collaboration and a focus on the patient experience. Head of Insights Lori Ellis joins BioSpace’s editorial team to discuss what’s she’s observed so far at the meeting and what she’s looking forward to in the coming days. In global geopolitical news, the BIOSECURE Act was unexpectedly left out of the DoD’s spending bill. But that doesn’t mean the legislation is going anywhere, and the U.S. House of Representatives committee’s recent request that the FBI put together a briefing on GenScript Biotech highlights the ripple effect the act is having on the Chinese market. Other news includes the busy regulatory activity from the FDA, approving the first new treatment in nearly a decade for a rare liver disease. This week, the agency will rule on full approval and a potential label expansion for Sarepta’s Elevidys. And GLP-1s, as always, are making headlines. A new report from Global Data forecast the obesity market will reach $111 billion by 2033. This includes the blockbuster GLP-1s as well as alternatives being pursued by multiple companies. Just last week, Sytis Bio launched with a lead program consisting of an oral drug meant to mimic the effects of oral bypass surgery. Finally, there’s some good news on the financing front, as J.P. Morgan raised over $500 million in its first biotech VC fund. It’s the latest sign of the uptick in VC funds devoted to biopharma and tech industries.

BioSpace's Lori Ellis and Chantal Dresner are bringing live updates from #DIA2024 in San Diego this week where the first day kicked off with an inspiring story from Tom and Emily Whitehead of the Emily Whitehead Foundation, setting the tone for patient-centric discussions. The following panel featuring Emer Cooke, Chair, ICMRA an Executive Director, European Medicines Agency; Peter Marks, Director, CBER, FDA; Carsten Linnerman, CEO, Neogene Therapeutics, AstraZeneca Group; Dean Kamen, Founder, DEKA; and Stacy Hurt, Chief Patient Officer, Parexel emphasized the imperative of collaboration to improve outcomes, between regulatory bodies as well as with patients. Tune in this week to hear more from the event!

The big news of this week so far was Monday’s FDA advisory committee for Eli Lilly’s Alzheimer’s drug donanemab, where the vote was unanimous in support of the anti-amyloid antibody. If approved, donanemab would be a direct competitor of Biogen and Eisai’s Leqembi, but analysts believe there’s plenty of room in the market for both, and even predict that donanemab’s potential approval could be beneficial for Leqembi in the long run by increasing investments in advocacy and infrastructure. The mood was decidedly different from last week’s FDA adcomm, which voted overwhelmingly against approving Lykos Therapeutics’ MDMA-assisted PTSD therapy. Interestingly, that vote came down less an hour before BioSpace Senior Editor Heather McKenzie hosted a panel at BIO where Dan Karlin, chief medical officer at MindMed, said he’d hoped the conversation would have been different. In other conference news, Eli Lilly and partners Boehringer Ingelheim and Zealand Pharma presented mid-stage results for their GLP-1 products in the MASH space at the Congress of the European Association for the Study of the Liver. These companies are eyeing a piece of the MASH market that Madrigal Pharmaceuticals first tapped into earlier this year when its therapy, Rezdiffra, was approved in March. Finally, CBRE published a report on challenges in finding R&D and manufacturing talent, and thousands of layoffs continue to hit the biopharma industry. According to our Layoff Tracker, there have been 14,000 positions cut this year, with BMS and Bayer being the unfortunate leaders, each with more than 1,500 layoffs underway. Still, analysts are optimistic that the second half of 2024 could be better, as an uptick in the financial markets might provide companies with money to spend on growing their workforces.

How do we ensure strategic vision and thoughtful implementation when pursuing new opportunities in science and technology? Our guests discuss challenges around reimbursement, intellectual property, change management and the critical nature of early engagement. Additionally, we are joined by Tamei Elliott of DIA who shares insights on key discussions and themes of the upcoming meeting in San Diego. This is the third and final episode in our preparation for the roundtable discussion: ⁠⁠Pioneering New Frontiers: Advanced Drug Delivery Technologies and Cell/Gene Therapies in Combination Products⁠⁠ taking place at the ⁠⁠DIA 2024 Global Annual Meeting⁠⁠. James Wabby, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, AbbVie is chairing the discussion between Rob Schulz, President and Chief operating officer of Suttons Creek, William Daunch, Chief Technology Officer, Focal Medical, Inc, Andrea Gray, Biomedical Engineer Advisor, CBER, FDA, and Michael Lehmicke, Senior Vice President, Science and Industry Affairs, Alliance for Regenerative Medicine. Lori Ellis will be covering DIA Global Annual meeting 2024, June 16-20 in San Diego. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠⁠⁠BioSpace⁠⁠⁠ Guests ⁠⁠James Wabby⁠⁠, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, ⁠⁠AbbVie, United States⁠⁠ ⁠⁠Rob Schulz⁠⁠, President and COO, ⁠⁠Suttons Creek, Inc., United States Tamei Elliott, Associate Director, Scientific Programs (Americas), DIA

Conference season kicked into high gear this week with the American Society of Clinical Oncology (ASCO) annual meeting in Chicago and the BIO International Convention in San Diego. Check out BioSpace’s ASCO24 Tracker with all the biggest data and other news from the cancer meeting, which wrapped up on Tuesday. Among the trial results highlighted at ASCO24 were BMS’ Phase III study results in liver cancer. There was also a lot of news on non-small cell lung cancer (NSCLC): Pfizer presented results from a Phase III trial evaluating its ALK inhibitor Lorbrena, BMS provided three updates on its immunotherapy Opdivo and Opdivo-based combinations, while Gilead unveiled additional details on a failed Phase III trial for its antibody-drug conjugate (ADC) Trodelvy. Currently, there is one ADC approved to treat NSCLC, AstraZeneca and Daiichi Sankyo’s Enhertu, for patients with HER2-mutated metastatic disease. While the companies’ investigational ADC Dato-DXd is making some headway against NSCLC, experts recently told BioSpace that more compelling surface targets for lung cancer are needed in order for ADCs to really make a difference. When it comes to M&A, Big Pharma continues to show strong interest in oncology, particularly ADCs and radiopharmaceuticals. However, last week’s biggest deal was Merck’s $3 billion acquisition of EyeBio and its first-in-class trispecific antibody Restoret, marking the company’s return to the ophthalmology space after nearly a decade. For now, it appears Big Pharma favors smaller M&A over megabillion-dollar deals. Recent buying activity also indicates a potential resurgence in the appetites of larger companies for psychiatric drug development.

Regulators suggest developers engage in discussions early, however those conversations cannot take place unless developers are sure of the asset's category. Existing confusion surrounding advanced therapy products may become more challenging as both science and technology evolve. With both developers and regulators, two risk adverse entities, addressing this evolution and continually updated regulations, advanced therapy product development continues to be a collaborative process. This is the second episode in our preparation for the roundtable discussion: ⁠Pioneering New Frontiers: Advanced Drug Delivery Technologies and Cell/Gene Therapies in Combination Products⁠ taking place at the ⁠DIA 2024 Global Annual Meeting⁠. James Wabby, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, AbbVie is chairing the discussion between Rob Schulz, President and Chief operating officer of Suttons Creek, William Daunch, Chief Technology Officer, Focal Medical, Inc, Andrea Gray, Biomedical Engineer Advisor, CBER, FDA, and Michael Lehmicke, Senior Vice President, Science and Industry Affairs, Alliance for Regenerative Medicine. Lori Ellis will be covering DIA Global Annual meeting 2024, June 16-20 in San Diego. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠⁠BioSpace⁠⁠ Guests ⁠James Wabby⁠, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, ⁠AbbVie, United States⁠ ⁠Rob Schulz⁠, President and COO, ⁠Suttons Creek, Inc., United States

At its Investor Day 2024 event last week, AstraZeneca announced plans to reach $80 billion in total revenue by 2030—up from $45.8 billion in 2023—and to launch 20 new medicines by the end of the decade. Does AstraZeneca have the assets and developmental pipeline to hit this lofty target? Meanwhile, Bayer, BMS and Pfizer are looking to conserve cash through cost-savings plans that include layoffs. It’s a trend we’ll be keeping a close eye on as companies face potential implications from upcoming patent expirations, the Inflation Reduction Act’s Drug Price Negotiation Program and investor expectations. On the R&D front, Takeda took a deep dive into the hot molecular glue degrader space, signing a potential $1.2 billion exclusive licensing deal with China-based Degron Therapeutics. The new partners will be going after various oncology, neuroscience and inflammatory disease targets. Takeda follows Novo Nordisk, Roche’s Genentech and Merck, who have all recently struck R&D deals focused on molecular glues degraders. And the conference season again kicks into high gear this weekend with the American Society of Clinical Oncology (ASCO)’s annual meeting starting Friday in Chicago and the 2024 BIO International Convention beginning Monday in San Diego. BioSpace’s Heather McKenzie will be moderating a BIO panel focused on closing the gaps in neuropsychiatric drug development, so if you’re heading to San Diego, please pop in and say hello!

With every new advancement in either science or technology there is increased excitement as we see the potential. Cell and gene therapies (CGT) as combination products are emerging as the stage in the evolution of technology and science merging together. While the potential benefits for patients are great, there are risks and challenges that must be carefully considered. This discussion is part of one of a precursor of the roundtable discussion: Pioneering New Frontiers: Advanced Drug Delivery Technologies and Cell/Gene Therapies in Combination Products taking place at the DIA 2024 Global Annual Meeting. James Wabby, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, AbbVie is chairing the discussion between Rob Schulz, President and Chief operating officer of Suttons Creek, William Daunch, Chief Technology Officer, Focal Medical, Inc, Andrea Gray, Biomedical Engineer Advisor, CBER, FDA, and Michael Lehmicke, Senior Vice President, Science and Industry Affairs, Alliance for Regenerative Medicine. Lori Ellis will be covering DIA Global Annual meeting 2024, June 16-20 in San Diego. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠BioSpace⁠ Guests James Wabby, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, AbbVie, United States Rob Schulz, President and COO, Suttons Creek, Inc., United States

Bayer’s recent layoffs of 1,500 mark a step toward CEO Bill Anderson’s stated goal of removing managerial layers at the company—“fewer bosses, fewer rules,” he told The Wall Street Journal. The move follows BMS’ announcement that it will let go more than 2,000 people. In other news, J&J expanded its dermatology portfolio, paying $850 million in cash plus potential milestone payments for California-based Proteologix and its Phase I–ready atopic dermatitis candidate. J&J is competing with Eli Lilly, which continues to actively pursue this space as well, with positive Phase IIIb results for its candidate in March. Roche’s Genentech is looking to jump into the hot GLP-1 space with positive results from a Phase Ib trial for its investigational weight-loss treatment CT-388. The once-weekly subcutaneous injection was part of Roche’s acquisition of Carmot Therapeutics in December for $2.7 billion—a hefty sum as Roche looks to compete with frontrunners Novo Nordisk and Eli Lilly. Separately, Lilly announced a late-stage win for its weekly insulin product, which outperformed Novo’s Tresiba in adults with type 2 diabetes. We also touch on Novo’s latest positive study on Wegovy and a couple of big approvals last week, including BMS’ third indication for its CAR T therapy Breyanzi and Amgen’s Imdelltra, the first bispecific T-cell engager therapy for advanced small cell lung cancer. Finally, we discuss this week’s special edition of ClinicaSpace, focused on psychedelics for mental health disorders. There’s a pretty extensive pipeline of therapies of psilocybin, DMT and more, for depression, substance abuse disorders and other neuropsychiatric diseases. The first psychedelic therapy for a neuropsychiatric disease—Lykos Therapeutics MDMA for PTSD—is up for FDA approval this summer. The FDA is convening an adcomm on June 4 to discuss the therapy.

Bayer made news this week announcing 1,500 layoffs as part of an effort to save €500 million ($540 million) in 2024 and €2 billion ($2.16 billion) in 2026, reminiscent of BMS’ announcement of a major restructuring that will involve 2,200 layoffs and aims to save $1.5 billion by the end of 2025. Bayer’s news just about rounds out the Q1 2024 earnings season highlighted by strong sales of GLP-1 weight loss drugs. However, it was Amgen that emerged as a big winner, with revenue increasing over 22% from Q1 2023. In other news this week, Takeda took another deep dive into the Alzheimer’s space with a deal worth up to $2.2 billion with AC Immune for global rights to an immunotherapy that targets toxic forms of amyloid beta. And the BIOSECURE Act got an update, now naming WuXi Biologics among the Chinese firms that U.S. companies are expected to cut ties with, and setting a deadline of 2032. Finally, we discuss the major takeaways from last week’s 2024 American Society of Gene & Cell Therapy (ASGCT) annual meeting. While the cell and gene therapy space faces many challenges, promising updates included Regeneron’s news that its gene therapy improved hearing in two young deaf children. And throughout the week, Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), spoke with positivity on how the community is now working to address the challenges it now faces, touching on topics such as accelerated approval, global regulatory convergence and the Bespoke Gene Therapy Consortium, aimed at accelerating the delivery of AAV-based gene therapies for rare diseases.

12 billion dollars: one of the boldest women’s health research moves an administration has made for women’s health research. On a global scale, this has caused countries and sovereign states to start investing in women’s health. But in the United States, the funding is not guaranteed. It depends on congressional approval, which is complicated during an election year. As the United States continues to grapple with politics and science meeting, the global healthcare sector is already beginning to reframe the language and invest in women’s health research. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠⁠BioSpace⁠⁠ Guests ⁠⁠Sans Thakur⁠⁠, Founder and Chairwoman of ⁠⁠Tower Capital⁠⁠ ⁠⁠Chia Chia Sun⁠⁠, Chief Commercial Officer at ⁠⁠Fab Biopharma⁠⁠ ⁠⁠Elisa Cascade⁠⁠, Chief Product Officer of at ⁠⁠Advarra⁠⁠ Shawana Moore, Medical Advisor on the ⁠⁠Advisory Council for Healthy Women⁠⁠ ⁠⁠Katie Schubert⁠⁠, President and Chief Executive Officer of the ⁠⁠Society for Women’s Health

In this edition of The Weekly, Managing Editor Jef Akst and News Editor Greg Slabodkin share their insights from the American Society of Gene & Cell Therapy’s 27th annual conference, including advances around adeno-associated viruses and the ongoing discussion about the FDA’s accelerated approval program and how it relates to rare diseases. Also, on Tuesday, Eli Lilly announced that an advisory committee meeting will be held for its Alzheimer’s drug donanemab on June 10. Lilly is aiming for full approval of the anti-amyloid antibody after accelerated approval was denied in January 2023. And the race between Vertex and bluebird bio’s gene therapies Casgevy and Lyfgenia is heating up.

In this special edition of The Weekly, we discuss the much-anticipated annual meeting of the American Society of Gene & Cell Therapy in Baltimore. It starts Tuesday and will hit on wide-ranging issues facing the cell and gene therapy (CGT) space, from new therapeutic advances to safety concerns and regulatory considerations to manufacturing and commercialization. Big hitters in the CGT space will be there. Sarepta, whose gene therapy for Duchenne muscular dystrophy Elevidys received accelerated approval in June 2023, will be presenting, as will CRISPR Therapeutics, which in the last few months brought to market Casgevy, its Vertex-partnered CRISPR-based therapy, for sickle-cell disease and beta thalassemia. There will also be plenty of big names, including Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. Marks will be speaking in two sessions at ASGCT 2024, one on global regulatory convergence and the other on the regulation of CGTs, from IND to BLA. Stay tuned for this week’s regular episode on Wednesday when we discuss what we’re seeing and hearing in Baltimore.