BioSpace

On the sidelines of this year’s J.P. Morgan Healthcare Conference in San Francisco, BioSpace Senior Editor Annalee Armstrong sat down with Dannielle Appelhans, CEO of COUR, a member of BioSpace’s NextGen Class of 2025. Their discussion here, focused on the company’s long history leading up to its $105 million in series A funding at the start of 2024 and the possibility of an initial public offering in the future, among other things, represents the first of a special series of The Weekly focused on how NextGen companies are navigating the current business environment. HostAnnalee Armstrong, Senior Editor, BioSpaceGuestDannielle Appelhans, CEO, COURFor a complete list of NextGen 2025 companies, click here. The NextGen Class of 2025 is brought to you by Pliancy.

BioSpace’s Head of Insights Lori Ellis and Tom Whitehead, co-founder of the Emily Whitehead Foundation discuss the challenges navigated by cancer patients, weaving in their personal experiences with treatment and how they would encourage those undergoing treatment to speak up and advocate for themselves.This episode is presented by the ⁠Genscript Biotech Global Forum 2025.⁠Host⁠⁠Lori Ellis⁠⁠, Head of Insights, BioSpaceGuests⁠⁠Tom Whitehead,⁠⁠ Author, Keynote speaker; Co-Founder, Emily Whitehead FoundationDisclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

We saw more action on the M&A front this week as Novartisbought back its blood thinner abelacimab and the rest of Anthos Therapeutics for up to $3.1 billion and still ahead, a potential buyout of SpringWorks Therapeutics by Merck KGaA, which confirmed it’s in “advanced discussions” with the biotech. Meanwhile, Eli Lilly signed aglobal licensing deal for a MASH asset with South Korea’s OliX Pharmaceuticals.As Q4 and full-year 2024 earnings continue to roll in, BMS, Eli Lilly,Novo Nordisk, AstraZeneca, Eisai, Vertex and Gilead all reported their results in the past week. Notably, BMS continues to reorganize, now targeting an additional $2 billion in savings through 2027, and AstraZeneca axed two rare disease drugs from its $39 billion acquisition of Alexion. When it comes to revenue growth, Novo’s Wegovy and Lilly’s Zepbound continue to climb at a striking pace—a phenomenon that has at times driven the weight loss drugs into shortage and spawning a controversialshadow market of off-brand versions.Speaking of controversy, Regeneron is suing its Dupixent partner Sanofi, claiming the French pharma has failed to provide it with adequate information regarding sales of the blockbuster anti-inflammatory drug—which missed analyst expectations in Q4.On the clinical side, the pulmonary fibrosis space continues to see positive results, with Boehringer Ingelheim notching its second Phase III win in six months for nerandomilast. It wasn’t all good news, however, as Pliant Therapeutics suspended dosing and enrollment in a Phase IIb/III study of its idiopathic pulmonary fibrosis candidate, causing its stock to crash.Finally, BioSpace released a special edition of ClinicaSpace this week focused on the resurgent cardiovascular space—just in time for Valentine’s Day! Sign uphere to receive your copy.

U.S. political maneuverings are keeping biopharma on its toes. Tuesday, Robert F. Kennedy, Jr. passed a major hurdle as the Senate Finance Committee voted to advance his nomination for HHS secretary to a floor vote of the entire Senate. If confirmed, RFK Jr. could have an impact on myriad regulatory issues, including PDUFA negotiations. In other political news, Donald Trump announced his promised tariffs on Canada, Mexico and China—the last of which could have particular implications for biopharma companies looking to China for promising new drug candidates. Both were subjects of conversation as leading pharma companies—including Pfizer, Merck and Regeneron—reported their Q4 and full-year 2024 earnings. Notably, Pfizer is eyeing deals between $10 billion and $15 billion, while Merck took a 12% stock hit on Gardasil challenges in China and Regeneron issued its first-ever dividends. On the regulatory front, the FDA greenlit Vertex’s Jounavx last week as the first new drug for acute pain in more than two decades. Journavx leads a non-opioid pain space that is seeing significant momentum this year, with Tris Pharma, Algiax Pharamceuticals and SiteOne Therapeutics all announcing positive data in the past month. Meanwhile, in obesity news, the U.K.’s pharmacies regulator rolled out stricter guidelines for online pharmacies selling medicines including Novo Nordisk’s Wegovy and Eli Lilly’s Mounjaro, and an obesity-focused deal inked with Novo last year wasn’t enough to keep Omega Therapeutics afloat as the Massachusetts-based biotech revealed it is heading toward bankruptcy. Finally, a new CEO will be taking the reins at Takeda in 2026—but the selection of Julie Kim is an exception in an industry still struggling for gender parity at the top leadership ranks.

BioSpace's Head of Insights Lori Ellis and Tom Whitehead sit down and discuss cell and gene therapies from the perspectives of patients. What do caregivers and patients consider to be risks when lives are on the line? This episode is presented by the Genscript Biotech Global Forum 2025. Host ⁠Lori Ellis⁠, Head of Insights, BioSpace Guests ⁠Tom Whitehead,⁠ Author, Keynote speaker; Co-Founder, Emily Whitehead Foundation Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

In the week since Donald Trump took office, he’s caused quite the stir with healthcare-centered moves that include ordering the Department of Health and Human Services to stop communications, hiring and travel and announcing that he would withdraw the U.S. from the World Health Organization. Wednesday, the U.S. Senate Finance Committee convenes to vote on the controversial nomination of Robert F. Kennedy, Jr. for health secretary—a vote that Jefferies analysts said they expect to be “close.” Biogen continues to grab headlines this month, as the latest chapter in the Sage saga saw the smaller biotech rejecting its neuro partner’s unsolicited buyout offer. Meanwhile, Biogen laid off an undisclosed number of employees from its research unit, just as a higher dose of its Ionis-partnered spinal muscular atrophy therapy Spinraza was accepted for review by both the FDA and EMA. Elsewhere, the weight loss space continues to click on all cylinders, with Versant Ventures debuting its newest obesity biotech Helicore Biopharma on Tuesday with $65 million in series A funds, and two obesity-focused companies, Aardvark Therapeutics and Metsera, seeking entry to the public markets. These up-and-comers will have to compete against the likes of Eli Lilly and Novo Nordisk, the latter of which reported data last week showing that its next-gen obesity drug amycretin could elicit up to 22% weight-loss. And Veru announced that its enobosarm could significantly improve the quality of weight loss in seniors also taking Novo’s Wegovy. Another busy therapeutic space is Duchenne muscular dystrophy, where analysts predict a lot of action in the next couple of years, with a number of data readouts and regulatory submissions. And finally, Annalee Armstrong caught up at JPM with Novavax CEO John Jacobs, who said the vaccine maker is at a pivot point.

Donald Trump was sworn in as the 47th president of the United States on Monday, bringing with him a host of healthcare nominees and potential changes to the FDA, M&A and drug pricing. The new administration was a key focus point at the 2025 J.P. Morgan Healthcare Conference last week, where Annalee Armstrong spoke to executives from several companies about what they’re expecting from a second Trump term. Also at JPM, Mirador Therapeutics CEO Mark McKenna boldly predicted that 2025 would see the return of the megamerger. Speaking of M&A, Annalee spoke with leaders from Biogen, who declined to address the company’s unsolicited takeover bid for Sage Therapeutics. For its part, Sage sued Biogen last week, “seeking preliminary injunctive relief to enforce a standstill agreement.” Meanwhile, Gilead CEO Daniel O’Day addressed the split of Galapagos—a company Gilead has poured more than $5 billion into since 2019. Check out more personal stories from JPM here. In the obesity space, Novo Nordisk reported data from a Phase III trial showing that a high dose of Wegovy elicited more weight loss than the approved regimen—but still fell short of results posted by Eli Lilly’s Zepbound. Relatedly, Wegovy and sister drug Ozempic are both on the list of the next fifteen drugs whose prices could be negotiated under the Inflation Reduction Act. Finally, the FDA on Friday made its first high-profile decision of the year, greenlighting AstraZeneca and Daiichi Sankyo’s Dato-DXd—now Datroway—to treat certain types of advanced breast cancer. The approval is the first for the highly touted antibody-drug conjugate, which is also under FDA review for EGFR-mutated non-small cell lung cacner. Analysts expect 2025 to be a pivotal year for the ADC.

In this bonus episode, BioSpace's vice president of marketing Chantal Dresner and careers editor Angela Gabriel take a look at Q4 job market performance and what we expect to see ahead. We dive into year-on-year data and recent job trends. Want to receive our latest quarterly job market reports? Subscribe to Career Insider for our job market updates, job trends, career advice and more.

In this episode of Denatured, Dr. Peter Marks M.D., PhD. gives his thoughts on the future of cell and gene therapies. He speaks candidly about opportunities and realities, manufacturing and its role in efficacy and the CGT runway. Dr. Marks was keynote speaker at the GenScript Biotech Forum in San Francisco this week. Host ⁠Lori Ellis⁠, Head of Insights, BioSpace Guest ⁠Peter Marks M.D., PhD.⁠, Director, Center for Biologics Evaluation and Research (CBER)

The J.P. Morgan Healthcare Conference kicked off Monday with a flood of high-value deals, reinvigorating sentiment across the biopharma industry. Johnson & Johnson made the biggest splash, acquiring neurology leader Intra-Cellular Therapies for $14.6 billion, while GSK picked up precision therapy specialist IDRx for $1B upfront and Eli Lilly laid down up to $2.5 billion for Scorpion’s PI3Kα inhibitor program. Meanwhile, the immunology and inflammation space continues to fire on all cylinders as Gilead invests up to $1.7 billion for LEO Pharma’s preclinical oral small molecule STAT6 program. And those are only the deals accepted by both parties. Prior to the conference, Biogen offered to acquire its struggling neuro partner Sage Therapeutics for around $469M. The proposal follows a catastrophic run for Sage, which has seen its shares fall more than 90% in the past two years. 2024 sales and earnings forecasts have also generated attention this week, with Sarepta reporting that Duchenne muscular dystrophy gene therapy Elevidys beat analysts’ expectations in the fourth quarter, and Eli Lilly projecting a full-year revenue miss driven largely by lower-than-expected sales of GLP-1 blockbusters Zepbound and Mounjaro. As expected, obesity has been a hot topic at JPM, with Pfizer CEO Albert Bourla announcing that his company is going “all in” in the space. This follows new FDA guidance revealed last week recommending a minimum weight loss threshold for drug developers. Among the many companies taking notice is newcomer Verdiva Bio, which launched last week with more than $410 million in opening funds. Also debuting last week was Kardigan, which raised $300 million to tackle heart disease. Kadigan joins a resurgent cardiovascular space, where several companies—including those developing gene therapies—are targeting myriad diseases. Finally, BioSpace senior editor Annalee Armstrong caught up with Daphne Zohar, CEO of BioSpace NextGen 2025 company Seaport Therapeutics Daphne Zohar, who offered her thoughts on the current state of the neuropsychiatric space.

TEASER: This episode is little sneak peak into upcoming podcasts as well as topics that will be discussed at the 2025 GenScript Biotech Global Forum on January 15 in San Francisco. Listen in as Peter Marks and Tom Whitehead share their thoughts on the CGT space for 2025. Register for the event here. Host Lori Ellis, Head of Insights, BioSpace Guests Peter Marks M.D., PhD., Director, Center for Biologics Evaluation and Research (CBER) Tom Whitehead, Author, Keynote speaker; Co-Founder, Emily Whitehead Foundation

Happy New Year! BioSpace released our NextGen: Class of 2025 this week, highlighting 25 biopharma startups to watch this year. The companies on this list are not afraid of a challenge, wading into some of biopharma’s most competitive therapeutic spaces. As analysts predict an uptick in M&A in ’25, could some of them be potential targets? One NextGen 2025 company that could already be fielding calls is Metsera, which reported promising Phase II data for its investigational subcutaneous GLP-1 therapy on Tuesday. The GLP-1 space continues to garner significant attention, with both Eli Lilly and Novo Nordisk seeking to protect their drugs against competition from compounders. This strategy makes a lot of sense as the sizzling class continues to expand into heart disease, sleep apnea and metabolic dysfunction-associated steatohepatitis, among other indications. On the regulatory side, the FDA approved 55 novel medicines in 2024—including a few inaugural company approvals—and is looking ahead to several key PDUFA dates in Q1, 2025. Meanwhile, the agency released three new guidance documents seeking to provide clarity around confirmatory trials supporting accelerated approvals, the use of AI in drug development and the use of tissue biopsies in clinical trials. Finally, BioSpace senior editor Annalee Armstrong heads to the J.P. Morgan Healthcare Conference next week as biopharma seeks an M&A spark to improve the currently a gloomy sentiment. Stay with us for all the biggest news coming out of JPM.

This conversation focused on a few remaining topics discussed at length at Jefferies late last year, with the same topics anticipated to make an appearance at JPM. In this episode, our guests the industry’s need to catch up with women’s health issues and the innovative lead the APAC region has taken in clinical trials. Host ⁠⁠Lori Ellis⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Miguel Forte⁠⁠, President, International Society for Cell & Gene Therapy; Board Member, ARM; CEO and Board Member, Kiji Therapeutics ⁠⁠Ali Pashazadeh,⁠⁠ Founder, Treehill Partners

2025 is set to change the industry. The new administration is poised to challenge many existing processes. The question is whether they will be based on data-driven analysis or ideologies and beliefs. Our guests address this concern along with the existing challenges CEOs face, particularly manufacturing processes. Additionally, they discuss weight loss drugs, focusing on GLP-1s, with medical precision. Host ⁠Lori Ellis⁠, Head of Insights, BioSpace Guests ⁠Miguel Forte⁠, President, International Society for Cell & Gene Therapy; Board Member, ARM; CEO and Board Member, Kiji Therapeutics ⁠Ali Pashazadeh,⁠ Founder, Treehill Partners

Note: BioSpace is taking a break for the holidays. The next episode of The Weekly will air Jan. 8, 2025. Happy New Year! Novo dominated headlines over the last week, with a kidney disease label expansion for Novo Nordisk’s Ozempic in Europe but a newly flagged safety risk for the drug, with a Danish regulator requesting that the EMA review reports of a rare eye disease in patients who took the GLP-1. Meanwhile, over the weekend Novo Nordisk’s parent company, Novo Holdings, and CDMO Catalent announced that they had received all regulatory clearances to close the proposed $16.5 billion acquisition. The deal, which will see Novo Holdings sell three sites to Novo Nordisk, is the biggest manufacturing investment this year but certainly not the only, with supply chain becoming a focus for biotech and Big Pharmas alike. Meanwhile, 2024 proved to be a snacky year for M&A activity and a tough environment for IPOs, with this year’s newly public biotechs mostly seeing their stock prices plummet after entering the public markets. Moderna, while not new to the markets, has similarly seen its stock tank. President-elect Donald Trump’s nomination of well-known anti-vaccine advocate Robert F. Kennedy Jr. to head the Department of Health and Human Services certainly didn’t help. On the clinical research side of the industry, 2024 saw its fair share of flops and victories. One of the most striking in each category was the failure of AbbVie’s emraclidine. The Big Pharma acquired the schizophrenia hopeful with its nearly $9B Cerevel buy late last year. The flops came not long after the approval of BMS’ Cobenfy, which came out of the even-bigger purchase of Karuna and was the first new schizophrenia medication in more than three decades.

This week, GSK and Gilead and Arcellx presented key data at the American Society of Hematology (ASH) annual meeting as they vie for a competitive advantage in multiple myeloma. Meanwhile, Vertex unveiled positive long-term data for its CRISPR Therapeutics–partnered gene therapy Casgevy in sickle cell disease—results BMO Capital Markets analysts said should help Casgevy keep its edge over bluebird bio’s Lyfgenia. On that note, Casgevy and Lyfgenia have a new outcomes-based payment model after the Centers for Medicare and Medicaid Service (CMS) said both companies have agreed to participate in a voluntary program to improve access to the gene therapies. Speaking of access, Eli Lilly and Novo Nordisk both announced significant manufacturing investments aimed at shoring up production of their diabetes and weight loss blockbusters tirzepatide and semaglutide. And in related news, the European Commission gave its blessing to Novo Holdings’ controversial acquisition of contract manufacturing firm Catalent. Elsewhere, AbbVie got a much-needed win for Cerevel-acquired Parkinson’s disease therapy tavapadon—a month after the deal’s cornerstone asset emraclidine failed in schizophrenia—while uniQure announced it has aligned with the FDA on “key elements” of the accelerated approval pathway for its investigational gene therapy for Huntington’s, AMT-130.

“Cautious optimism” is circling once again as the industry approaches 2025. However, many companies are treading water, barely holding on as the market continues at a slow pace. Ali Pashazadeh and Miguel Forte have an open discussion about the state of the market. Host Lori Ellis, Head of Insights, BioSpace Guests Miguel Forte, President, International Society for Cell & Gene Therapy; Board Member, ARM; CEO and Board Member, Kiji Therapeutics Ali Pashazadeh, Founder, Treehill Partners

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.