BioSpace

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.