BioSpace

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.