BioSpace

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

On the regulatory front, the FDA gave BridgeBio—and

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

Finally, the next generation of antibody-drug conjugates remains hot, with Danish biotech Adcendo reeling in $135M

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

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

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

With less than a week until Election Day, we unpack

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.