BioSpace

Summit Therapeutics made headlines this weekend at

Last week, BioMarin held a public address intended to calm anxious investors after rounds of layoffs and pipeline

And on the weight loss front, Terns Pharmaceuticals is

Eli Lilly shook up the weight loss market again last week,

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

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

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%.