BioSpace

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.

This is the second episode of Denatured's discussion on diversity, equity and inclusion (DE&I). Data is the essential piece for both science and technology. The lack of DE&I data is becoming even more imperative as we move towards personalized medicines. Without understanding why there are differences in outcomes, the industry will continue to see worsening outcomes. However, DE&I is still not at the forefront of the mind during clinical trial designs, which influences not only the training but the application of AI models. Additionally, what this means not only for life sciences but also for healthcare providers as more of the healthcare industry adopts AI to assist with patient care. 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

The biggest news of the last week was easily the expanded approval of Sarepta’s Duchenne muscular dystrophy gene therapy, Elevidys. CBER Director Peter Marks again overruled FDA staff members and review teams to grant the therapy full approval and a broad label expansion despite its missing the primary endpoint in a Phase III confirmatory study. This approval has us thinking about other big FDA decisions to watch this year, first and foremost, Eli Lilly’s anti-amyloid antibody donanemab. If given the green light, donanemab will be a direct competitor to Biogen and Eisai’s Leqembi, also a disease-modifying anti-amyloid antibody. Beyond that, Verona Pharma is expecting a decision Wednesday on ensifentrine, which could be the first novel mechanism for chronic obstructive pulmonary disease in over a decade, and in August, the FDA is set to decide on Lykos’ MDMA-assisted PTSD therapy, which an advisory committee voted against earlier this month. Then this week, Alnylam scored big with a Phase III win in transthyretin amyloidosis with cardiomyopathy (ATTR-CM). In what Alnylam CMO Pushkal Garg called “overwhelmingly positive data,” Amuvttra significantly lowered the risk of death and recurrent cardiovascular events in patients with ATTR-CM. Meanwhile, biopharma conference season continues with the American Diabetes Association’s annual conference held this past weekend. Eli Lilly’s blockbuster drug Zepbound “significantly improved” disease severity in patients with obesity and sleep apnea, and Altimmune racked up a Phase II victory for pemvidutide, which was effective at helping patients lose weight while retaining lean muscle.

BioSpace's ⁠Lori Ellis⁠ and ⁠Chantal Dresner⁠ are bringing live updates from ⁠#DIA2024⁠ in San Diego this week where we've been attending sessions on trial design, digital twins, cell and gene therapy regulation, clinical research workforce trends, professional development and many more. We discuss some of our key takeaways and event highlights, including Lori's exclusive interview with CBER director Dr. Peter Marks. Hosts ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠⁠⁠BioSpace⁠⁠⁠   Chantal Dresner, VP of Marketing, BioSpace⁠⁠⁠

Diversity, equity and inclusion (DEI) continues to be a challenge for drug and medical device developers. While there has been some improvement in clinical trials, our guests note that unless there is a commercial or regulatory need, DEI is still an afterthought for many developers when designing clinical trials. Host ⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace Guests ⁠⁠Ali Pashazadeh⁠⁠, CEO,Treehill Partners 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 Todd Rudo, Chief Medical Officer, Clario