JPM2024: On the Ground in San Francisco

Pictured: The Westin St. Francis Hotel in Union Square San Francisco/Tyler Patchen

BioSpace is in San Francisco, providing updates on the latest news and industry developments from JPM24. Stay with us!

Updated Jan. 12, 10:30 a.m. EST/7:30 a.m. PST:

JPM2024 Recap: Cautious Optimism

BioSpace News Editor Greg Slabodkin gives his takeaways from this year’s J.P. Morgan Healthcare Conference. After a challenging 2023, the biopharma industry appears to be turning a corner, with excitement about an improved outlook for the sector. Certain areas in particular stood out, including cell and gene therapy, artificial intelligence (AI), neuroscience and GLP1. At the same time, attendees acknowledged several challenges that lay ahead, including policy considerations such as the ramifications of the Inflation Reduction Act on drug development.

“It’s not all downhill from here—significant hurdles still face the biopharma industry—but the sector feels invigorated to take on the challenge,” Slabodkin wrote. “Let the journey begin!”

Takeda Touts Promise of Latest Narcolepsy Candidate

Saftey issues may have quashed the development of Takeda’s earlier orexin candidate, but Elena Koundourakis says the company “bounced back big time.” Koundourakis, who leads the company’s orexin franchise, told BioSpace that the company quickly shifted its focus from the previous orexin candidate, TAK-994, to its latest orexin product, TAK-816, and will soon have results to share from an ongoing Phase II trial. “It has the potential to serve [an] unmet need in several indications,” Koundourakis said.

Cancer Biotech Executives Look Ahead to the Coming Year

BioSpace sat down with several executives in the cancer space to talk about their coming year and the critical role of JPM. Olema Oncology CEO Sean Bohen said that despite the headwinds of 2023, his company was able to raise money and saw its stock price rise. For the wider biotech world, Bohen is also hopeful that successes in the beginning of the year will attract more investors and capital to the cancer sector. But he cautioned that the “fundamental challenge” cancer biotechs faced in 2023 hasn’t changed much.

IRA Favors Biologics Over Small Molecule Drugs, Industry Analysts Say

The potential impact of the Inflation Reduction Act’s drug pricing provisions on small molecule drugs was on the minds of industry stakeholders in San Francisco this week for the J.P. Morgan Healthcare Conference. At the crux of the matter is a disparity in which biologics are spared from price negotiations under the Inflation Reduction Act (IRA) for 13 years following approval, while the grace period for small molecules is only nine years.

Panelists John Stanford of Incubate, Gaurav Gupta of J.P. Morgan Life Sciences Private Capital and Kirsten Axelsen of DLA Piper said at a Sunday session that the policy could deter investment in small molecule drugs and stifle the development of treatments for conditions such as cancer.

Sanofi May Dive Into Biopharma’s Hottest Space

Sanofi wants in on biopharma’s biggest trend. After the company’s GLP-1 drug failed a mid-stage trial nearly five years ago, Sanofi R&D head Houman Ashrafian told investors at JPM it may consider potential next-generation products that could be more targeted with fewer side effects, Endpoints News reported. “There are genetic signals on where to dig for obesity drugs 2.0,” Ashrafian said.

Several companies, including leaders Novo Nordisk and Eli Lilly, along with AstraZeneca, are already on the hunt for next-gen GLP-1 drugs that can reduce side effects and bolster current medications’ benefits. And personalized approaches to treating obesity and efforts to match patients with the right medicines are underway at academic institutions such as the Mayo Clinic.

VCs Confident in Biotech Activity for ’24

Panelists at JPM’s Biotech Showcase gave positive projections as the year begins and offered advice to those in the space. Andrew Lam, the principal of special investments at Ally Bridge Group, Gabe Cavazos, a senior managing director of investment banking at Leerink Partners and Maha Katabi, general partner at Sofinnova Investments, expressed cautious optimism about M&A and IPO activity in the year ahead and offered advice for companies on thriving in the current environment.

Madrigal Ready for Resmetirom Launch if FDA Says Go

Madrigal Pharmaceuticals is gearing up for a potential accelerated approval for its nonalcoholic steatohepatitis (NASH) drug, resmetirom, with CEO Bill Sibold telling investors at JPM that the company will be “ready to go” if the FDA gives the green light in March, Endpoints News reported.

Sibold called NASH “the veritable graveyard of drug development” but said Madrigal is “planning for success.”

Indeed, the space has seen a string of failures, the latest being the late-stage trial flop of Akero Therapeutics’ efruxifermin (EFX). This followed the high-profile failure of Intercept Pharmaceuticals’ obeticholic acid (OCA) tablets—which received a second FDA rejection in June 2023—due largely to safety concerns. Resmetirom hit both primary endpoints and a key secondary endpoint in its pivotal Phase III trial in December 2022.

Generative AI Will Become More Entrenched in the Life Sciences

Artificial intelligence—specifically its potential uses in biopharma—has commanded a lot of attention at the Biotech Showcase this week, and Wednesday was no exception as a panel hosted by Boston Consulting Group discussed how to best leverage generative AI.

Parry Bhatia, chief AI officer at GE Healthcare, told attendees that large language models, already being used in science and technology, will be further incorporated into the vast healthcare ecosystem.

A general theme at the panel was collaboration as John Doyle, global chief technology officer for healthcare and life sciences at Microsoft, said, “It’s not actually about the technology. It’s about us all collectively working together to solve the same problem.”

Regeneron Looks to Team Up with Novo on Wegovy Combo

Regeneron Pharmaceuticals is planning a clinical trial of a combo therapy involving its antibody drugs and Novo Nordisk’s Wegovy to see if it can help reduce the muscle mass loss seen with Wegovy and other GLP-1 agonists on their own. “We’ll be initiating our trial this year to test these muscle preservation agents in combination with semaglutide,” CEO George Yancopoulos said on Tuesday, according to Seeking Alpha. He referenced preclinical data on the silencing of the GPR75 gene, which he suggested could maintain muscle mass and prevent weight gain.

BioMarin to Resolve Supply Issues and Reach Bigger Markets

Alexander Hardy said his top priority as the new CEO of BioMarin is the commercial success of Voxzogo, the company’s dwarfism drug that received a label expansion in October to include younger children and not just those five years and older, Fierce Pharma reported. The therapy faced supply issues last year, but Hardy said those will be resolved by the middle of 2025, and the company will seek additional indications for Voxzogo. The second priority is Roctavian, BioMarin’s recently approved gene therapy for hemophilia A, Hardy noted, and beyond that will be some challenging decisions on which R&D projects to keep and which to cull.

“We’ve gotta be very disciplined through the early stages of research with our spend. We’ve gotta have high bars for success,” he said, according to Fierce.

AstraZeneca Eyes Five Big Disease Areas

Oncology, cardiovascular disease, rare diseases, renal and metabolism, respiratory and immunology, and vaccines and immunotherapies—these are the five disease areas that AstraZeneca intends to be a top-three company in by 2030, according to a presentation by CFO Aradhana Sarin, Fierce Pharma reported. Part of this optimism comes from 15 new therapies Sarin says could reach the market by that year.

“We know not everything’s gonna work out. But I think there are multiple elements where I don’t think we still get credit,” Sarin said, according to Fierce.

The pharma giant is improving its position in various sectors with big-ticket acquisitions. Last month alone, AstraZeneca struck two deals worth more than $1 billion and a third for $247 million. In November, the company inked a deal worth up to $2 billion for Eccogene’s oral GLP-1 prospect.

Seeing Sunshine, Halia Therapeutics Targets Series C

In December, Utah.–based Halia Therapeutics dosed the first patient in a Phase IIa study of its lead asset, HT-6184, in lower-risk myelodysplastic syndromes. In an interview at JPM, Halia CEO David Bearss told BioSpace the company is confident that the asset can show improvement. Ultimately, he said, Halia is looking for a positive start to the year.

“This year will be an amazing year for us to have three Phase II clinical programs,” Bearss said. “We’ll also file another [Investigational New Drug Application] for a program targeting neuroinflammation, Alzheimer’s and Parkinson’s, so that’ll be interesting to get back to where we started.”

Halia is also looking to close a Series C funding round, which Bearss said the company hopes to announce by the end of January. Halia also may be heading for the public markets, with Bearss expressing confidence that the market is on an upswing.

“Right now, there seems to be a little sun shining in our world in terms of an uptick in M&A activity and [the IPO window] may be creaking open,” Bearss said, adding that “lots of people” are indicating they will give that avenue a shot. “For us as a smaller company that is still raising private capital, that’s good news . . . because we can point and say, ‘Listen, the cycle is coming back, the windows are opening, and partners are looking for opportunity.’”

Triumvira Unconcerned About FDA CAR T Probe

In an interview with BioSpace, Triumvira president and chief operating officer Rob Williamson said oncology-based cell therapy companies are not concerned about the FDA’s investigation into malignancies potentially caused by CAR T cell therapies. However, he noted that “cell therapy within biotech is still in the penalty box,” and indicated that companies who do not differentiate may be seen less favorably than other biotechs in 2024.

Williamson said the past few years have been an “awful” time for private and public biotechs but called the recent uptick in deals during the fourth quarter of 2023 “heartening.”

Big Tech is Coming for AI—How Will it Affect Biotech?

A panel on artificial intelligence and machine learning held by the Biotech Showcase Tuesday focused on the surge of Big Tech companies into both AI and biotech. “Big Tech is coming for AI and it’s coming in a big way,” said panel moderator Beth Rogozinski, CEO of Oncoustics.

However, Atomwise CEO Abraham Heifets noted that there is a “mismatch of business models” between Big Tech and biotech, and said the recent deals between Alphabet’s Isomorphic Labs and Eli Lilly and Novartis look “like traditional tech mentality.”

Meanwhile, Debiopharm Innovation Fund Managing Director Marc Cikes said, “The impact of AI for drug discovery is still largely unknown,” citing the structure of deals between Big Tech and AI companies and the public market valuation of AI-drug discovery companies.

Lilly Looks to Partner with Smaller Biotechs

Eli Lilly CEO David Ricks told a roomful of people from the biopharma industry that “we’re open for business on external innovation,” Fierce Biotech reported. And that doesn’t mean purchasing drug candidates and dumping the rest of the company—in particular, the people—he clarified.

“Most of the Big Pharma companies in our industry, and certainly Lilly for the longest time, thought about acquisition as grabbing an asset. Essentially, go into a company, take their work, thank them very much,” Ricks said. “We think about now, acquisition is not just assets, but people and methodologies and ways to make even more medicines.” Ricks declined, however, to provide specifics on when the industry might see Lilly’s next move on the M&A front.

Merck Doesn’t Fear Keytruda Patent Cliff

Merck’s blockbuster drug Keytruda will lose its patent protection in 2028, but CEO Rob Davis said “it’s just another year,” Fierce Pharma reported. While he acknowledges that the business will inevitably take a hit, he said the company is focused on its wide-ranging portfolio and growth over the next couple of decades. Specifically, Davis projected that by the mid-2030s the company could reach more than $20 billion in sales from its oncology drug candidates alone, up from Merck’s projection at last year’s JPM conference of $10 billion.

Novo Nordisk Banks on Wegovy, Welcomes Competition

Lars Fruergaard Jorgensen, CEO of Novo Nordisk, said on Tuesday that there is room for both injectable and oral treatments in the obesity market, which is projected to reach $100 billion by 2030, adding that he “welcomes competition” in the space, Reuters reported. Jorgensen noted that he thinks patients are more likely to stay on Novo’s obesity drug Wegovy longer than patients have taken obesity drugs in the past, in part due to the drug’s effectiveness. “We know from all medical interventions, stay time is not like a 100% . . . but I think we will see a significantly higher stay time than what we have seen so far on obesity treatment.”

Catalent also Banks on Wegovy

On Tuesday, Catalent CEO Alessandro Maselli highlighted the contract manufacturer’s role in producing GLP-1s such as Wegovy and Eli Lilly’s Zepbound, leading to a surge in the company’s stock prices, which climbed to the highest level in more than four months, Seeking Alpha reported. He noted that Catalent estimates it will receive less than $100 million in 2024 income from manufacturing these therapies but said that this revenue stream could one day amount to $500 million, as the company has plans to expand its manufacturing capacity over the next couple of years.

BioNTech Looks Ahead to 2025 as Revenue Falls

BioNTech continues to face the COVID-19 cliff, with projected 2024 revenue down to about $3.3 billion from the more than $4.3 billion it estimated for 2023, but the company expects 2025 to show growth once again, according to a JPM presentation on Tuesday, Reuters reported. According to the presentation, this projection is based on higher prices for BioNTech’s COVID shots, developed and marketed in collaboration with Pfizer, as well as proceeds from additional mRNA vaccines in development for other respiratory diseases.

In addition, CEO and co-founder Ugur Sahin said the company plans to renew its focus in oncology, with at least 10 late-stage cancer drugs yielding results by the end of the year. “We continued our vaccine leadership in the fight against COVID-19 and significantly expanded our mid- and late-stage oncology pipeline.”

Sanofi Focuses on Existing Pipeline While Open to M&As

In what is projected to be a year of many mergers and acquisitions, Sanofi will need to see some “game-changing science” to jump on the M&A bandwagon, according to CEO Paul Hudson. “It has to be something that is really fundamentally going to change something for patients,” he said at JPM on Tuesday, Endpoints News reported. “But we’re open-minded.”

Meanwhile, Hudson said, the company is focused on its existing therapies, largely in the immunology and inflammation space, which the company projects can bring in upward of $2.2 billion. “I’m extremely proud of how far we’ve come. It feels like a lot longer than four years that we’ve been on this journey. But we are becoming a new organization,” he said. “It feels very good to be leading Sanofi at this exact moment.”

Illumina’s Luck Is Changing: CEO

Illumina’s luck may be changing—at least that was the tone struck by CEO Jacob Thaysen during his presentation at JPM. “Clearly, we’ve been through some rough times here over the past few years, but I see a strong commitment for everyone in Illumina to get beyond that and get back to the growth that I truly believe we are just in the starting point of,” Thaysen said, according to Endpoints News. And while Illumina announced last month that it would divest Grail, after a long antitrust battle, the “how” is still up in the air as CFO Joydeep Goswami said during the presentation that “all options are on the table” for the divestiture, from a sale to a spinout.

Also on Tuesday, Illumina reported preliminary earnings of $1,115 million for Q4 2023, up 3% over the same period in 2022.

Roche Bullish on ‘Diverse Portfolio and Broad Pipeline’

In a corporate presentation at JPM Tuesday, Roche executives expressed excitement about the company’s blood cancer portfolio, saying that recently approved T-cell engagers Lunsumio and Columvi will continue to fuel a turnaround after a period of declining hematology sales, Fierce Pharma reported.

Roche estimates that sales of its hematology products will see a 14% compound annual growth rate through 2026, “driven by a diverse portfolio and broad pipeline,” the Swiss pharma stated in its corporate presentation.

Roche was high on its overall portfolio as well, boasting 16 blockbuster drugs in 2023, more than double its 2022 total of 7. The company touts a Number 1 ranking drug in both neurology and hemophilia A, according to the presentation.

AI, Neuroscience and GLP-1 Medicines Will Continue to Make Headlines: Expert Panel

Tuesday Morning, a panel hosted by Boston Consulting Group (BCG) showcased expert perspectives on the trends to watch for in 2024. Experts from Merck and CVC, among others, told the room that advances in AI and general AI application will become more prevalent this year, a theme that will continue into the future.

“This convergence with technology and AI coming into the space is having a profound impact on medicine,” Cathrin Petty, managing director and head of Healthcare at CVC, said.

Neuroscience and GLP-1 medicines will continue to make headlines this year, the panelists said, and while early-stage companies will still struggle to find funding, this market should see some prioritization in 2024.

Sarepta CEO Confident in Elevidys Label Expansion

Ever since Sarepta Therapeutics won accelerated approval for Elevidys, the first gene therapy for Duchenne muscular dystrophy, in June 2023, the company has aimed to expand its label from patients for 4 to 5 years who are able to walk to all ambulatory patients. On Tuesday at JPM, Sarepta CEO Douglas Ingram said the company has an opportunity to get that restrictive labeling removed “not in years, but in months,” Seeking Alpha reported.

In December, Sarepta submitted an efficacy supplement for the gene therapy’s label to expand the labeling, and Ingram said he expects the FDA to conduct a priority review on the submission without an Advisory Committee meeting, per Seeking Alpha. Ingram projected a six-month review period and target action date in August 2024.

Elevidys, which in October missed the primary endpoint in a Phase III trial, brought in $200.4 million for its maker in 2023, according to a report released by Sarepta prior to its JPM presentation.

Codagenix Focused on Nasal Vaccines, for COVID and Other Diseases

Codagenix is staying the course in developing its live nasal vaccines for COVID-19 and other infectious diseases, CEO Paul Grint told BioSpace. He said the company initially targeted respiratory diseases such as influenza and RSV, but pivoted to COVID-19 after the pandemic broke out. “We very quickly generated a COVID vaccine, did some small-scale Phase I studies, and showed intriguing data.” Grint added that Codagenix hopes to release more clinical data this year.

Cell and Gene Therapy Sector Project to Grow

This year could see up to 17 new cell and gene therapies approved in the U.S. and Europe, according to a presentation by the Alliance for Regenerative Medicine (ARM) at the Biotech Showcase 2024 investor conference on Monday. That’s more than double the approvals seen in 2023, which was already a record-setting year, with seven U.S. approvals and one in the E.U.

Nearing the finish line in 2024 are five gene therapies for rare genetic diseases, as well as the first-ever adoptive cell therapy for solid tumors, an allogeneic T-cell therapy and additional therapies to treat hemophilia A, hemophilia B and dystrophic epidermolysis bullosa. “We’re optimistic that we’ll see a lot of these pulled through to the finish line,” ARM CEO Tim Hunt said.

Johnson & Johnson CEO on Sales Predictions

Johnson & Johnson CEO Joaquin Duato on Monday addressed discrepancies between the company’s projections and those from Wall Street analysts, according to Fierce Pharma. He noted, for example, that J&J expects 2027 sales of its new multiple myeloma therapy Tecvayli to be 25% higher than analysts estimated, and Talvey’s sales for that year to be double Wall Street predictions, with both products bringing in upward of $5 billion. Similarly, J&J’s cancer combo, Rybrevant and lazertinib, could be worth twice as much as analysts forecast, Duato said, with sales of that therapy also reaching the $5 billion mark.

BMS CEO on the Company’s Big Buys

Last month, Bristol Myers Squibb bought out Karuna Therapeutics for $14 billion and RayzeBio for $4.1 billion. But in his JPM presentation on Monday, new CEO Chris Boerner was more focused on the company’s overall pipeline, Fierce Pharma reported, stating that BMS could bring 16 new products to market before the end of the decade. “I don’t think our pipeline has ever been richer,” Boerner said.

Biogen CEO on Leqembi’s Insurance Coverage

Biogen CEO Christopher Viehbacher said on Monday that getting insurers to pay for Leqembi, the newly approved Alzheimer’s treatment developed in partnership with Eisai, “has not been an issue,” BioPharma Dive reported. But he said the drug’s launch does face challenges, both due to the number of people who could benefit from the therapy and the “lengthy sales cycle”; Viehbacher noted that it can take two to three months for patients to get in with a neurologist and be prescribed Leqembi.

“Up until now, there hasn’t really been any disease-modifying treatments for Alzheimer’s . . . so now there’s kind of a rush,” he said. “I think the primary care physicians will play a role at some point, because patients coming in [are having trouble finding] an appointment for a neurologist.”

Pfizer Eyes Obesity Market After Phase II Flop

Pfizer may have dropped one weight-loss drug candidate in December, but CEO Albert Bourla said on Monday that the company still has its sights set on the obesity market, which is projected to reach $100 billion by 2030, Reuters reported. While the company is unlikely to shell out billions to secure a therapy in the later stages of development, Bourla said, it is on the lookout for earlier-stage candidates it can scoop up. “Pfizer’s position is that we believe that obesity is a place that we have the ability to play and win. So we will have to play.”

Ultragenyx to Close $50 million Series A

California-based Ultragenyx will soon secure $50 million in Series A funding for Amlogenyx, its Alzheimer’s gene therapy spinout, CEO Emil Kakkis told Endpoints News at JPM on Monday. “We have a lead investor signed on, and we have several investors who are on board. . . . We’re trying to get the last two, three people to finish it.”

Gilead CEO Emphasizes HIV and Oncology Products

During Gilead’s presentation at the J.P. Morgan Healthcare Conference this morning, CEO Dan O’Day said the company is off to a “good start.” Keep reading for more of O’Day’s comments.

“Let me just say that I think this is an incredibly important year for the company. . . . The build that we’ve been doing over the past four years is really coming to fruition . . . and importantly, this becomes a very catalyst-rich year for us. I may repeat this a couple of times: we have close to two dozen clinical trials reading out this year, of which five (are) Phase III readouts.

“We’re coming off a really strong momentum and series of execution of quarters over the past years and 2023. We’re entering this year with this solid commercial execution of validation on the medicines that are available for patients today. On top of that, we have many new updates coming across our portfolio.”

“Oncology is now annualizing over $3 trillion at a run rate . . . with a clear opportunity for future growth and promising clinical momentum.”

“Perhaps the core and the legacy of Gilead, the HIV portfolio, continues to perform very well with medicines on the market today.”

“We’re very confident [in] a durable growth output for the future . . . Gilead has no significant patent expirations between now and the early part of the next decade.”

Cell and Gene Therapy Continues to Thrive

The Alliance for Regenerative Medicine (ARM)’s annual Cell & Gene State of the Industry Briefing is taking place Monday morning at the Biotech Showcase 2024, with updates on the sector’s recent advances and outlook for the year. We’ll see what data ARM presents. In the meantime, healthcare industry professionals scored cell and gene therapy (CGT) as the industry trend to have the greatest impact on the pharma industry in 2024, according to recent survey results from GlobalData, which forecasts the worldwide CGT market will reach $80 billion by 2029, with oncology continuing as the lead indication and area of major development.

Politics Highlight Start of JPM

The Biden administration’s policies impacting the biopharma industry, including implementation of the Inflation Reduction Act, intellectual property challenges and mergers and acquisitions, took center stage on Sunday in San Francisco ahead of the start of the J.P. Morgan Healthcare Conference.

Sunday’s event, part of JPM Week 2024, was hosted by Incubate, a lobbying group for venture capital firms in the life sciences. Incubate Executive Director John Stanford said that policy is very much of interest to stakeholders attending this year’s JPM conference—a significant change from previous years.

Stanford said that Incubate’s focus this year is on three policy areas: the implementation of the Inflation Reduction Act (IRA)’s Drug Price Negotiation Program, the Biden administration’s intention to invoke so-called march-in rights to take back patents of specific expensive drugs that rely on federally funded research, and the “changing dynamics” at the Federal Trade Commission related to mergers and acquisitions.

Under the IRA, the Centers for Medicare and Medicaid Services in August 2023 announced the first ten drugs covered under Medicare Part D that will be subject to price negotiations. By Sept. 1, 2024, CMS will publish maximum fair prices agreed upon between the agency and participating drug companies, which will go into effect in 2026.

“These prices will be announced in September of a presidential election year. I would be hard-pressed to imagine the Biden administration announcing fairly meager cuts,” Stanford said. “I think we will see draconian cuts and I think the political pressures will drive some of that.”