Sweat Equity - Your Weekly Biotech News Fix | Ep. 554
The Week of 1/13/2025
Hello Avatar! Another week of biotech news is in the history books. We are proud to bring you the latest edition of Sweat Equity, our weekly biotech news fix. The content is designed to be consumed as a quick scroll to bring readers up to speed on key events from the week. For those of you interested to read a bit more, we provide links to the source articles.
We are now publishing 7x per week according to the following cadence:
Mondays: Stock Analysis & Biotech Catalysts
Tuesdays: Biotech hot topics (X-article)
Wednesdays: Podcast
Thursdays: Public & Private Biotech Markets
Fridays: Your Weekly Biotech Fix
Saturdays: Podcast
Sundays: Biotech Strategic Topics
We are also publishing unique content on X - be sure to follow up if you are not already @BowTiedBiotech. And to check-out the archive of our work on X you can find it on our website at: BowtiedBiotech.subtack.com/x-articles.
SUBSCRIBE TO PODCAST HERE:
Please help spread the work by subscribing and hitting the share button if you are enjoying content!
BIOTECH NEWS UPDATES FOR THE WEEK 1/13/25
‘The bar has risen’: China’s biotech gains push US companies to adapt
The advancement of China's biotech sector is prompting U.S. companies to rethink their strategies due to heightened competition. U.S. entrepreneurs are discovering that Chinese biotechs are often already working on potential drug targets they have identified. This has been affirmed by David Li of Meliora Therapeutics, who noted the rapid pace of innovation and high-intensity competition among startups during his visits to Shanghai and Suzhou. The trend of major pharmaceutical companies increasingly licensing experimental drugs from China is placing more pressure on U.S. companies to differentiate themselves and is shifting the dynamics of the biotech industry.
The shift has been rapid, with licensing deals between China and western countries increasing significantly over the past two years, despite growing competition between the U.S. and China in the biotech sector. There is a consensus among executives and investors interviewed at the J.P. Morgan Healthcare Conference that this trend will not only continue, but accelerate, forcing U.S. biotechs to work harder to maintain their competitive edge. Paul Hastings, CEO of cell therapy maker Nkarta and former chair of the U.S. lobbying group the Biotechnology Innovation Organization, agrees this does show that the U.S. is losing its edge in the biotech sector. As an example, the innovative drug ivonescimab was developed by Akeso Therapeutics, a China-based company.
The Rise of China Innovation in the Time of Trump
During the incoming Presidency of Donald Trump, experts have emphasized the importance of treating the pharmaceutical industry differently amidst potential trade wars with China. Notably, drugs developed in China have gained mainstream acceptance, with pharmaceutical executives viewing these assets as innovative and superior options instead of mere replication. Despite the threat of tariffs, most experts maintain confidence in the new administration’s understanding and recognition of the potential benefits that these innovative drugs could bring to patients.
However, there is still uncertainty surrounding potential tariffs on Chinese imports which has not been clearly defined, particularly in the field of intellectual property and imported drugs for development. Companies are, therefore, advised not to proceed as they traditionally would in negotiating deals. There are concerns that assets licensed from China might face punitive measures down the line, especially if the FDA rejects them, like an Eli Lilly–partnered drug that had to undergo a new study after being licensed from a Chinese company.
Roche’s new deals head tries to navigate a more ‘complicated’ and ‘expensive’ biotech world
Roche, a major Swiss pharmaceutical company, is challenged with competition that potentially threatens its older, successful biologic drugs that anticipate a drop in sales by approximately $8 billion within three years. Despite these obstacles, Roche officials have publically showed optimism by restructuring the company and reprioritizing research ventures. They have ensured their shareholders that the potential financial losses will be offset by new products and some experimental medicines. These new products are a mix of internally developed assets and acquisitions from recent purchases.
Roche has excelled in acquisitions, with over $1 billion sealed in three deals since the summer of 2023, including the $7.2 billion purchase of Televant. Moreover, the company has also embarked on several research and licensing agreements. Some of these partnerships include the collaboration with AI chip maker Nvidia, investments in genetic medicines and advancements in the realm of protein degraders and ADC cancer therapies. Roche also outlined its strategy of not losing out on any opportunities available and mentioned its plans of tapping into the rapidly growing Chinese biotechnology market. Boris Zaïtra, appointed as the head of corporate business development at Roche, expressed confidence in being able to efficiently navigate the biotech space, despite its complexity and high costs.
JPM25: The FDA’s future, AbbVie’s second thoughts and Lilly’s lesson
The J.P. Morgan Healthcare Conference brings to the fore key ongoing issues in the biotech industry involving company strategies and regulatory prospects. Notably, the CEO of AbbVie indicated the company may tread lightly in the realm of psychiatry following a costly event. Alternatively, Lilly offered some insights regarding its miscalculation of GLP-1 drug demand. With the conclusion of the conference, industry stakeholders now shift focus toward the impending inauguration of President-elect Donald Trump, anticipating potential implications for the Food and Drug Administration (FDA) and the Department of Health and Human Services. Despite potentially disruptive rumors, pharma executives and investors express a general confidence in the continuity of the regulatory process, regardless of whether Trump's nominee, Marty Makary, gets the Senate's approval to run the FDA.
However, the FDA will soon lack a number of veteran leaders, following the stepping down or planned departure of figures such as Namandjé Bumpus and Patrizia Cavazzoni post-Trump's election. Expectations are that more details on Trump's healthcare agenda will be presented in the coming weeks as Kennedy and Makary face Senate vetting. The challenges observed at the J.P. Morgan Healthcare Conference may extend beyond the meeting, with industry players such as HSBC Innovation Banking citing headwinds stemming from interest rates and possible regulatory instability due to the Trump administration. This raises concerns over volatility, as uncertainty within the biotech industry could persist.
JPM25: Amgen’s defense, Merck’s patent ‘hill’ and Viking’s long-term planning
At the J.P. Morgan Healthcare Conference, Amgen sought to defend their weight loss drug, MariTide, after Phase 2 trial results failed to convince investors of its superiority over existing drugs. This led to questions regarding MariTide's potential and the company's optimistic outlook prior to releasing the data. However, Amgen executives argue that MariTide acts faster and requires less frequent dosing than other medications. They also relayed that its profile and their presentation of data were without exaggeration. Peter Griffith, Amgen's CFO, blamed a large share price drop following the trial results on trading behavior exaggerating the initial decline. He also mentioned data suggesting that MariTide allows for comparable weight loss at an earlier time point and may be administered once a month or less.
This lack of investor faith impacted Eli Lilly, whose shares fell 7%, affecting the stocks of other obesity drug developers, leading to a biotech stock index sliding by almost 3%. Eli Lilly has previously enjoyed success with its weight loss and diabetes medications, but for the second time, it has disappointed investors with its revenue numbers. Despite investors' doubts, Amgen announced that it will move forward with late-stage studies for MariTide, signaling its confidence in the drug's potential.
JPM25: Deals, Summit’s bravado and gene therapy headwinds
The annual J.P. Morgan Healthcare Conference has been held in San Francisco, with key industry players converging at the Westin St. Francis to network and plan for the future of healthcare. This year's conference, however, had an undercurrent of tension due to the recent murder of United Healthcare CEO Brian Thompson. This led to heightened security, including an increased presence from J.P. Morgan and Westin St. Francis security personnel as well as San Francisco Police Department officers.
M&A activity, typically a barometer of investment in biotechnology, has recently been in decline, severely affecting stock prices and causing frustration among shareholders. The J.P. Morgan Conference, in recent years, has not seen much M&A activity but is still viewed as a major platform for potential deals. This downturn in M&A excitement alongside heavy security measures at the conference reflect signs of caution from top healthcare companies.
JPM25 Day One: Pfizer, BioNTech, Moderna and More Present Pipeline Updates
The 2025 J.P. Morgan Healthcare Conference saw biopharma executives announce key business deals and updates, predominantly in cancer, obesity and vaccine development. Among them, Pfizer unveiled its intention to invest completely into its obesity drug 'danu', formally known as danuglipron. CEO Albert Bourla said late-stage studies are planned for H2 of 2025. Pfizer's strategy is to enter the obesity market with either oral or injectable versions with a distinctive mechanism of action, steering clear from direct competition with injectable drugs currently dominating the market, namely Novo Nordisk's Wegovy and Eli Lilly's Zepbound.
Meanwhile, Regeneron's CEO Leonard Schleifer provided updates on itepekimab, an experimental treatment for chronic obstructive pulmonary disease (COPD). The drug is expected to deliver Phase III data this year, marking a significant expansion to the company's roster if it stands alongside Dupixent, the company's existing COPD drug. Also, Phase III results are anticipated this year for two cancer drugs - fianlimab - a checkpoint inhibitor for melanoma, and linvoseltamab - a bispecific antibody for multiple myeloma. Ordspono, an antibody employed for lymphoma treatment and already approved in the EU, is preparing for an FDA approval resubmission, following a rejection in 2024 due to patient enrollment issues. Sarepta Therapeutics reported a successful market take for its Duchenne muscular dystrophy gene therapy, Elevidys, with revenues nearing $400 million.
JPM25 Day Two: Roche, Amgen, Merck, Lilly and Biogen
The second day of the J.P. Morgan Healthcare Conference (JPM25) had big pharma companies including Merck, Lilly, and Amgen laying out their growth strategies for the year, following insights gleaned from overcoming challenges in 2024. Merck is aiming to diversify its pipeline as it gears up for the expiry of key patent protections of its blockbuster PD-1 inhibitor, Keytruda, in 2028. Despite recording more than $21.6 billion in the first nine months of 2024, the firm anticipates a drop in Keytruda sales once biosimilar competitors enter the market. Efforts are underway to strengthen its portfolio, implying the expansion of the oncology business while also working on a subcutaneous formulation of Keytruda.
Meanwhile, Roche announced a careful utilization of its annual $10 billion allocation for potential mergers and acquisitions (M&A). CEO Teresa Graham highlighted that the company is not in a rush to spend this fund unless worthwhile deals are detected. This prudent fiscal control was demonstrated in a few recent deals, including a $900 million partnership with COUR Pharmaceuticals and a likely $1.8 billion contract with Flare Therapeutics in November 2024. Roche's major deal was the acquisition of Poseida Therapeutics for $1.5 billion.
JPM25 Day 3: AbbVie, Gilead, GSK and Dyne
On the third day of the 2025 J.P. Morgan Healthcare Conference, pharmaceutical giants AbbVie, Gilead, and GSK shared their business strategies. AbbVie reaffirmed its goal to achieve mid-single-digit topline and earnings-per-share growth in 2025 despite experiencing mid-stage failure with their antipsychotic drug candidate emraclidine. They plan to attain the growth target by focusing on their primary domain of expertise, inflammation and immunology, expecting high single-digit sales growth until 2030.
Gilead defended the accessibility program proposed for its HIV pre-exposure prophylactic, lenacapavir amid criticism. In October 2024, the company announced it had made a deal with generic developers to provide a low-cost version of lenacapavir in more than 100 resource-limited countries. However, the details thereof sparked controversy. AbbVie's CEO, Robert Michael, although confirming a dip in capital commitment to psychiatric therapies also indicated there is still hope for the neuropsychiatry space as Emraclidine testing with higher doses continues.
At JPM, Biogen CEO tries to take down the deal temperature
Biogen CEO, Chris Viehbacher, made a presentation for Wall Street analysts on January 14, 2025, to help them better understand the company's current interests and investments, particularly in the realm of business development. Although the company has observed a decline in their top drugs, and disappointing sales from their newest Alzheimer's treatment, the management team played down opportunities for future business deals. Instead, Viehbacher highlighted their recent successes, including the advancement of an antibody therapy and a couple of lupus drugs, acquired in a $1.2 billion acquisition.
Continuing with his presentation at the J.P. Morgan Healthcare Conference, Viehbacher explained that Biogen is not actively seeking to make new deals, but are rather focusing on those that make strategic or financial sense. One such acquisition is that of rare disease specialist Reata Pharmaceuticals, which handed Biogen a marketed therapy called Skyclarys. However, similar late-stage assets like Skyclarys are hard to find and sometimes too expensive. The CEO maintained he prefers smaller deals, particularly for drugs in the early- to mid-point of human testing. Lastly, he revealed that Biogen had sent an unsolicited offer letter to Sage Therapeutics, a co-developing partner, proposing a purchasing deal.
The People Behind Biopharma’s Big Wins: Personal Stories From JPM25
The article delves into the personal stories of several professionals in the biotech and pharmaceutical industry, focusing on the human side of drug development as the author shares experiences from recent interviews conducted during JPM25, a healthcare conference. Stories illustrate the passion and drive that these people bring to their work. One instance is highlighted with Priyah Singhal, the Head of Development at Biogen. Though she could not discuss the company's unsolicited bid to buy Sage Therapeutics, her zeal for her work, particularly in tackling diseases such as amyotrophic lateral sclerosis and Alzheimer's, is clear.
Annemarie Hanekamp, the Chief Commercial Officer at BioNTech, is another professional who is mentioned. Hanekamp, who assumed her position over the summer, is tasked with developing a commercial team to aid BioNTech in achieving its ambition of becoming a fully integrated drug company. The author emhasizes that personal interactions such as these offer invaluable insights into the people who drive the narratives of their industry, transcending the boundaries of pre-determined interview questions to explore the human aspects of drug development.
Biopharma Deals Start Flowing at JPM25—With One Dwarfing Last Year’s Activity
The J.P. Morgan Healthcare Conference recently initiated several biopharma deals, with significant transactions being announced by Eli Lilly, GSK, Gilead, and J&J. The latter topped the list with a $14.6 billion bid to acquire Intra-Cellular, a company that develops pills for schizophrenia and bipolar depression. This deal is considerably larger than any biotech acquisition in 2024 and has the potential to rejuvenate the biopharma sector's merger and acquisition activities and initial public offerings back to pre-pandemic levels.
In the context of increased external innovation requirements by large pharmaceutical firms and legislative influences such as the Inflation Reduction Act's drug pricing negotiation provisions, these deals serve as critical collaborations. Novartis's Chief Medical Officer, Shreeram Aradhye, suggested that his firm's business development team schedules are fully booked for substantial meetings. Furthermore, CEOs from Vir Biotechnologies and Montai Therapeutics cautioned that many large pharmaceutical companies are dealing with upcoming patent expirations, compelling them to engage in deals.
5 questions facing emerging biotech in 2025
Emerging biotech companies have been divided into two groups as 2025 begins: the 'haves' and the 'have-nots.' There was an increase in venture funding last year, however, a significant portion of this was concentrated in nine-figure 'megarounds.' Initial public offerings were mainly available to companies fitting into a certain niche, and a multitude of already public U.S. biotechs saw their stocks slide as pharma companies found other opportunities for deals. Piper Sandler analysts have described the current situation as a stock-pickers' market, characterized by a few successful companies and many more struggling. Immuno-oncology and cardiometabolic research companies have found it easier to secure investments.
Of several challenges on the horizon, a key question for biotech startups in 2025 is how licensing deals in China will affect U.S. biotech. For years, pharma companies and U.S. biotechs have turned to China to manufacture drugs or run clinical trials, and there is now an increasing trend to look to China to acquire new drug prospects to feed into their pipelines. After a dip in deals following a few regulatory setbacks with China-discovered drugs in 2022, record number and value of deals involving Chinese drugs were reached in 2024. Additionally, pharma companies are now drawing one-third of their in-licensed molecules from China, a significant increase from the 10% - 12% range during 2020-2022.
5 questions facing pharma in 2025
The biotechnology and pharmaceutical industries face significant uncertainty, with President-elect Donald Trump indicating potential disruption through unorthodox nominations for critical healthcare roles. These political risks coincide with unstable times for the industry, which is dealing with wider economic issues. According to Baird analysts, key aspects affecting biopharma performance, namely monetary policy, tariffs, and healthcare reforms, remain uncertain. Despite the broader political and economic uncertainties expected in 2025, there are several therapeutic trends poised to drive investments within the sector. These trends are primarily in metabolic and immune disease drug research.
Key questions facing the pharma industry in 2025 center around potential regulatory impacts of the Trump administration. This includes the implications of possible negotiations on drug prices and Medicare, especially since the U.S government plans to announce its targeted set of drugs for negotiations by February 1. One of the significant uncertainties is whether Trump would attempt to abolish Medicare drug price negotiations. The incoming administration could use the opportunity to lower some drug prices as a way to claim political victories. Furthermore, doubts remain about the potential role of Martin Makary in the Food and Drug Administration under the new administration.
Lilly’s Omvoh approved by FDA for Crohn’s
The U.S. Food and Drug Administration (FDA) has given approval to Eli Lilly's drug, Omvoh, for treatment of adult patients with Crohn's disease. This expands the potential market for Omvoh, a drug initially approved in 2023, which was already being used for adults suffering from ulcerative colitis, another form of inflammatory bowel disease (IBD). The latest approval is based on a study demonstrating that Omvoh could benefit patients whose disease was not sufficiently controlled by prior treatments. Omvoh has also been listed as a first-line therapy by two of the three largest pharmacy benefits managers, implying it can be used by patients without a requirement of having failed on another drug first.
Omvoh is among a batch of new drugs Lilly has developed to capitalize on its success in diabetes and obesity treatments, sectors that have bolstered the company's earnings and elevated its share prices. Drug manufacturers have recently invested heavily in gastrointestinal fields and extended the application of widely used drugs like AbbVie’s Skyrizi to include ulcerative colitis and Crohn's disease. Omvoh and Skyrizi function similarly by specifically targeting a subunit of the IL-23 protein to mitigate inflammation. Even though Omvoh's sales have been fairly modest thus far (with revenues of $41 million in the third quarter), Lilly highly regards its potential for future growth. Lilly has also announced that Omvoh is the first biologic in 15 years to enter the market by providing two years of Phase 3 efficacy data in Crohn's disease.
Federal watchdog cites concerns with FDA’s accelerated approval process
The Food and Drug Administration (FDA) needs to implement stricter standards for their accelerated approval process to prevent wastage on ineffective treatments, a federal watchdog report has revealed. The review, initiated after controversy surrounding the 2021 approval of Biogen’s Alzheimer’s drug Aduhelm, scrutinized 24 FDA reviews that led to accelerated approvals of new medicines. The report identified concerns with three cases, including the Aduhelm approval, citing that in these instances, the FDA failed to adhere to its best practices in data analysis, had poorly documented sponsor meetings and lacked oversight exacerbated by agency conflicts.
The inspector general’s office suggested two main changes to improve the accelerated approvals process. Firstly, meetings with sponsors need more thorough documentation, an advice FDA agreed with and has begun to improve its procedures for. Secondly, the office recommended outlining specific factors that would activate greater oversight, which FDA disagreed with. The inspector general's office suggested the FDA's accelerated approvals council should hold mandatory meetings when FDA reviewers don't reach consensus on an application, or when a drug maker relies on analyses not included in the original plan to win approval for a medicine, among other factors. This accelerated approval pathway allows drugs promising signs in research, such as disease-linked biomarker reduction, to reach the market quicker.
FTC again goes after PBMs’ business practices in new report
The Federal Trade Commission (FTC) has published a second report on the business practices of pharmacy benefit managers (PBMs), accusing CVS’ Caremark, Cigna’s Express Scripts and UnitedHealth’s Optum Rx of increasing the price of life-saving drugs exponentially. The report argues that these PBMs, often referred to as the "big three", have prioritized the most profitable prescriptions for their own affiliated pharmacies over independent operators, and have paid themselves more than other pharmacies for dispensing drugs. This second report expands on the FTC’s initial report, released last summer, focusing on a wider variety of drugs due to criticism that the first report relied on limited data.
The FTC initiated an investigation into the PBMs three years ago, scrutinizing their role in negotiating discounted drug prices with pharmaceutical companies and reimbursing pharmacies for dispensing these drugs. PBMs assert that they save money for both health plans and patients by encouraging drugmakers to lower their costs. Nevertheless, the FTC's initial report posited that the industry consolidation has led to a few major players obtaining undue influence, benefiting at the expense of patients and independent pharmacies. Subsequently, the FTC sued Caremark, Express Scripts, and Optum Rx, which jointly account for 80% of U.S. prescriptions, alleging that they artificially increased the cost of insulin. The PBMs and two Republican FTC commissioners, along with some pharmacy market experts, contested the report, asserting it was biased and reliant on selective data. In response, the 'big three' countersued the FTC.
J&J to buy psychiatric drug developer Intra-Cellular for $14.6B
Johnson & Johnson (J&J) has announced it will acquire Intra-Cellular Therapies, a pharmaceutical company that develops drugs for brain diseases, for approximately $14.6 billion. If successful, this will be the largest biotech acquisition since early 2023. J&J is primarily interested in Intra-Cellular’s drug, Caplyta, which is approved in the US for treating schizophrenia and bipolar depression. The company has also applied to the FDA for permission to use Caplyta to treat major depressive disorder, a condition that affects ten times the number of people as schizophrenia.
The acquisition aligns with J&J's established focus on neuroscience, demonstrating its commitment to "transforming care and advancing research in some of today’s most devastating neuropsychiatric and neurodegenerative disorders," according to J&J CEO Joaquin Duato. Over the past ten years, J&J has maintained its investment in neuroscience, even as many of its peers have exited the field. The company's current portfolio includes several formulations of Invega, a medication for schizophrenia, and Spravato, a derivative of ketamine approved for treatment-resistant depression. The deal is considered strategic, given J&J's interest in and prior investment in brain drugs.
Lilly pads cancer drug pipeline with Scorpion deal
Eli Lilly, a multinational pharmaceutical company, has undertaken a $2.5 billion acquisition of an experimental cancer drug from the privately-held biotechnology startup, Scorpion Therapeutics. As a result of this deal, Scorpion will divest its additional assets and staff into a newly-formed, independent company, while Lilly will hold a minority share. The new entity will be owned by Scorpion's existing shareholders, such as Atlas Venture, Vida Ventures, and Omega Funds, with Scorpion's current CEO, Adam Friedman, leading the new company along with the startup's management team.
The acquisition gives Lilly access to Scorpion's STX-678 medication, aimed at managing tumors driven by PI3Ka gene mutations, which are common in more than 160,000 annual cases of breast, gynecological, and head and neck cancers in the United States alone. Current medications targeting PI3Ka mutations, such as Novartis' Piqray and AstraZeneca’s Truqap, can be tough to tolerate and don't readily reach brain tissue, which is often affected by these tumors. Scorpion, however, asserts that STX-678 could be more selective and potent than existing PI3Ka inhibitors, potentially overcoming these limitations. The drug is currently in Phase 1/2 trials for various tumor types, including a common type of breast cancer. The deal bolsters Lilly's cancer drug portfolio, enhancing its metabolism disease business which it significantly invested in with the acquisition of Loxo Oncology in 2019.
Moderna shares tumble on slashed sales guidance
Shares in Moderna fell more than 20% after the company reduced its revenue forecast for 2025 by $1 billion, predicting revenues of between $1.5 billion and $2.5 billion. This is a noticeable drop from their previous estimate range of $2.5 billion to $3.5 billion. The revision follows a trend of lower than expected vaccine sales, resulting in Moderna hastening its previously announced cost-cutting initiatives, aiming to cut costs by $1 billion this year. CEO Stéphane Bancel recognized their overly optimistic projections for their respiratory syncytial virus (RSV) vaccine.
The previous year was challenging for vaccine manufacturers as the demand for both COVID-19 and RSV vaccines saw a significant decline. Analyst Michael Yee revealed that COVID vaccines involving Moderna, Pfizer, and Novavax dropped by 8% by the end of 2024 compared to the prior year. Moderna’s RSV vaccine, mResvia, experienced a tough market debut. Stiff competition from Pfizer and GSK, alongside updated CDC guidelines that tightened recommendation for RSV immunization, negatively affected the expected uptake. Moderna’s production pipeline remains promising, but investors remain cautious about the company’s future success.
Tune raises $175M for hepatitis B medicine, epigenetic research
Genetic medicine startup, Tune Therapeutics, has secured an additional $175 million funding. The focus for Tune is to develop therapies capable of adjusting genes to treat disease, as opposed to editing them through DNA cutting or replacement. This latest financial injection adds to high recent private financing figures in the biotech industry. Notably, Tune's announcement hit the headlines just before the start of the J.P. Morgan Healthcare Conference, which acts as a significant market opener for both the biotechnology and pharmaceutical sectors.
Tune's lead therapy, Tune-401, is created to treat chronic hepatitis B. The therapy works by silencing the viral DNA within infected cells. Globally, over 250 million individuals are estimated to live with chronic hepatitis B infections, which can lead to liver failure and cancer. Tune's co-founder and CFO, Akira Matsuno, likened their therapeutic strategy to shutting off the water supply to address a leak. The company has initiated a Phase 1 trial of Tune-401 in New Zealand and Hong Kong. The potential applications of this epigenetic editing approach, which is viewed as more efficient and less invasive than DNA-cutting technologies like CRISPR, are broad and could be used to turn genes on or off.
Intellia to stop work on rare disease therapy, lay off staff
Intellia Therapeutics plans to halt its work on a principal drug research program, NTLA-3001, targeting alpha-1 antitrypsin deficiency, a rare lung disease. The company will also lay off more than a quarter of its workforce as part of a restructuring process, which aims to prioritize resources around its two advanced experimental medicines. There's also an expected leadership change with the retirement of Laura Sepp-Lorenzino, Intellia's chief scientific officer since May 2019. Birgit Schultes, who has led Intellia's immunology and cell therapy work since 2017, will replace Sepp-Lorenzino as CSO.
This is the second consecutive year that Intellia starts with layoffs and research cuts. In January 2024, the CRISPR gene editing company announced that it would be eliminating 15% of its workforce and suspending some exploratory research. This year, the company's priority has shifted somewhat. Intellia will focus on clinical execution for its two programs targeting hereditary angioedema and transthyretin amyloidosis, and will work on advancing commercial readiness in preparation for the launch of these medicines. The programs, NTLA-2002 and nex-z, are currently in Phase 3, and Intellia plans to expedite enrollment into these trials.
Patrizia Cavazzoni, key FDA official, to leave agency
Patrizia Cavazzoni, the influential director of the FDA's main drug review office, will step down next week, according to an internal advisory. Her decision to leave reportedly follows decisions by several other key FDA leaders – including Namandjé Bumpus, the principal deputy commissioner, and longtime agency veterans Douglas Throckmorton and Robert Temple – to exit the regulator ahead of the anticipated shift in Presidential administration. Cavazzoni, who joined the FDA's Center for Drug Evaluation and Research seven years ago and became its head in 2021, stated in an email to staff that the time had come for her to be more present for her family.
While it is common for staff changes to occur between administrations, the future of the FDA appears to be facing significant disruption given the anticipated changes signalled by President-elect Donald Trump. Trump has nominated Robert F. Kennedy Jr. to lead the Department of Health and Human Services, which supervises the FDA, and has pledged to allow Kennedy to bring radical changes to the healthcare sector. Notably, Kennedy has publicly criticised the FDA as corrupt and accused it of waging a war on public health.
Pfizer’s PD-1 drug succeeds; AbbVie writes down $3.5B in Cerevel assets
Pfizer's PD-1 inhibitor, sasanlimab, demonstrated positive outcomes during a clinical trial in bladder cancer patients, where its efficacy in delaying disease complications and death outpaced standard therapy. As a subcutaneous immunotherapy, sasanlimab stands to compete with similar drugs like Merck's Keytruda and Bristol Myers Squibb's Opdivo. Currently, such inhibitors have approval for treating more advanced stages of disease. Should sasanlimab secure FDA approval, it would be the fourth PD-1 or PD-L1 inhibitor available as an under-the-skin shot.
On the flip side, AbbVie is grappling with financial repercussions from its acquisition of Cerevel Therapeutics, taking an impairment charge of approximately $3.5 billion. The acquisition was primarily for Cerevel's pipeline of experimental brain drugs, one of which was a potential new treatment for schizophrenia. However, this drug suffered a significant setback during clinical trials effectively curbing its development trajectory. This failure resulted in a substantial loss in AbbVie's market value in excess of $40 billion.
Lilly’s I&I Push Gains Momentum With Omvoh Expansion
The FDA has granted approval for Eli Lilly's antibody Omvoh (mirikizumab) in the treatment of Crohn's disease, representing a significant step in Lilly's plans for expansion and revenue diversification into inflammation therapy. BMO Capital Markets analysts, in an investor note, refer to this expansion as a crucial part of the company's strategy to diversify its portfolio beyond obesity. However, they view Omvoh's prospects in treating Crohn's disease as modest, especially since it was unable to demonstrate superiority over Stelara. The VIVID-1 Phase III trial results, posted in October 2024, showed that more patients treated with Omvoh achieved a histologic response after 52 weeks compared to Stelara. Despite this, Lilly acknowledged that the effect was only marginally statistically significant.
The data which supported the FDA approval revealed a 53% clinical remission rate and a 46% endoscopic response rate at 1 year, with both metrics showing significant superiority to a placebo. However, Stelara remains a challenging 'hurdle' to clear in the inflammatory bowel disease (IBD) space, and analysts predict that Omvoh's uptake for Crohn's disease will be challenging. They project peak worldwide revenue of $801 million across IBD indications by 2032. In conclusion, the analysts believe that despite the modest expectations, Lilly's label expansion indicates their headway in the inflammation and immunology area, as they continue to diversify their portfolio. A key component of this has been the $3.2 billion acquisition of Morphic Holding in July 2024, giving Lilly access to the drug candidate MORF-057.
BioNTech Strives to Become Commercial ‘Powerhouse’ While Maintaining Humility
BioNTech, previously known for its COVID-19 vaccine, is seeking to transition back to its roots as an oncology company. Annemarie Hanekamp, the new Chief Commercial Officer, will be overseeing this transformation. She brings a strong resume marked by leading significant commercial shifts within the pharmaceutical sector, a skill set BioNTech sees as essential for their transition. The company is setting ambitious targets for the future, preparing to launch its first oncology product in 2026 and hoping to commercialize therapeutics in 10 indications by 2030.
Hanekamp indicates that BioNTech is aware of the challenges associated with commercializing assets but is prepared to take risks. Instead of developing drugs sequentially, BioNTech and other companies are moving to develop their pipelines in parallel, which could help identify successful products sooner, despite the increased risk. Hanekamp added that the company's plan isn't just about shareholder returns, but also about procuring new treatment options for patients as swiftly as possible.
Passage Bio Cutting 55% of Staff to Help Extend Cash Runway
Passage Bio, a Philadelphia-based clinical-stage genetic medicines company, plans to reduce its workforce by about 55% to extend its cash runway into the first quarter of 2027. The reduction is expected to affect approximately 32 of the company's 58 employees as of December 31, 2023, and will result in severance and exit costs of about $2 million, primarily incurred during the second quarter. The company, which has locations in Philadelphia and Hopewell Township, New Jersey, did not specify if the layoffs would impact both sites. As part of its strategic changes, Passage Bio is also transitioning to an outsourced analytical testing model to support the advancement of its PBFT02 program.
Along with the staffing cutbacks, Passage Bio reported updated data from its ongoing Phase I/II upliFT-D clinical trial evaluating PBFT02 in frontotemporal dementia (FTD) with granulin mutations. According to company President and CEO, Will Chou, the first dose of PBFT02 consistently increased cerebrospinal fluid progranulin expression, showing early signs of improvement in a disease progression biomarker. For the purpose of discussions with health authorities regarding a registrational pathway, the company plans to introduce a second dose and share additional data in the latter half of 2025. Furthermore, Passage Bio completed the process development and scale-up of a high-productivity, suspension-based manufacturing process for PBFT02, enhancing yield rates and presenting the possibility of lower production costs.
IGM Biosciences Lays Off 73% of Staff, Shrinks Pipeline
IGM Biosciences is letting go of 73% of its workforce and discontinuing the development of two autoimmune drug candidates. The company, which had 198 employees as of September 30, could only have less than 55 after the downsizing. IGM's decision to cease work on its two bispecific antibody T cell engagers, imvotamab and IGM-2644, followed disappointing interim data from Phase Ib studies. The depth and consistency of B cell depletion in tests of imvotamab in rheumatoid arthritis and systemic lupus erythematosus didn’t meet IGM’s criteria for success. The IGM-2644 will also be discontinued due to strategic considerations. The company's shares were subsequently downgraded by BMO Capital Markets after the announcement.
The cutbacks have taken the market by surprise and highlight uncertainties in T cell-mediated approaches for cancer and autoimmune diseases. As a result of the staff reduction and drug candidate discard, IGM's collaboration with Sanofi is now the only source of revenue for the smaller firm. Together they aim to produce, develop, and commercialise IgM antibody agonists against immunology and inflammation targets. The company is reportedly evaluating "internal options as well as potential strategic alternatives" to increase shareholder value. The downsize and pipeline adjustment marks the second revision within six months, as the company relocated resources away from oncology to concentrate on T cell engagers in autoimmune disease last September.
Apellis, Barinthus, Repare Among Latest to Trim Workforces
The biopharma industry faced a difficult year in 2024, as many companies reduced their workforce significantly, and this trend appears to continue into 2025 as Apellis Pharmaceuticals, Barinthus Biotherapeutics, and Repare trim their workforces. Apellis Pharmaceuticals stated it would lay off 40 employees as part of their strategy to centralize its commercial operations in the U.S, resulting in layoffs primarily impacting overseas staff. Additional changes in the organization include the step down of the Chief Operating Officer, Adam Townsend, effective from February 21. These disclosures were announced during a preview of their fourth-quarter results at the 2025 JP Morgan Healthcare Conference, where the company reported $709 million in preliminary full-year U.S. net product revenues.
Moving forward, Apellis plans to file for the label expansion of Empvali into two rare kidney diseases, complement 3 glomerulopathy and primary immune complex-mediated membranoproliferative glomerulonephritis, in early 2025. On the other hand, Barinthus Biotherapeutics is planning to cut 65% of its workforce across its U.K. and Germantown, Maryland locations. This drastic staff reduction comes as the company seeks to decrease costs while focusing more on immunology and inflammation indications. Further details about these layoffs and other companies likely to implement similar measures will be tracked by BioSpace throughout 2025.
The Top 12 Companies Hiring in Biopharma Now
The biopharma industry continues to experience layoffs, with an estimated 24,000 jobs eliminated over the last year. Unfortunately, December 2024 job postings reflected this trend, showing an 8% decrease compared to November, matching the previous month-over-month decline. There was also a 16% reduction in year-over-year data, displaying fewer job postings on the BioSpace website in December 2024 than in December 2023. This year-over-year dip, however, was slightly less than November's 20%.
Despite these challenges, some pharma and biotech companies are currently hiring. Based on recent job posting volumes, the top 12 biopharma companies hiring now are: AbbVie, Eli Lilly, Takeda, Amgen, Novo Nordisk, Regeneron, Gilead Sciences, Daiichi Sankyo, Moderna, CSL, BioMarin Pharmaceutical, and GenScript. In addition to these companies, Eagle Analytical is also actively hiring. While there are fewer job postings, opportunities still exist within the biopharma industry.
Regenxbio Jumps on Potential $800M Rare Disease Deal With Nippon Shinyaku
Regenxbio has entered into a strategic partnership with Kyoto-based Nippon Shinyaku to develop novel gene therapies for the rare metabolic disorder mucopolysaccharidosis. Regenxbio will receive an upfront payment of $110 million once the transaction closes and is eligible for up to $700 million in potential development, regulatory and sales milestones. In return for this investment, Nippon Shinyaku will gain access to two of Regenxbio’s gene therapies - RGX-121 and RGX-111. The stock of Regenxbio surged nearly 20% before markets opened following the announcement of the deal.
RGX-121, which is being tested for mucopolysaccharidosis type II, works by delivering a functioning copy of the iduronate-2-sulfatase gene, which is faulty in those with the disorder. A pivotal trial for RGX-121 showed significant reduction in the cerebrospinal fluid levels of a key disease biomarker and improved the acquisition of neurodevelopmental skills in treated children. The second asset, RGX-111, is being developed for mucopolysaccharidosis type I, a rare metabolic disease caused by a mutation. This collaboration marks an important milestone for Regenxbio, particularly as their shares hit an all-time low of $6.95 at close of business before this deal.
Pfizer Cautiously Optimistic on Obesity Pill as Kailera Challenges Lilly’s Zepbound With Strong Phase II Data
During the 2025 J.P. Morgan Healthcare Conference, Pfizer made a strong commitment towards tackling obesity, with CEO Albert Bourla expressing cautious optimism for their oral candidate danuglipron. The pharmaceutical giant plans to go "all in" on obesity, leveraging its increasing expertise to make sound decisions, particularly regarding danuglipron. This drug, which is seen as a once-daily option for chronic weight management, is a GLP-1 receptor agonist. It works similarly to already-approved treatments semaglutide from Novo Nordisk and tirzepatide from Eli Lilly, by promoting the secretion of insulin from the pancreas, slowing gastric emptying, and suppressing appetite. Danuglipron aims to be more convenient than competitors, potentially helping Pfizer secure a unique market position.
However, some analysts remain uncertain about Pfizer's strategy. In a July 2024 interview, Guggenheim Securities analyst Vamil Divan expressed skepticism that their approach will prove successful, questioning if they might be acting out of desperation to make their mark in the weight-loss field. Likewise, BMO Capital Markets analysts noted Pfizer's hesitation to expand on danuglipron expectations. Despite earlier setbacks with the development of danuglipron, including the discontinuation of a twice-daily formulation in December 2023 due to high rates of toxicity during phase IIb trials, Pfizer remains committed to proceeding with a late-stage study for the drug later this year.
Metsera Moves Fast in Crowded Obesity Market With IPO Plans
Metsera, a biotech firm dedicated to the obesity market, has announced its intentions to go public as part of its strategy to establish itself as a key player in the lucrative weight-loss industry. As yet, the company has not disclosed the amount it expects to raise through its Initial Public Offering (IPO) and has not provided a target closing date. Once the IPO is finalized, Metsera will appear on the Nasdaq Global Market under the ticker $MTSR. The firm plans to funnel most of the funds acquired from the IPO towards its planned Phase III trial for its main obesity candidate MET-097i, an injectable, ultra-long-acting GLP-1 receptor agonist that is already in Phase II trials. Residual funds from the IPO will supply the working capital and other general operating expenses.
Previously, Metsera revealed early Phase IIa data for MET-097i, showing a 11.3% weight loss after 12 weeks, with some patients reporting as high as 20% weight loss. Metsera referred to this reduction as "clinically meaningful and statistically significant". Furthermore, pharmacological exposure of MET-097i showed an increase in the drug’s concentration over the 12 week period, suggesting it could be administered monthly. The company is currently enrolled in a parallel Phase IIb study of MET-097i, which is fully staffed and likely to release preliminary data in mid-2025. Additionally, Metsera plans to initiate other investigations into MET-097i across obesity and type 2 diabetes throughout the year.
Gilead Continues Diversification Drive With Potential $1.7B Inflammation Pact With LEO
Gilead Sciences has formed a strategic partnership with Danish biotech company LEO Pharma. This partnership aims to progress programs for undisclosed inflammatory targets. Transaction details remain limited, but include an upfront payment of $250 million and up to $1.7 billion in potential total deal consideration along with royalties. With Gilead's investment, the pharmaceutical behemoth will secure global rights to develop, manufacture and market LEO's preclinical oral small molecule STAT6 program targeting certain inflammatory conditions. Gilead will also claim ownership of LEO's preclinical targeted protein degraders.
LEO will maintain global rights to cultivate its STAT6 programs as topical treatments in dermatology while retaining the option to co-market the oral therapies for dermatology outside the U.S. As part of the deal, Gilead may be entitled to tiered royalties from the sales of topical STAT6 products. STAT6 (signal transducer and activator of transcription 6), a transcription factor, is linked with IL-4 and IL-13 signaling cascades and is crucial to immune-mediated diseases like asthma, atopic dermatitis, and chronic obstructive pulmonary disease. Preliminary studies have shown that targeting STAT6 can combat inflammatory targets effectively, and could provide an oral substitute to typically injected therapies.
GSK Targets Rare Cancer With $1B+ Acquisition of IDRx
GSK plans a significant acquisition of precision therapy specialist IDRx and its rare cancer drug candidate, CCTV reports. This deal, involving an upfront payment of $1 billion, plus up to $150 million in success-based regulatory approval milestones, will give GSK a promising small molecule drug for gastrointestinal stromal tumors (GIST) pending a completion of an antitrust review. This move will enable GSK to take over the development of IDRX-42, a first- and second-line treatment for GIST, a rare cancer that afflicts between 4,000 to 6,000 individuals in the US, typically linked to alterations within the KIT gene. IDRX-42 can inhibit both GIST progression and the development of resistance mutations.
IDRX-42, that has received the FDA’s Orphan Drug designation, is currently in the initial stages of clinical testing in advanced GIST. Recent data has shown IDRX-42 has substantial anti-tumor activity, reflecting a 29% objective response rate across all treated patients. The response rate is 53% when focussed on a second-line treatment setting. GSK believes broad activity against known KIT mutations gives IDRX-42 high potential for a best-in-class profile. The acquisition moves GSK into a leadership position within the rare cancer drug market, though acquisition is subject to clearance from antitrust regulators and other specific closing conditions.
Seaport, With 20/20 Hindsight, Takes Lessons From Neuropsych’s Stumbles
Seaport Therapeutics is drawing lessons from existing challenges and failures in neuropsychiatric drug development to improve their strategies. The company's leadership team, seasoned in the intricacies of neuropsychiatry drugs, is capitalizing on their experience in this field to enhance clinical trial design and utilize proven mechanisms. The team played a significant role in the development and approval of Karuna Therapeutics' schizophrenia drug, KarXT, underscoring their expertise in the sector. However, they also acknowledge the failures in neuropsychiatry, such as the unsuccessful drug developments by Cerevel and Sage Therapeutics.
Daphne Zohar, Seaport Therapeutics' CEO, has personal motives driving the company's mission, making the venture deeply personal. Zohar, who has family members suffering from depression and anxiety, is keen on making significant strides in this sphere. Highlighting its innovative approach, Seaport is utilising the Glyph platform, which employs the lymphatic system to bypass the liver, reducing potential liver toxicity and enhancing oral bioavailability. Leveraging their strong track record, Seaport has managed to secure early investors from its predecessor, Karuna.
5 Cardio Gene Therapies on the Near Horizon
Benefiting from positive early data and foundational technological and conceptual groundwork, gene therapies are progressing in clinics for cardiovascular diseases such as cardiomyopathy, congestive heart failure, and chronic refractory angina. According to Faraz Ali, CEO of Tenaya Therapeutics, precision and gene therapies are on the horizon for heart diseases. Cardiovascular diseases are the leading cause of death globally, claiming around 18 million lives each year per WHO data. These diseases refer to a diverse range of conditions affecting the heart and blood vessels, from highly prevalent disorders like heart failure and coronary artery disease to rarer conditions like cardiomyopathy associated with Duchenne muscular dystrophy or transthyretin amyloidosis.
Initially, lifestyle modifications such as diet and exercise are the first intervention options for cardiovascular diseases. Depending on the symptoms, medicines like aspirin and beta-blockers are prescribed. However, gene therapies are gaining momentum in this field. Ali cites early successes with Rocket Pharmaceuticals' work on Danon disease and Lexeo Therapeutics' progress on Friedreich's ataxia cardiomyopathy as examples of AAV gene therapy for genetic cardiomyopathy showing early clinical benefits. Gene therapy, which involves using a healthy gene to improve or restore the expression of a specific protein, has now been validated as an effective treatment approach. Plus, the techniques and tools that scientists use to identify and isolate genes of interest and package them into therapeutic particles have been significantly optimized.
CONCLUSION
There you have it, another week of SWEAT EQUITY - Your Weekly Biotech News Fix. We hope you enjoyed it, please drop a comment with any feedback you may have.
We are now publishing 7x per week according to the following cadence:
Mondays: BioBucks: Stock Analysis & Biotech Catalysts
Tuesdays: X-article on biotech hot topics
Wednesdays: Podcast format of X-article
Thursdays: Insiders Track: Public & Private Biotech Markets
Fridays: Sweat Equity: Your Weekly Biotech Fix
Saturdays: Podcast format of Weekly News Update
Sundays: Building Biotech: Strategic Topics
SUBSCRIBE TO PODCAST HERE:
Sundays and Mondays are under the paid umbrella, everything else is FREE. It is $5/month and we encourage you to support our efforts. It is less than 1 cup coffee these days and keeps us motivated to keep producing the hybrid science and business biotech focused content you will not find anywhere else all in the same place.
As a reminder, if looking to go deeper into the topics we cover check out our website BowTiedBiotech.com, or DM us on twitter, or email us: bowtiedbiotech@gmail.com
ABOUT BOWTIEDBIOTECH
As a reminder, the purpose of the BowTiedBiotech substack is two-fold. Primarily, we aim to provide our scientist audience the tools to build a biotech company and ultimately translate their ideas into medicines for patients. Secondarily, biotech investors may find this substack useful as we will be providing weekly market updates of the public AND private markets as well as heavily leveraging current financing events as teaching examples.
DISCLAIMER
None of this is to be deemed legal or financial advice of any kind. All updates are sourced from publicly available disclosures. Insights are *opinions* written by an anonymous cartoon/scientist/investor.
TOP BOWTIEDBIOTECH NEWSLETTERS