The Biotech Beat: 3.11-3.17.24
Week of 3/11 - 3/17/24
by Joey Bose
??Upshot
?? CAR-T Breakthrough: Half of Glioblastoma Patients See Stable Disease or Better in Phase 1 Trial. In a groundbreaking Phase 1 clinical trial conducted by Mustang Bio and City of Hope, half of the participants with recurrent glioblastoma experienced stable disease or better following treatment with chimeric antigen receptor T-cell (CAR-T) therapy.
?? Revolutionizing Healthcare: Google Cloud's Generative AI Alleviates Administrative Burden. Google Cloud announced updates to its AI-powered search capabilities for healthcare and life sciences, aimed at reducing administrative work and supporting clinicians.
?? A Cure in Limbo: Hemophilia Patients Weigh Risks of Gene Therapy. Despite decades of research and the recent approval of two gene therapies for hemophilia, Hemgenix and Roctavian, patient uptake has been surprisingly low. Here's why.
?? Breaking the NASH Barrier: Madrigal's Rezdiffra Paves the Way for Liver Disease Treatment. Madrigal Pharmaceuticals' resmetirom, branded as Rezdiffra, has received accelerated approval as the first treatment for metabolic dysfunction-associated steatohepatitis (MASH).
?? Pharma Giants Bet Big on R&D: A New Era of Innovation and Deal-Making. The pharmaceutical industry has experienced a significant shift in research and development (R&D) spending, with companies like Merck, Roche, and Johnson & Johnson leading the charge.
?? LianBio's Journey: A Tale of Ambition and Market Realities in China's Pharma Landscape. LianBio aimed to bridge the gap between Western biopharmaceutical companies and the Chinese market by in-licensing experimental therapies for development and commercialization in China... but they failed.
?? FDA's Nod to Amyloid Reduction: A Shift in Alzheimer's Drug Development. The FDA has updated its guidance on Alzheimer's drug development, suggesting that amyloid reduction can serve as a surrogate endpoint for predicting clinical benefit in certain circumstances.
?? Gene Therapy's Tough Choice: Curing Sickle Cell Disease at the Cost of Fertility. Two new gene therapies for sickle cell disease offer a potential cure for the condition but come with a significant drawback: the risk of infertility.
?? Biden's 2025 Budget Blueprint: A Balancing Act Between Health Care Goals and Fiscal Constraints. Biden's 2025 budget proposal of $130.7 billion reflects a decrease from the previous year and aims to address key health care priorities while navigating fiscal constraints.
?? Victory for Hospitals: Appeals Court Upholds Arkansas Law on 340B Drug Discount Program. A U.S. appeals court has upheld an Arkansas law that supports hospitals participating in the 340B drug discount program, a big win for hospitals.
?? Research, Development & Drug Approvals ??
?? CAR-T Breakthrough: Half of Glioblastoma Patients See Stable Disease or Better in Phase 1 Trial
The Facts
In a groundbreaking Phase 1 clinical trial conducted by Mustang Bio and City of Hope, half of the participants with recurrent glioblastoma experienced stable disease or better following treatment with chimeric antigen receptor T-cell (CAR-T) therapy. Notably, one patient has remained cancer-free for over five years. The study, published in Nature Medicine, represents the largest CAR-T therapy trial for glioblastoma to date and highlights the potential benefits of directly injecting CAR-T cells into brain tumors and cerebrospinal fluid. The trial involved 58 patients, all of whom had undergone at least one recurrence of the disease prior to enrollment. The overall median survival duration for the trial participants was eight months, with a subgroup receiving CAR-T cells in both their tumors and cerebrospinal fluid achieving a median survival of 10.2 months.
Our Opinion
The results of this Phase 1 trial are a beacon of hope in the fight against glioblastoma, one of the most aggressive and lethal brain tumors. The ability of CAR-T therapy to achieve stable disease or better in half of the subjects is a significant milestone, especially considering the poor prognosis associated with recurrent glioblastoma. This study not only demonstrates the feasibility of directly injecting CAR-T cells into the brain but also opens new avenues for the development of more effective treatments. The encouraging survival rates and the absence of severe neurotoxicity underscore the potential of CAR-T therapy to revolutionize the treatment landscape for glioblastoma, offering a glimmer of hope to patients with limited options.
Your Turn
How do the results of this CAR-T therapy trial compare to the outcomes of other treatment modalities for recurrent glioblastoma, such as chemotherapy or radiation?
?? Revolutionizing Healthcare: Google Cloud's Generative AI Alleviates Administrative Burden
The Facts
At the HIMSS 2024 Global Conference, Google Cloud announced updates to its AI-powered search capabilities for healthcare and life sciences, aimed at reducing administrative work and supporting clinicians. The Vertex AI Search for Healthcare enables medically-tuned searches across various data types, integrating with MedLM, HDE, and FHIR APIs. This advancement is part of Google's broader strategy to address issues like workforce shortages and provider burnout in healthcare. The new capabilities, including a gen AI model for chest X-ray classification and a condition summary API, promise to improve operational efficiency and patient care quality.
Our Opinion
The integration of generative AI into healthcare represents a pivotal moment for the biotech industry. Google Cloud's advancements in AI-powered search and analytics tools have the potential to transform how healthcare providers access and interpret patient data, leading to more informed decision-making and better patient outcomes. This technology could be a game-changer in reducing the administrative burden on clinicians, allowing them to focus more on patient care. As the industry continues to evolve, companies that leverage AI to addre
Your Turn
What challenges do you foresee in the widespread adoption of AI technologies in healthcare settings, and how can they be addressed?
?? A Cure in Limbo: Hemophilia Patients Weigh Risks of Gene Therapy
The Facts
Despite decades of research and the recent approval of two gene therapies for hemophilia, Hemgenix and Roctavian, patient uptake has been surprisingly low. Hemgenix, approved for hemophilia B, and Roctavian, for hemophilia A, carry hefty price tags of $3.5 million and $2.9 million, respectively. However, insurance coverage has not been the primary barrier, as these one-time treatments are cost-competitive with lifelong conventional therapies. The hesitation stems from concerns about the longevity of the therapy's effectiveness, potential side effects, and the fact that current treatments allow many patients to live relatively normal lives. The slow adoption of these gene therapies raises questions about the future of the field, especially as the industry faces challenges in developing profitable business models for one-time treatments.
Our Opinion
The cautious approach of hemophilia patients toward gene therapy reflects a broader challenge in the biotech industry: balancing the promise of revolutionary treatments with patient trust and practical considerations. While gene therapy offers the tantalizing prospect of a cure, the uncertainties surrounding its long-term efficacy and potential side effects make patients hesitant to abandon tried-and-true treatments. This situation underscores the importance of continued research, patient education, and transparent communication to build confidence in new therapies. It also highlights the need for the industry to develop sustainable business models that can support the development of groundbreaking treatments while ensuring patient access and affordability.
Your Turn
Considering the unique challenges of gene therapy, how can regulatory agencies and healthcare providers work together to ensure patient safety while facilitating access to potentially life-changing treatments?
?? Breaking the NASH Barrier: Madrigal's Rezdiffra Paves the Way for Liver Disease Treatment
The Facts
Madrigal Pharmaceuticals' resmetirom, branded as Rezdiffra, has received accelerated approval as the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), formerly known as nonalcoholic steatohepatitis (NASH). Targeting patients with stage 2 and 3 fibrosis, Rezdiffra is set to hit the market in April at a cost of $47,400 per year. The drug's approval marks a significant milestone in addressing a disease that has long challenged scientists and investors, with previous efforts failing due to safety concerns or negative trial results. Rezdiffra's success in Phase III trials, showing resolution or improvement in MASH without drug-induced liver injury, positions it as a potential game-changer for patients and the medical community alike.
Our Opinion
The approval of Rezdiffra is a monumental achievement in the biotech industry, signaling a new era in the treatment of liver diseases. This breakthrough underscores the importance of persistence and innovation in drug development, especially for conditions with unmet medical needs. As the first approved therapy for MASH, Rezdiffra not only offers hope to hundreds of thousands of patients but also sets the stage for further advancements in the field. The drug's success could catalyze investment and research into additional therapies, potentially transforming the landscape of liver disease treatment and improving patient outcomes.
Your Turn
Considering the high cost of Rezdiffra, what strategies could be implemented to ensure equitable access and affordability for all eligible patients?
?? Investment, M&A, and IPOs ??
?? Pharma Giants Bet Big on R&D: A New Era of Innovation and Deal-Making
The Facts
The pharmaceutical industry has experienced a significant shift in research and development (R&D) spending, with companies like Merck, Roche, and Johnson & Johnson leading the charge. Merck's R&D expenditure skyrocketed by 125% to $30.5 billion in 2023, driven by a series of mergers and acquisitions (M&A) and drug licensing deals. Roche and J&J also saw substantial increases in their R&D budgets. The industry's focus has shifted towards late-stage assets, with a preference for de-risked drug programs and a move away from heavy preclinical risks. Immunology and oncology remain popular areas of investment. The trend is expected to continue into 2024, with the Big 15 pharma companies demonstrating a strong desire and financial capacity to invest in R&D.
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Our Opinion
The surge in R&D spending by pharmaceutical giants reflects a strategic pivot towards innovation and a quest for the next wave of blockbuster drugs. This investment is crucial for addressing unmet medical needs and driving future growth in the industry. The emphasis on late-stage assets and de-risked programs suggests a more cautious approach to R&D, balancing the pursuit of innovation with the need to manage financial risks. As the industry navigates patent cliffs and the need for new revenue streams, this renewed focus on R&D could herald a new era of breakthroughs and transformative therapies that will shape the future of healthcare.
Your Turn
What challenges and opportunities do pharmaceutical companies face as they increase their R&D spending in the current economic environment?
?? LianBio's Journey: A Tale of Ambition and Market Realities in China's Pharma Landscape
The Facts
LianBio, launched in 2020 by American investment firm Perceptive Advisors, aimed to bridge the gap between Western biopharmaceutical companies and the Chinese market by in-licensing experimental therapies for development and commercialization in China. The company raised over $700 million and was part of a broader trend of significant investment in China in-licensing deals between 2019 and 2021. However, the market collapsed due to pricing pressures, intense competition, and a global funding crunch. By 2023, LianBio had initiated a strategic review, sold back rights to its main drug candidates, and announced plans to wind down operations. This shift reflects broader challenges in the Chinese pharmaceutical market, where deep discounts, competitive dynamics, and regulatory reforms have reshaped the landscape for foreign and domestic companies alike.
Our Opinion
LianBio's rise and fall exemplify the volatile nature of the pharmaceutical industry in China. While the market remains the second-largest in the world and is hungry for innovative drugs, the dynamics of pricing, competition, and regulatory environment pose significant challenges. Companies venturing into China must navigate these complexities with a nuanced strategy, focusing on asset selection, market positioning, and long-term sustainability. The shift in the industry also highlights the need for adaptable business models that can withstand market fluctuations and capitalize on emerging opportunities. As the Chinese pharmaceutical landscape continues to evolve, lessons from LianBio's experience will be crucial for future entrants and existing players aiming to succeed in this competitive market.
Your Turn
How can foreign biopharmaceutical companies adapt their strategies to succeed in the ever-changing Chinese pharmaceutical market?
?? Politics & Policy ???
?? FDA's Nod to Amyloid Reduction: A Shift in Alzheimer's Drug Development?
The Facts
The FDA has updated its guidance on Alzheimer's drug development, suggesting that amyloid reduction can serve as a surrogate endpoint for predicting clinical benefit in certain circumstances. This update follows the controversial approval of Aduhelm and the recent nod to Leqembi, both targeting amyloid beta aggregates. While the FDA still considers cognitive and functional measures as generally acceptable co-primary endpoints, the new draft guidance acknowledges the challenges in early-stage Alzheimer's trials. It proposes alternative approaches, including cognitive assessments or surrogate endpoints like amyloid reduction, which may allow for shorter trial durations.
Our Opinion
The FDA's updated guidance reflects a nuanced understanding of the complexities in Alzheimer's drug development, especially in early-stage disease. By considering amyloid reduction as a surrogate endpoint, the agency is opening doors for innovative trial designs and potentially faster paths to approval. However, this approach also underscores the need for continued research to validate the link between amyloid reduction and clinical outcomes. As the field progresses, it will be crucial to balance the urgency for new treatments with the rigor of scientific validation to ensure that approved therapies truly benefit patients.
Your Turn
What are the implications of using surrogate endpoints like amyloid reduction for patients, clinicians, and the broader healthcare system?
?? Gene Therapy's Tough Choice: Curing Sickle Cell Disease at the Cost of Fertility
The Facts
Two new gene therapies for sickle cell disease, developed by Bluebird Bio Inc. and partners Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG, offer a potential cure for the condition but come with a significant drawback: the risk of infertility due to the toxic drugs used in the treatment process. This has led to concerns among patients and their families, as they face the difficult decision of choosing between a cure for a debilitating condition and the ability to have biological children in the future. The Biden administration has proposed a plan to help cover the cost of fertility preservation for Medicaid recipients receiving these treatments, but it won't take effect until next year and participation is voluntary.
Our Opinion
The dilemma faced by patients with sickle cell disease highlights a broader issue in the development of gene therapies: the need to consider the long-term quality of life implications of treatment. While the scientific breakthroughs represented by these therapies are undoubtedly significant, they also raise ethical and practical questions about patient autonomy and informed consent. As gene therapy continues to advance, it will be essential to ensure that patients are fully informed of the potential risks and benefits, including the impact on fertility, and that support mechanisms are in place to help them make the best decisions for their future.
Your Turn
What measures could be implemented to ensure that all patients, regardless of insurance status, have access to fertility preservation services when undergoing gene therapy?
?? Biden's 2025 Budget Blueprint: A Balancing Act Between Health Care Goals and Fiscal Constraints
The Facts
President Biden's 2025 budget proposal of $130.7 billion reflects a decrease from the previous year and aims to address key health care priorities while navigating fiscal constraints. The plan emphasizes lowering health care costs, expanding insurance coverage, and enhancing cybersecurity in hospitals. It includes funding for addressing drug shortages, promoting biosimilar substitution, and supporting the Office of Climate Change and Health Equity. The budget also proposes changes to mental health care accessibility and maintains or marginally increases funding for various medical research and infectious disease programs. The proposal faces challenges in gaining congressional approval and balancing the budget amidst Republican calls for non-defense spending caps.
Our Opinion
President Biden's 2025 budget proposal is a strategic attempt to prioritize critical health care issues while acknowledging the need for fiscal responsibility. The focus on drug shortages, biosimilar substitution, and mental health care changes demonstrates a commitment to improving patient outcomes and reducing costs. However, the flat or marginally increased funding for several key programs, such as HIV/AIDS and rural health, raises concerns about meeting long-term public health goals. The proposal's success will depend on its ability to navigate the political landscape and secure bipartisan support for its initiatives.
Your Turn
What are the potential implications of the flat funding for HIV/AIDS and rural health programs on achieving public health objectives?
?? Victory for Hospitals: Appeals Court Upholds Arkansas Law on 340B Drug Discount Program
The Facts
A U.S. appeals court has upheld an Arkansas law that supports hospitals participating in the 340B drug discount program, allowing them to ship medicines to certain pharmacies. This decision is a setback for the pharmaceutical industry, which has been limiting discounts for hospitals that use contract pharmacies. The 340B program, designed to help low-income and rural patients, has seen discounted purchases reach $53.7 billion in 2022. The ruling is expected to encourage other states to adopt similar laws, potentially increasing access to discounted medicines for vulnerable populations.
Our Opinion
The appeals court ruling is a significant win for hospitals and clinics serving low-income and rural patients. By upholding the Arkansas law, the court has reinforced the importance of the 340B program in ensuring access to affordable medications. This decision highlights the need for a balanced approach that considers both the interests of the pharmaceutical industry and the healthcare needs of underserved communities. As more states consider similar legislation, it is crucial to maintain the integrity of the 340B program and its mission to support healthcare providers in delivering essential services.
Your Turn
How can states balance the interests of the pharmaceutical industry with the healthcare needs of underserved communities when considering similar legislation?
Disclaimer: The contents of this article are not to be construed with investment advice. The information presented in this article is a compilation of current events, technical analyses, corporate press releases, and the author's personal viewpoints about the biotechnology industry. While efforts have been made to provide accurate and timely information, there may be inadvertent errors, omissions, or inaccuracies. Therefore, investment decisions should not be made solely based on the content of this article. The article may contain statements that are forward-looking in nature, encompassing predictions and future expectations that are subject to inherent risks and uncertainties; as such, actual outcomes may significantly deviate from those expressed or implied herein. This article serves purely as an informational and entertainment resource, and should not be construed as an endorsement to purchase or sell any financial securities. Prior to engaging in any investment activities, it is imperative that you conduct comprehensive due diligence and consult with a qualified financial advisor.