The Biotech Beat: 11.6-11.12.23

Week of 11/6 - 11/12/23

by Joey Bose and Alex Crombie

??Upshot

?? The Next Frontier in Cancer Therapy: mRNA Infusions Aim to Revolutionize CAR-T Treatments: Discover how mRNA technology is set to transform cancer treatment with simpler, more accessible therapies.

?? Lilly's Zepbound Ignites Weight Loss Drug Market with FDA Approval, Challenging Novo Nordisk's Wegovy: Eli Lilly's FDA-approved Zepbound enters the weight loss market, offering a new challenge to Novo Nordisk's dominance.

?? Brewing Innovation: Synthetic Yeast Genome Project Nears Completion, Poised to Transform Biotech Medicine Production: The completion of the Synthetic Yeast Genome Project promises to unlock new potentials in biotech medicine production.

?? AstraZeneca's Strategic Play: Acquiring Eccogene's GLP-1 for Obesity Therapy Enhancement and Comorbidity Management: AstraZeneca's acquisition of Eccogene's GLP-1 therapy represents a strategic move to enhance treatment options for obesity and related comorbidities.

?? Heart-Healthy Weight Loss: Novo Nordisk's Wegovy Demonstrates Cardiovascular Benefits in Landmark Trial: Novo Nordisk's Wegovy shows promising cardiovascular benefits, signaling a new era in heart-healthy weight loss solutions.

?? Healthcare Chiefs Clash: Negotiating Drug Prices in America's Medicare Landscape: Top health officials debate the future of drug pricing, emphasizing the critical role of Medicare negotiations.

?? FTC Fights Pharma Patent Overreach, Aiming to Enhance Generic Drug Competition: The FTC is challenging pharmaceutical patents to foster competition and improve access to generic drugs.

?? Weighty Decisions: Policymakers Tackle Rising Costs of Obesity Drugs Amid Healthcare Debate: Policymakers consider innovative strategies to manage the soaring costs of obesity drugs in the healthcare debate.

? Senate Finance Committee Unanimously Backs Bills to Tame Drug Costs and PBM Practices: The Senate Finance Committee takes a united stand to regulate drug costs and pharmacy benefit manager practices.

?? Biotech startup CARGO Therapeutics looks to land one of the biggest Nasdaq listings in a barren year for life sciences initial public offerings: CARGO Therapeutics aims for a standout Nasdaq debut, potentially invigorating the biotech IPO scene.

?? AstraZeneca has recruited a new star to its budding GLP-1 show: AstraZeneca gears up to compete in the GLP-1 space with a promising new candidate from Eccogene.

?? Research, Development & Drug Approvals ??

The Next Frontier in Cancer Therapy: mRNA Infusions Aim to Revolutionize CAR-T Treatments

The Facts

Biotechnology firms, with Moderna at the helm, are pioneering a potentially groundbreaking approach to cancer treatment through mRNA infusions, offering a promising alternative to traditional CAR-T cell therapies. This innovative method involves directly engineering immune cells in vivo, rather than the current complex and side-effect-heavy ex vivo process. Carisma Therapeutics has showcased the first data in collaboration with Moderna, indicating successful macrophage engineering in mice to combat cancer, notably in metastatic pancreatic cancer models. Capstan and Orna Therapeutics have also revealed early successes, with Orna's circular RNA potentially allowing for longer-lasting CAR protein expression. Despite the anticipation, human trials remain on the horizon, with Orna's clinical trial expected in 2024.

Our Opinion

The shift towards in vivo engineering of immune cells via mRNA could represent a seismic shift in precision medicine, drastically simplifying and potentially democratizing access to CAR-T therapies. By reducing the need for individualized manufacturing and mitigating severe side effects, these treatments could become more widely available, significantly impacting cancer treatment paradigms. This leap forward not only holds immense promise for cancer patients but could also catalyze a broader application in treating autoimmune diseases, thus marking an important milestone in personalized medicine and reinforcing the transformative potential of mRNA technology, previously evidenced by its success in COVID-19 vaccines.

Your Turn

1. How might mRNA-based in vivo CAR-T therapies change the landscape for patients currently facing limited access to personalized cancer treatments?
2. Given the success of mRNA in COVID-19 vaccines, what implications could these developments have for investor confidence in mRNA technology's applicability to other diseases?
3. With the expectation of multiple doses due to mRNA's short-lived nature, what considerations should be taken into account to ensure patient compliance and treatment efficacy in a real-world setting?

Lilly's Zepbound Ignites Weight Loss Drug Market with FDA Approval, Challenging Novo Nordisk's Wegovy

The Facts

Eli Lilly's tirzepatide, branded as Zepbound for obesity, has received FDA approval, becoming a formidable rival to Novo Nordisk's Wegovy. Zepbound, targeting GLP-1 and GIP hormones, boasts up to 21% weight loss in trials—higher than Wegovy's 15%. Despite common side effects and a costly price tag of $1,059.87 per month, Zepbound is positioned to seize a significant share of the burgeoning $100 billion obesity treatment market. With Lilly’s anticipation of increased manufacturing capacity and a prospective broad patient base, Zepbound is expected to be available post-Thanksgiving. Meanwhile, Wegovy’s pre-existing cardiovascular benefit data may give it an edge with insurers.

Our Opinion

The approval of Zepbound marks a pivotal moment in obesity management, offering substantial weight loss potential with the added convenience of self-administration. This advancement is not just a win for Eli Lilly but represents a significant stride for the biotech industry in addressing a critical public health issue. The competitive pricing and the high efficacy of Zepbound could catalyze a market shift, benefiting consumers with more options and potentially driving down costs over time. However, the challenge remains in ensuring equitable access and managing supply to meet demand, which will be crucial for sustained industry growth and public health impact.

Your Turn

1. With Zepbound entering the market, how do you foresee the competition shaping the pricing and accessibility of obesity medications?
2. Considering the importance of long-term data for insurers, how should companies like Lilly navigate the interim period before such data becomes available for Zepbound?
3. How might the introduction of Zepbound influence research into personalized obesity treatments and the broader spectrum of weight management options?

Brewing Innovation: Synthetic Yeast Genome Project Nears Completion, Poised to Transform Biotech Medicine Production

The Facts

The Synthetic Yeast Genome Project (Sc2.0), an ambitious international endeavor spanning over 15 years, is approaching its goal of creating yeast cells with fully synthetic genomes. This groundbreaking work aims to deepen our understanding of genome function and advance the production of complex medications. The consortium, led by synthetic biologist Jef Boeke, has published 10 papers detailing the construction of most chromosomes, with efforts now focused on consolidating them into a single organism, anticipated to be completed within two years. The synthetic yeast, already comprising 54% of the organism's DNA, has had its genome decluttered by 10%, with non-essential repetitive DNA removed. Upon completion, this synthetic organism could revolutionize the biotech industry by streamlining the manufacture of biochemicals, drugs, vaccines, and biomaterials.

Our Opinion

The near completion of the synthetic yeast genome represents not just a scientific marvel but a beacon for biotechnological innovation. By reconstructing and understanding the yeast genome, scientists pave the way for revolutionary advancements in drug and vaccine production, potentially leading to more efficient and cost-effective methods. This project exemplifies the intersection of fundamental research and practical applications, demonstrating the potential to create more robust and stable genomes than their natural counterparts. For the biotech industry, the implications are profound, opening up possibilities for custom-designed organisms tailored for specific biomanufacturing processes, which could significantly accelerate the development and production of new therapies.

Your Turn

1. How could the completion of the synthetic yeast genome project impact the future of drug and vaccine production in terms of efficiency and cost?
2. In what ways might the synthetic genome be utilized to produce more complex medicinal compounds that are currently challenging to manufacture?
3. Given the potential for custom-designed organisms, what ethical considerations should the biotech industry keep in mind as this technology advances?

AstraZeneca's Strategic Play: Acquiring Eccogene's GLP-1 for Obesity Therapy Enhancement and Comorbidity Management

The Facts

AstraZeneca has made a strategic acquisition of Eccogene's GLP-1 obesity drug program for $185 million, signaling a commitment to not just the obesity drug market but also the potential of combination therapies. CEO Pascal Soriot aims to integrate ECC5004 with AstraZeneca's cardio pipeline drugs, such as the blockbuster Farxiga, to address obesity and related comorbidities like hypertension, diabetes, and heart disease. AstraZeneca's move reflects an anticipation of enduring obesity prevalence, with over a billion people globally affected. ECC5004 stands out due to its oral administration, which may offer better tolerability and fewer side effects compared to first-generation injectables. While still in early phases, with ECC5004 completing Phase I trials, AstraZeneca is preparing for rapid progression to Phase II, positioning itself alongside rivals like Novo Nordisk and Eli Lilly, who are also exploring combination therapies.

Our Opinion

AstraZeneca's investment in Eccogene's GLP-1 therapy represents a savvy and forward-thinking approach to tackling obesity and its associated health conditions. By planning to combine ECC5004 with existing cardiovascular and diabetes treatments, AstraZeneca is not just expanding its portfolio but potentially revolutionizing the way we approach complex, interrelated health issues. This holistic strategy could greatly enhance patient outcomes and underscore the importance of integrated care in chronic disease management. If successful, AstraZeneca's approach may set a new standard in the industry, showcasing the power of strategic drug combinations over single-therapy interventions.

Your Turn

1. How do you think AstraZeneca's strategy of combining ECC5004 with cardiovascular treatments will affect the management of comorbidities in obesity?
2. What impact could the oral administration of ECC5004 have on patient compliance and the overall success of obesity treatment regimens?
3. Considering the competitive nature of the obesity drug market, how should AstraZeneca position ECC5004 to differentiate it from injectable therapies offered by rivals like Eli Lilly and Novo Nordisk?

Heart-Healthy Weight Loss: Novo Nordisk's Wegovy Demonstrates Cardiovascular Benefits in Landmark Trial

The Facts

Novo Nordisk's obesity drug Wegovy has made significant strides by reducing major cardiovascular events by 20% in a landmark trial, affirming the health benefits beyond mere weight loss. Detailed results from the trial, named Select, revealed a 28% reduction in heart attacks and a 19% decrease in all-cause mortality among patients. The trial's success has the potential to reshape the perspective of insurers who have been hesitant to cover obesity drugs, seeing them as cosmetic rather than medical necessities. Wegovy's performance in this longest trial to date, lasting five years, highlights its enduring efficacy and safety, with patients maintaining an average 10% weight loss. This pivotal achievement may not only enhance Novo Nordisk’s market position but also influence Medicare coverage policies, despite the drug's annual cost exceeding $16,000.

Our Opinion

The Select trial's results mark a watershed moment for the biotech industry, as it underscores obesity's tangible cardiovascular risks and the effectiveness of pharmacological interventions. Novo Nordisk's Wegovy, with its newfound cardiovascular benefits, presents a compelling case for the reassessment of obesity drugs from lifestyle to essential medical treatments. This shift could pave the way for improved patient care and potentially lower overall healthcare costs by preemptively addressing the comorbidities of obesity. Such data substantiating the therapeutic value of obesity drugs could encourage a more receptive stance from payers and regulators, fostering a landscape where medical treatments for obesity are as standard and accessible as those for other chronic conditions.

Your Turn

1. How might the cardiovascular benefits of Wegovy influence the perception and insurance coverage of obesity drugs?
2. In light of Wegovy's demonstrated long-term efficacy and cardiovascular benefits, what steps can healthcare providers take to ensure broader patient access to such treatments?
3. Considering the substantial cost of Wegovy, how should Novo Nordisk address affordability to ensure the drug's benefits are realized across diverse socioeconomic groups?

?? Politics & Policy ???

Healthcare Chiefs Clash: Negotiating Drug Prices in America's Medicare Landscape

The Facts

At the Milken Future of Health Summit, contrasting views were presented by current HHS Secretary Xavier Becerra and former Secretary Alex Azar regarding the negotiation of drug prices under Medicare. Becerra defended the Inflation Reduction Act's provision allowing Medicare to negotiate drug prices, despite criticisms that it could delay drug launches and stifle innovation. Azar, contrasting, voiced concerns over international pricing disparities and the impact on patient out-of-pocket expenses. Seventy-six percent of Americans support federal negotiation, a slight decrease from previous polls. The debate underscores the complexity of drug pricing reform and the challenges in balancing innovation, affordability, and access.

Our Opinion

The debate between the two health secretaries highlights a pivotal moment in American healthcare policy. Empowering Medicare to negotiate drug prices could be a transformative step toward making essential medications more affordable for millions. While concerns about innovation and access are valid, the overwhelming public support indicates a mandate for change. The pharmaceutical industry's profitability, even in countries with lower drug prices, suggests that a balance can be struck. This move could lead to a more sustainable healthcare system where patient welfare and industry innovation coexist without the former being compromised by prohibitively high costs.

Your Turn

1. How do you believe Medicare's new negotiation powers will impact the cost of drugs for patients and the pace of pharmaceutical innovation?
2. In what ways can the government ensure that drug price negotiations lead to actual savings for consumers without hindering the development of new therapies?
3. Should the focus on drug cost reduction consider the global disparity in drug pricing, and how might this affect the pharmaceutical industry's investment in research and development?

FTC Fights Pharma Patent Overreach, Aiming to Enhance Generic Drug Competition

The Facts

The FTC is actively challenging over 100 patents on brand-name drugs listed inaccurately in the FDA's Orange Book, targeting major pharmaceutical companies like AbbVie and AstraZeneca. This crackdown is a response to strategies that allegedly delay generic competition and extend brand monopolies, affecting drug affordability. By giving companies 30 days to rectify or affirm their patent listings, the FTC is enforcing compliance and promoting fair competition. The initiative supports the Biden administration's broader efforts to combat patent abuses and aligns with proposed legislation to refine patent reviews, balancing innovation with public access to more affordable medications.

Our Opinion

The FTC's rigorous stance on patent listings represents a decisive step toward dismantling artificial barriers to generic drug entry, which can artificially inflate healthcare costs. These actions align with a growing consensus that while patent protection is vital for fostering innovation, it should not be leveraged to unjustly hinder generic market entry and prolong high drug prices. This scrutiny could not only precipitate a drop in medication prices but also stimulate the pharmaceutical industry to invest in genuinely innovative treatments rather than minor modifications with limited therapeutic advancement. By prioritizing public health interests over excessive profit margins, the FTC's move could catalyze a more equitable and economically efficient healthcare system.

The Facts

1. How might the FTC's challenge to the listing of patents in the Orange Book influence the future pricing strategies of pharmaceutical companies?
2. In what ways can patent law be reformed to encourage genuine innovation while preventing the misuse of patent extensions for minor drug modifications?
3. What are the potential implications for patient access to medications if the FTC's push for more rigorous scrutiny of patent listings is successful?

Weighty Decisions: Policymakers Tackle Rising Costs of Obesity Drugs Amid Healthcare Debate

The Facts

Washington policymakers are grappling with the surging costs of obesity drugs like Novo Nordisk's Wegovy and Ozempic, which have hit blockbuster sales. With a new FDA-approved rival, Eli Lilly's Zepbound, entering the market, the debate intensifies over strategies to manage these expenses. Senator Bill Cassidy suggests NIH-sponsored studies to explore transitioning patients from long-term drug therapy to dietary treatments after initial medication periods. The current annual costs of these drugs, reaching up to $16,000, challenge the cost-effectiveness for lifelong use. Legislation is being considered to permit Medicare coverage for these drugs, potentially expanding access to over 60 million Americans, while discussions continue on balancing drug cost containment with the stimulation of pharmaceutical innovation.

Our Opinion

The proposal to investigate alternatives to indefinite medication for weight management reflects a pragmatic approach to healthcare spending without compromising patient outcomes. It aligns with the ethos of sustainable healthcare practices, aiming to reduce long-term reliance on costly drugs. While the pharmaceutical industry argues that these drugs reduce rates of diabetes and heart disease, thereby saving costs, independent studies question their lifetime cost-effectiveness. This debate underscores the need for innovative solutions that can provide both economic relief and maintain medical advancements. Such a dual strategy could potentially offer a more balanced healthcare model, optimizing both patient care and industry growth.

Your Turn

1. How could NIH-sponsored studies on transitioning from drug therapy to dietary treatments impact the future use and funding of weight loss drugs?
2. What are the potential benefits and drawbacks of allowing Medicare to cover obesity drugs like Wegovy, Ozempic, and Zepbound?
3. In balancing drug innovation incentives with public health costs, what approaches can ensure that new treatments are both affordable and conducive to continued medical progress?

Senate Finance Committee Unanimously Backs Bills to Tame Drug Costs and PBM Practices

The Facts

The Senate Finance Committee has unanimously passed a suite of reforms targeting pharmacy benefit managers (PBMs) and aiming to reduce drug costs for Medicare and Medicaid beneficiaries. This bipartisan move seeks to enhance transparency in drug pricing negotiations and limit excessive cost-sharing, potentially reshaping the $4 trillion American healthcare system. The legislation mandates contracts with pharmacies in underserved areas and ensures patient cost-sharing reflects net drug prices post-rebates. While PBMs caution against the changes, citing potential instability in Part D plans, community pharmacists and patient advocates endorse the proposals, highlighting the struggle against PBM-dominated market practices.

Our Opinion

The committee's unified vote reflects a significant consensus on the need for reforms in the pharmaceutical pricing system, particularly concerning the role of PBMs. These measures could usher in an era of greater equity in drug pricing, potentially reducing the financial burden on patients and offering a lifeline to independent pharmacies. As the debate around healthcare costs continues, this legislative package represents a concrete step towards aligning drug pricing with national interests in affordability and access. However, it will be essential to monitor the impact of these changes to ensure they bolster, rather than destabilize, the Part D program, balancing patient benefits with the health system's overall sustainability.

Your Turn

1. How could the proposed changes to PBM practices impact the overall costs of prescription drugs for Medicare and Medicaid beneficiaries?
2. What might be the long-term effects on independent pharmacies if this legislation is passed, and how will it affect their role in the healthcare system?
3. Considering the PBMs' concerns, how can policymakers ensure that the reforms strengthen rather than destabilize Medicare Part D plans?

?? Investment, M&A, and IPOs ??

CARGO Therapeutics Set to Navigate Nasdaq with Promising IPO in a Challenging Market

The Facts

CARGO Therapeutics, a biotech startup focusing on blood cancer cell therapy, is positioning itself for one of the most significant Nasdaq listings of the year, targeting a $300 million IPO at a proposed price of $16 per share. With 18.75 million shares on offer, CARGO seeks to capitalize on its innovative CD22 CAR-T cell therapy, despite a tough year for life sciences IPOs. The company's IPO could provide a runway through 2025 when it expects interim data from its lead blood cancer program. This move comes at a time when many biotechs are trading below their IPO price, indicating a cautious investor stance towards the sector.

Our Opinion

CARGO's IPO ambition amidst a difficult market environment for biotech firms highlights both the resilience and the potential of innovative therapies in the industry. While the broader market for IPOs remains tepid, with macroenvironmental factors and a retreat of generalists from biotech, the fact that companies like CARGO are proceeding with listings suggests confidence in the long-term value of their scientific advancements. Such a move reflects a strategic gamble that high-stakes research can attract enough investment to support significant medical breakthroughs, despite current market hesitancies.

Your Turn

1. How might CARGO Therapeutics' IPO influence the perception of risk and potential reward for investors considering the biotech sector?
2. In what ways can startups like CARGO navigate the challenges presented by the current state of the market to achieve successful public offerings?
3. Given the current market conditions, what factors should investors consider when evaluating the potential of new biotech IPOs like that of CARGO Therapeutics?

AstraZeneca Dives into the GLP-1 Arena with Eccogene’s Oral Candidate Acquisition

The Facts

AstraZeneca has made a strategic move by acquiring ECC5004, an oral GLP-1 receptor agonist candidate from Chinese biotech Eccogene, with an upfront payment of $185 million. This acquisition is seen as a bid to compete in the lucrative GLP-1 market, targeting type 2 diabetes and weight loss treatments. Despite being behind market leaders like Novo Nordisk and Eli Lilly, AstraZeneca's leadership expresses confidence in ECC5004's potential to become a "best in class" option. The deal, which includes $1.825 billion in milestones, grants AstraZeneca exclusive rights outside of China and reflects its commitment to expanding its cardiometabolic disease portfolio.

Our Opinion

AstraZeneca's investment in Eccogene's ECC5004 could signal an emerging shift in the treatment of cardiometabolic conditions, leveraging the convenience of oral administration to potentially improve patient adherence and outcomes. With the GLP-1 market rapidly expanding, AstraZeneca's entry, albeit trailing industry giants, underscores the importance of continual innovation in addressing the global burden of obesity and diabetes. The deal's structure, which preserves co-development and co-commercialization rights for Eccogene in China, also illustrates a collaborative approach to global drug development and commercialization, potentially benefiting a wider patient population.

Your Turn

1. What impact could AstraZeneca's new oral GLP-1 candidate have on the current landscape of diabetes and weight loss therapies?
2. How might AstraZeneca's later entry into the GLP-1 market shape its strategy for competing with established players like Novo Nordisk and Eli Lilly?
3. Considering the substantial milestone payments involved, what does this deal indicate about the value and future potential of ECC5004 and the overall GLP-1 market?