Sharoni Billik, MBA, MBS

Amylyx Pharmaceuticals has acquired avexitide, an investigational therapy for hyperinsulinemic hypoglycemia, from Eiger BioPharmaceuticals. Avexitide, a novel GLP-1 receptor antagonist, has potential applications in treating post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI). The U.S. FDA has granted avexitide Breakthrough Therapy Designation for PBH and congenital HI, along with an Orphan Drug Designation for treating hyperinsulinemic hypoglycemia. Phase 2 studies have shown promising results in reducing hypoglycemic events, paving the way for a Phase 3 trial in PBH in 2025. Amylyx's acquisition underscores its commitment to advancing endocrine and neuroscience treatments for unmet medical needs. Learn more: https://lnkd.in/dK4xSW_r Ryan Miller Tammy Sarnelli Machelle Manuel

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Eli Lilly and Company’s Zepbound was superior to Wegovy in head-to-head trial showing an average weight loss of 20.2% vs. 13.7%, at 72 weeks, from 751 participants. This equates to 47% greater weight loss with Zepbound; in addition, 31.6% of people taking Zepbound achieved at least 25% body weight loss compared to 16.1% of those taking Wegovy. Impressive stuff! On the day of the announcement, Eli Lilly shares rose 2.5% to a three-week high, adding some $19 billion to its market value and taking it over $790 billion, while Novo Nordisk's Copenhagen-listed shares fell as much as 1.8%. I look forward to full publication of the data, specific to how the outcomes varied across patient profiles, to deliver personalized care. https://lnkd.in/ezm-UUyR

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Get to know the current & future MAFLD/MASH agents! Rezdiffra from Madrigal Pharmaceuticals has transformed the metabolic dysfunction-associated steatohepatitis (MASH) treatment area with its approval earlier this year. Looking at the attached PDF, it seems like that first-mover advantage could last some time based on the latest clinical development status from the closest MAFLD/MASH pipeline competitors. However, what is less certain is whether use of the GIP/GLP-1 agents for other conditions like obesity and type 2 diabetes will ultimately reduce the potential opportunity for MAFLD/MASH therapies. It is possible that treatment of patients for diabetes and obesity (often precursors of MAFLD/MASH) could shrink the target MAFLD/MASH patient population in a few years. Stay tuned to see if any upcoming clinical updates change the timing of and opportunity for a second FDA-approval in MASH. #MAFLD #MASH #prescriptiondrugs #drugspipeline #healthcareinnovation #pharmaceuticals #medicalresearch #biotech #clinicaltrials #drugdevelopment

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SEQSTER and Antidote are transforming patient matching and clinical research efficiency. SEQSTER’s patient-centric platform offers a comprehensive view of medical history, genomic data, and lifestyle factors, while Antidote’s engagement platform accelerates clinical trial recruitment. By integrating SEQSTER’s technology, Antidote accurately matches patients to trials, ensuring better patient quality and faster recruitment. This partnership facilitates real-time access to participant health data, streamlining the path to medical advancements. Ardy Arianpour, CEO and Co-Founder of SEQSTER, highlights the transformative potential of this collaboration in addressing recruitment challenges. Samantha Veeck, Co-CEO of Antidote, emphasizes the importance of connecting suitable patients to trials through advanced technology and personalization. The partnership also promotes diversity in drug development by providing access to patient records, enhancing representation of underserved populations in trials. This meets FDA diversity requirements and improves research generalizability. Integrating SEQSTER’s and Antidote’s technologies simplifies patient enrollment, reduces study initiation times, and enhances patient engagement and data accuracy. This partnership equips researchers with the tools for successful clinical research and innovative treatments. Learn more: https://lnkd.in/daJE_Dsk 🇨🇦Dana Hosseini ⬥ Ardy Arianpour

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Recently, Dr. Alexandra MacLean, CEO of Avenue Therapeutics, hosted Dr. Christopher Grunseich (National Institute of Neurological Disorders and Stroke) and Dr. Tahseen Mozaffar (UC Irvine) in a virtual key opinion leader event to discuss #SpinalBulbarMuscularAtrophy (#SBMA), also known as #KennedysDisease. Currently, there are no FDA approved treatments for this rare neurodegenerative disease. The event focused on the current understanding of the condition including unmet needs, and an overview of Avenue’s Phase 1/2a trial of #AJ201 for the treatment of #KennedysDisease. Click here to view a replay of the event: https://lnkd.in/e5ZiDaiW

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The collaboration between Virpax Pharmaceuticals Inc. (Nasdaq:VRPX) Pharmaceuticals and the National Center for Advancing Translational Sciences (NCATS) heralds a significant advancement in pain management research. Focused on the development of NES100, an intranasal peptide targeting acute and chronic non-cancer pain, this partnership utilizes innovative nanotechnology to enhance the delivery of enkephalin to the brain. By leveraging Molecular Envelope Technology (MET) and a specialized device, NES100 aims to provide analgesic effects without the adverse events commonly associated with opioids. This extended collaboration underscores the shift towards non-opioid alternatives for pain management, crucial in addressing the opioid crisis. The research aligns with the NIH’s HEAL initiative, emphasizing the national commitment to combat opioid addiction. The potential of NES100 lies in offering a non-addictive pain treatment, potentially transforming pain therapy and improving outcomes for individuals with chronic pain conditions. NES100's development presents a promising avenue for pain management, offering a safer alternative to traditional opioids. If proven effective in clinical trials, NES100 could emerge as a valuable tool for patients seeking pain relief. The intranasal delivery method not only enhances convenience but also holds the promise of improved patient adherence and treatment efficacy. 𝐑𝐞𝐚𝐝 𝐌𝐨𝐫𝐞: https://lnkd.in/dwMpJZf8 Gerald W. Bruce Matt Barnes

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Great day for California Life Sciences (CLS) attending Physicians for a Healthy California, Health Equity Leadership Summit! Panelist, Maria Jison, MD, (Executive Director, Global Clinical Head, Fasenra) of member company AstraZeneca spoke to the strides they've made using their AIRE Mobile Unit as a “clinic on wheels” that brings trial screenings directly to patients in diverse communities. The mobile unit is equipped with exam rooms and staffed with healthcare professionals who are ready to pre-screen patients and book both an appointment and transportation for patients to visit the clinical trial site they’re referred to. Per Maria, "You can't be successful without community engagement and building trust. It's not enough to just show up in a bus.....you really need to be part of the community. A multi-pronged engagement campaign is required. Outreach alone isn't enough — you need to partner with other organizations that are part of the community and trusted." #EquitableResources #PhysiciansForAHealthyCalifornia #HealthEquity

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Exciting developments in the world of oncology have emerged with Cantex Pharmaceuticals, Inc.' latest therapeutic candidate, Azeliragon, showing significant promise in the treatment of cancer. Preliminary clinical trials have demonstrated that Azeliragon may reduce tumor progression, providing new hope in the fight against cancer. Azeliragon's unique mechanism targets specific inflammatory responses associated with tumor growth, distinguishing it from traditional treatments. Azeliragon is part of a broader study exploring its potential to inhibit inflammatory pathways, which are often active in various cancer types. The therapy works by modulating the body's inflammatory response, which is crucial in the progression of many cancers. Early-stage trials have shown favorable outcomes in slowing disease progression, signaling potential for future success in more advanced trials. Continued research and upcoming trials will provide further insights into the efficacy and safety of Azeliragon, potentially positioning it as a breakthrough in oncology. To delve deeper into the specifics of Azeliragon's development and its impact on cancer treatment, check out the full interview here. #OncologyResearch #Azeliragon #CancerTherapy #ClinicalTrials #InnovativeTreatment #HealthcareInnovation David Schemelia Stephen Marcus, MD

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How do people feel about DTC advertising? A recent study suggests many doctors and patients find the ad spots to be more helpful than harmful. However, doctors and patients retain different things. Not surprisingly, physicians hold on to clinical information. Patients focus on nonclinical elements like the music and celebrity spokespeople. Survey sponsors recommend tailoring the ads to doctors’ and patients’ individual preferences. Specifically, increase the clinical information to appeal to physicians and increase “interactive and informative ad formats” for patients. https://lnkd.in/ehMva83H #DTC #Insights #Biotech

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23andMe is letting go of their therapeutics division and 200 employees This publicly-traded genetic testing company has recently went through a high profile data breach, multiple rounds of layoffs, losses that plunged the company’s stock, and all but the CEO resigning from its board. It’s most popular product is saliva-based testing kits, but most only needing to purchase it once. They’ve been struggling to find a profitable business model. With these cuts, they anticipate reducing operating expenses will save the company $35M annually. It’s been difficult to see these once fast growing companies hit a ceiling and figuring out how to keep growing with the new market demands. #healthtech #startupjourney #startups

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💊 Acurx Pharmaceuticals’ Ibezapolstat Shows Impressive Results! 🌟 Acurx Pharmaceuticals, Inc. (Nasdaq: ACXP). compared its top-line data with current standard-of-care drugs, and the results are in—Ibezapolstat has shown impressive positive outcomes! This breakthrough is a promising step forward in the fight against bacterial infections. 🔥Be sure to watch the full interview, airing on Bloomberg this Saturday, October 12 , 2024, at 6:30 PM ET. 🔥🔥Tap the link for the article https://lnkd.in/eFwBEEhd #acurxpharmaceuticals #wellness #health #healthcare #cure #medicine #pharmacy #science #businessupdate #businessnews #business #news #newtothestreet

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Exciting news in biotech - Q1 2024 saw a 46% surge in venture funding, signaling a strong recovery. Late-stage clinical developments and innovative therapies like ADCs and radiopharmaceuticals received significant investments. The important question is what does this mean for the future of biotech? How will these trends impact the development of new therapies? And how is investor confidence changing as the IPO market sort of re-opens? Interesting thoughts to consider. You can read more on our blog: https://ow.ly/bpCF50S2iY6 #Biotech #VentureCapital #HealthcareInvestments

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Sarepta Therapeutics reported strong third-quarter 2024 financial results, marked by a significant revenue increase and positive net income. Net product revenue reached $429.8 million, a 39% year-over-year surge. This growth is largely attributed to the successful launch and expanded label of ELEVIDYS, their gene therapy for Duchenne muscular dystrophy (DMD). ELEVIDYS alone generated $181.0 million in net product revenue, surpassing previous projections. Including royalties from Roche’s ex-US ELEVIDYS sales, total ELEVIDYS revenue reached $190.5 million. The company’s other PMO therapies for DMD, EXONDYS 51, VYONDYS 53, and AMONDYS 45, also performed well, collectively contributing $248.8 million in net product revenue during the quarter. Notably, the launch of ELEVIDYS hasn’t significantly impacted sales of these established treatments. Sarepta is prioritizing its portfolio and advancing its pipeline, anticipating a Biologics License Application submission for one Limb-girdle muscular dystrophy (LGMD) program by mid-2025, with two additional LGMD programs entering clinical trials. Financially, Sarepta achieved a GAAP net income of $33.6 million and a non-GAAP net income of $67.0 million for Q3 2024. This positive performance contrasts with a net loss in the same period of the previous year. Total revenues for the first nine months of 2024 totaled $1.24 billion, a 47% increase compared to the same period in 2023. This increase reflects ELEVIDYS’ launch and label expansion, alongside collaboration revenue from Roche’s declined option to acquire ex-US rights to an early-stage Duchenne program. Additional revenue streams included contract manufacturing and royalty payments from Roche related to ELEVIDYS. Cost of sales, excluding amortization of in-licensed rights, rose due to ELEVIDYS’ launch. Research and development expenses increased in Q3 2024 due to costs associated with terminating a manufacturing and supply agreement, offset by decreased spending on other programs. However, for the nine months, research and development expenses decreased due to ELEVIDYS commercial batch capitalization. Selling, general, and administrative expenses increased due to ongoing commercialization efforts for ELEVIDYS, litigation matters, charitable contributions, and compensation expenses. Other income increased significantly compared to the previous year, primarily due to a one-time impairment of a strategic investment in 2023. The company uses non-GAAP financial measures to evaluate operational performance and cash requirements internally and to provide investors with a clearer comparison of period-to-period results. 𝐑𝐞𝐚𝐝 𝐌𝐨𝐫𝐞: https://lnkd.in/dC7mTfX6 Nicole K. Paulk, PhD Kim M. Grabbe, M.S.

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𝐁𝐢𝐨𝐭𝐞𝐜𝐡 𝐚𝐧𝐝 𝐏𝐡𝐚𝐫𝐦𝐚 𝐔𝐩𝐝𝐚𝐭𝐞𝐬: September 18 - 19, 2024 • GC Therapeutics launches with $75M to revolutionize cell therapy • Roche’s positive antiviral Ph3 data • Tanai Therapeutics’ obesity endeavours further fuelled by Novo Nordisk • Upstream Bio files for IPO • Basilea Pharmaceutica extends BARDA partnership • Vanda Pharmaceuticals' gastroparesis small molecule rejected by FDA • Biotech small-cap market goes “meh” on US Fed rate cut • Achilles Therapeutics plc axes TIL-based therapy program • Study finds new-gen migraine meds not much better than OTC regulars (eg. tylenol, aspirin) More in today's TLDR Biotech newsletter: https://lnkd.in/g5XN9hre #biotech #pharma #pharmanews #biotechnews #tldrbiotech

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