Derrell Porter, M.D.

When investors think your gene therapy isn't good enough... That's what Lexeo is feeling right now on LX2006 for cardiomyopathy associated with Friedreich's Ataxia. Their Ph1/2 trial saw 50% of patients (8 total in the trial) experience a reduction of 10% in left ventricular mass index. There are only a handful of companies developing therapies for Friedreich's Ataxia, let alone the heart-related components of this rare disease. While Lexeo was optimistic about the data and taking it to discuss a future pivotal study with regulators, the market was not, knocking the stock down at present ~25%. The public markets are brutal with earlier stage companies. Depending on capital needs, it can be a bit easier to not be public when you're early in development. Regardless, another piece of positive news for AAV as it slowly comes out of its winter. Lines up with our beginning of the year predictions below! https://lnkd.in/drRreSCd

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My #ASCO2024 conversations surfaced a common thread among sponsors: the increasing # of amendments within oncology studies, sometimes up to 8-10. Sponsors already have difficulty with visibility into the initial protocol's adherence, much less subsequent amendments. Is this a concern in your mind? Here is an example of an amendment almost causing a a significant and dangerous deviation, which TruLab caught in real time to keep the patient safe and avoid a deviation. #protocoladherence #patientsafety #realtimevisiblity 

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The 340B program is a topic that can elicit a lot of strong opinions among manufacturers, legislators, healthcare providers, and patients. No matter what your role may be in pharma, or outside of our industry, you should want to learn more about this complex and controversial issue. In my role, I spend a lot of time thinking about the 340B drug pricing program. It has huge implications in terms of drug pricing, value creation, access, and, ultimately, the profitability of our brands. However, the program is intended to help vulnerable patient populations through a rebate process, paid for by manufacturers, that can fund healthcare infrastructure, delivery of medical services, and access to medications. These are worthy goals, but is the spirit and intent of this program delivering on its promise? To help us answer this question, Dr. Na-Ri Oh and I talked with William Sarraille, a nationally recognized legal healthcare expert, regulatory consultant, and former partner at Sidley Austin LLP. Bill is a true expert in this space and has done a great job on LinkedIn with his almost daily posts helping us all better understand this complex issue. Listen here: https://lnkd.in/geUHiV3f Our discussion focuses on some of the warped incentives and lack of transparency that exist within the program and questions whether the intended beneficiaries of the program, patients, are actually benefiting in any meaningful way. We all want an efficient program that improves patients' lives and we also discuss program and policy changes that could be considered to achieve these objectives. We hope you can join us for this spirited conversation with one of the leading policy voices in the country on this issue. #340B #healthcareaccess

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🌟 Candid Therapeutics: Disrupting Cancer Treatment with a $370M Splash 🌟 Hot off the $4.1 billion sale of RayzeBio to Bristol Myers Squibb, biotech trailblazer Ken Song, M.D., is back with a groundbreaking venture—Candid Therapeutics. With an impressive $370 million Series A funding, Candid Therapeutics is set to dominate the T-cell engager space, bolstered by strategic acquisitions of Vignette Bio and TRC 2004. 🔑 T-Cell Engagers: The Future of Cancer Therapy T-cell engagers are the next-gen bispecific antibodies, engineered to precisely target cancer cells. Unlike the cumbersome CAR T-cell therapies, Candid’s flagship assets, promise revolutionary cancer treatment options. 🧑‍⚕️ Meet the Dream Team Timothy Lu, M.D., Ph.D.: Chief Medical and Scientific Officer Bernie Huyghe, Ph.D.: Chief Technology Officer Arvind Kush: Chief Financial and Business Officer Candid Therapeutics is on a mission to transform the biotech landscape! #Biotech #TCell #cancer #autoimmune

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According to the latest analysis from BiopharmIQ by Amp, Pennsylvania holds the #3 position in CGT ecosystems for companies with under 50 employees. However, as these companies grow, I hope they choose to mature in Philadelphia. Recently, I've had numerous conversations about the region's shortage of leadership/ executive talent. It's intriguing considering that 80% of Big Pharma is located within 90 miles of the city. What the CGT sector needs is more experienced leadership, and Big Pharma could serve as an excellent source for it. For the latest updates on Life Science and Biotech news in the Philadelphia region, join The Informal Philadelphia Life Science Group below!

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As part of the Inflation Reduction Act’s (#IRA) first round of #Medicare negotiations, the Biden administration has announced significant price reductions on 10 high-cost #prescriptiondrugs. Set to go into effect in 2026, these cuts will range from 38% to 79%, impacting major treatments for #diabetes, #heartfailure, and #autoimmunediseases in an effort to lower healthcare costs for #Medicarepatients. While the administration celebrates the potential savings for Americans, the #pharmaceuticalindustry argues that the changes may do more harm to patients by limiting access to treatments and stifling innovation. The ongoing debate highlights the complex balance between affordability with the availability of new treatments. 

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