David Uffer

How do we minimize the number of protocol amendments and increase the operational feasibility of our clinical trials? This article below points out the benefits of using data, conversations and real world data to enhance the feasibility of the protocol design and inclusion/exclusion criteria: 1) Are we being too strict on our criteria in order to "control" for data variability? 2) Do are criteria allow for the intended patient population in the real world (if not, they need to) 3) How realistic is your design in how the study sites actually work? How they see and treat patients? How is your company ensuring the design meets these requirements? https://lnkd.in/gzJzFd-3

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🚨 The #FDA's latest draft guidance on accelerated approvals takes aim at "dangling" approvals—products still marketed despite failed confirmatory trials. The agency is now mandating tighter timelines (2-4 years for oncology), more transparency in withdrawal processes, and public involvement. 🕵️‍♂️ This marks a shift from flexibility to rigor, signaling that the accelerated pathway is no longer a free pass. For #biopharma, it's both a challenge and an opportunity to align trial strategies earlier. Is this the FDA tightening the reins—or just evolution in regulatory science? FDA website: https://lnkd.in/gqc-z-5e #pharmanews #breakingnews

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Not embarrassed to share Auxilius had a bit of cosmetic work done over the holidays. 💅 As of today, you’ll notice that the Auxilius website has a very different look and feel heading into 2025. While this is part of our effort to make clinical finance sexy (again?), we believe this more clearly reflects and articulates the way Auxilius supports the most innovative Biopharma finance and accounting teams in our industry. 🦾 The things we have in store for 2025 are anything but cosmetic. Auxilius customers will see a similar degree of change in our product this year. As our product becomes more robust (i.e., powerful, but big), we are making bold enhancements to our core workflows We are publicly launching three new offerings in 2025 — each designed to help our customers better plan and manage the most complex and variable components of R&D spend. Each has been designed and built collaboratively with early adopters among our 60+ customers, and we’re excited to make these available to the market broadly this year. 🔥 So yeah, I’ll say it. I’m fired up for 2025... https://www.auxilius.co/

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Recently I gave a talk at the LEAP conference on how precision medicine and diagnostics are the keys to value based care. In short, my perspective is that for patients, if we don't appropriately target care to their needs, there is no value to patients. If we treat them like the average, instead of using their data to tailor treatment, there is no value. If we don't correctly assess their risk, accurately diagnose their disease, prescribe therapies most likely to work, prevent adverse events, there is no value. But from a hospital system perspective, apparently that's not how we get "value". From STAT, the CEO of MassGeneral Brigham"...we have been aggressively pushing value-based care now for a number of years. Value-based care is one of the foundational goals and system priorities that we have. Value-based care, in its essence, means to take those patients who do not require the care in the academic medical centers and move them into a community setting, which is lower-cost, or an ambulatory site, which is even lower-cost than our academic medical centers." That definition of value based care means pushing patients to lower-cost centers, which reduces operational costs to a hospital system like MGB. But does that provide value to the patient? #valuebasedcare #precisionmedicine #diagnostics https://lnkd.in/eWUSXf_B

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I've been spending a lot of time thinking about the productivity problem in life sciences R&D - which, in my opinion, seems to be the single biggest problem to solve in all of life sciences.   Inefficiency takes on an entirely new meaning in biopharma vs. other industries where it simply amounts to sunk cost. In biopharma - particularly for the long tail of smaller biopharmas - it can be the difference between missing and meeting milestones, securing and not securing funding, life and death. Being efficient is increasingly a requirement for survival. Tech certainly isn't a golden bullet, but we see a lot of opportunity for it to help move the needle, especially if it's in combination with behavioral/cultural change.   Over the last month or so I've been sharing our high-level thoughts on this topic through a series of blog posts, the last of which was published today (see comments for links to the prior two posts). This really only begins to scratch the surface on our thinking here and we're eager to chat with folks in greater detail. If you’re working in this space or have insights to share, I'd love to hear from you! https://lnkd.in/g9EsD5b3

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In September, the results of the Phase III trial of biospecific antibody Ivenescimab delivering a meaningful improvement over Merck’s Keytruda in lung cancer sent Summit Therapeutics (NASDAQ: SMMT) stock soaring. However, few paid attention to the originator of the PD-1/VEGF targeting asset, Chinese biotech Akeso Bio. Akeso represents a cohort of emerging innovative drug companies from China, working at impressive speed to deliver advancements in patient care and next-generation therapeutics with global ambition. #deeptech #chinesedeeptech #bosonventures #Ivenescimab #biotech #immuology

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What is the cost of delay in drug discovery?   At imec.xpand, we see numerous companies that are developing disruptive technologies to accelerate drug discovery. What's striking is the wide range in projected financial benefits of this acceleration for pharmaceutical companies - from $0.5M to $8M per day. Are these figures grounded in reality? 🤔   Ken Getz, Executive Director and Research Professor at Tufts University School of Medicine, offers valuable insights. The higher estimates seem to stem from a 1993 study suggesting $4M per day, partially adjusted for inflation. Interestingly, their analysis shows that - over the past 30 years - the cost of delay has actually declined to approximately $0.5M per day, rather than increased. Ain't that unexpected?! 😮   It also means that accelerating drug discovery by one month still represents about $15M in value. So still worth it … but … the benefits brought by the new technology should extend beyond mere acceleration, at least if you want to generate some investor interest. A good example of what we find interesting at imec.xpand is e.g., a disruptive technology that not only significantly accelerates early drug discovery but also offers functional and molecular insights that help researchers at pharmaceutical companies to chose the better development candidate with a better safety profile.   Where do you see the greatest potential to accelerate drug discovery? And which additional benefits could the corresponding technology bring? Share your thoughts below.   And for those interested in Ken Getz's analysis: https://lnkd.in/ekUVg_XS   #drugdiscovery #pharmaceuticals #innovation #biotech #techbio #platform imec imec.xpand

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Fabulous to be on the investor panel for Mintz’s Life Sciences Pitch Event today, on yet another glorious fall day in Boston. ☀️🍁 Key feedback shared by our panel: 🔹 Ensure your target market is clear - especially when the population for your relevant indication is highly heterogenous 🔹 Share your current assumptions about how your product may be used (e.g., chronically vs. episodically) and open questions 🔹 If you're exploring a range of commercial use cases, share how you anticipate prioritizing across them over time 🔹 If your GTM strategy requires you to displace heavily entrenched solutions and/or processes, share how you will enable these transitions 🔹 Note the specific set of milestones to be achieved with investment 🔹 Always include target timelines and potential paths to exit Thank you to the incredible lineup of entrepreneurs who pitched today, and please stay in touch! Thank you to Dean Farmer and Charlie C. for facilitating today's event. Thank you to panelists David Fogel, CPA, Ben Littauer (he/him), Molly Hoult, and Richard Wagner for your contributions. Great to meet you, and thank you for all of your help, Emily (Krasowski) Yanachkov and Diana Malkin. #lifesciences #pitchevent #investor #panel #boston

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Large VC firms are raising billions but focusing on late-stage biotech companies with proven data and leadership. ARCH secured $3B and Flagship raised $3.6B - but they're backing just a few select companies. Meanwhile, smaller VCs like Wing Venture Capital and KdT Ventures target early-stage biotechs, taking calculated risks on innovative science. Early-stage companies now face tougher fundraising conditions compared to 2021's easy money era, with investors demanding more proof before writing checks. Key tip for biotechs: Research your investors carefully - each VC has specific focus areas and investment criteria. #OBI4You #VentureCapital #StartupFunding https://zurl.co/zmqt

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Biopharma's "mud-and-stick" season rolls on... From last week but worth noting Symbiotic Capital's new debt fund. This new vehicle from Arie Belldegrun's Bellco Capital is meant to provide debt to later-stage pharma. While these announcements never come on day 1 of a fund, what was truly astounding was that its already 40-50% deployed (!!!). https://lnkd.in/g7ehYBNR There's a lot of pain out there across the industry for a debt facility to be deployed that fast. You're seeing companies across the board hunker down, cut every program but their lead, and just focus on that, waiting (often times) for clinical readouts. Single assets are the new "platform". Which is why biopharma is in "mud and stick" season (my northeast friends will know what this is). Its not winter, but its definitely not spring. What are you seeing? Let me know! #pharma #investing #biotech

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🔥 Some very exciting investments over the last week or so! 🧬 Some really innovative companies and advancements! City Therapeutics ($135M): A Cambridge, MA-based biopharmaceutical company, raised $135 million in Series A funding to advance its RNAi-based medicine. Maven Clinic ($125M): A New York-based virtual clinic for women's and family health, Maven Clinic raised $125 million in Series F funding to enhance its partnerships in maternity care. Purespring Therapeutics ($105M): A London-based gene therapy company, they raised $105 million in Series B funding to support its pipeline, including initiating a Phase I/II clinical trial for IgAN. Glooko ($100M): Digital health company, Glooko raised $100 million in Series F funding to accelerate the global expansion of its digital healthcare platform for diabetes management. Cytovale ($100M): A SF-based company founded secured a $100 million Series D funding to develop rapid tests for early detection of sepsis. This accelerate bringing their innovative tools to market. Orasis Pharmaceuticals: Raised $78 million to launch Qlosi™, a new eye drop for the treatment of presbyopia. ShiraTronics, Inc.: Raised $66 million to support its FDA-approved pivotal trial, RELIEV-CM2, and seek premarket approval for its migraine therapy system. Basecamp Research: Raised $60 million to expand its data collection, strengthen its AI capabilities, and invest in computing to tackle more complex tasks in the life sciences. Arda Therapeutics Raised $43 million to expand its operations and development efforts in targeted cell depletion therapies for chronic diseases. Eleanor Health: Raised $30 million to expand its outpatient addiction treatment services. MARAbio Raised $19 million to support the commercial launch of its autism test in 2025 and the development of therapeutic interventions. Lymphatica Medtech: Raised €17.9 million to accelerate the clinical development and validation of LymphoDrain, an implantable device designed to treat lymphedema. Centaur Labs: Raised over $16 million to expand its operations and development efforts in health data annotation. Somagenetix: Raised CHF10 million to advance its leading gene therapy candidate, SGX-001, for phagocyte disorders. MiLaboratories: Raised an undisclosed amount to create software solutions that enhance biological research and development. Amprion Raised $6 million to expand its commercial reach and invest in research and development to improve brain disease diagnosis. Booster Therapeutics: Raised $15 million in seed funding. clock.bio: Raised $5.3 million to build its "Atlas of Rejuvenation Factors" by identifying genes influencing aging. LEVY Health Raised $4.5 million to integrate its virtual care pathways with electronic medical record software and expand its offerings to women's health and reproductive care. ALPHA FUSION INC. Raised ¥1.02 billion to expand its research and development efforts in targeted alpha therapy using Astatine-211 for cancer treatment.

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There's a deep personal history for me with this major acquisition today. Incyte (Rich Levy) had the vision at the time (2016) to let me design a very bold #phase2 #clinicaltrial with topical #ruxolitinib which ended up being the first FDA-approved JAK inhibitor in dermatology as OPZELURA® (ruxolitinib) HCP. Incyte acquires John E. Harris, MD, PhD's Villaris Therapeutics backed by Medicxi to go after #vitiligo. John E. Harris, MD, PhD and I are co-founders of Alys Pharmaceuticals which is going after #urticaria #itch #vitiligo and beyond again backed by Medicxi. In parallel in 2019, we publish an early and key paper in the journal Immunity (Meixiong et al. Immunity 2019) with Xinzhong Dong who discovered MRGPRX2 (actually, the entire family). I then start advising Escient Pharmaceuticals with the most bullish optimism about MRGPRX2 as target for #skin #diseases and witness a brilliant execution by Veena Viswanath et al. Incyte makes a bargain acquisition today for $750 million to transform skin disease in #dermatology and #allergy. The key point here is that MRGPRX2 antagonist will NOT be competing IMO with other more moderate-to-severe assets in #CSU like #barzolvolimab. They will mutually grow the market for #CSU across the mild to severe spectrum in a disease that is growing in incidence and poorly penetrated even by great drugs like #omalizumab. Keep in mind, #antihistamines are >$2 billion annual sales for #itch, which it does not even work for that well! This will accelerate new treatments for #urticaria and much beyond. Great for our patients.

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Excellent way to spend 1 minute, 30 seconds. DSO shares via an embedded video in the article below how Modality.AI is making an impact with patients. It is wonderful to have NIH SEED as a partner on this journey. Relentless Venture Fund David "DSO" Suendermann-Oeft #Neurotech #AI #BIO2024 #precisionmedicine #clinicaltrials #healthtech #healthcare #digitalhealth

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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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Takeda's CEO's annual letter hits on some key themes that many biopharma CEO's are converging on: value-based care, price transparency, breaking up the middlemen, and driving healthcare access and equity. "For several years I have been outlining Takeda’s thinking on health equity—including advocating for a transition to value-based health care that pays for outcomes and care quality, emphasizing the need to prepare for the next pandemic by ensuring global vaccine equity and explaining how the protection of intellectual property rights is essential to incentivize investment in R&D. One area where we would like to see greater progress this year is pharmacy benefit managers reform. We support efforts in Congress to delink rebates from list prices or wholesale acquisition cost. It is a complex system, but the bottom line is that the current system creates misaligned incentives that ultimately result in higher costs for the system and higher out-of-pocket costs for patients. One potential way to address these challenges is through greater transparency. Transparency at the point of purchase for patients would empower them to make decisions knowing exactly what their costs will be. This could lower costs for the entire system by building in greater pricing accountability along the chain so that no stakeholder can benefit from the inefficiencies that result in the prices that patients pay today." At COEUS, we are working with the world's leading biopharma companies to help transition them from a broken FFS model towards value-based care. We have built the world's leading platform that hosts the biggest CGT commercial launches, and rapidly moving into Oncology, Obesity/ Diabetes, and beyond. 1. Enabling Value-Based Healthcare: Our solution facilitates the transition to value-based healthcare by providing a powerful tool for creating and managing contracts that pay for outcomes and care quality. 2. Enhancing Transparency: By aggregating disparate healthcare data from various sources, our platform provides unprecedented transparency into the entire healthcare ecosystem. This empowers payers, providers, and patients to make informed decisions and helps stakeholders across the value-chain to tie price and value to therapeutic outcomes. 3. Driving Innovation: Our platform's ability to evaluate complex rules for value-based contracts, real-world evidence studies, and health equity measurements fosters innovation in the pharmaceutical industry. By providing a tool to measure the impact of new solutions, we help companies drive medical advancements while ensuring equitable access. 4. Improving Access and Affordability: Our solution helps identify areas where access and affordability can be improved without compromising innovation. By providing data-driven insights into the effectiveness of various interventions, we enable stakeholders to make informed decisions that benefit patients and the healthcare system as a whole. Visit https://coebra.ai/.

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May 6 2024 - 10 Notable Articles To Read Article: ‘Next wave of IPOs’ will arrive in summer, predicts biotech-focused VC partner Takeaway: The partner at Medicxi anticipates a surge in biotech initial public offerings (IPOs) during the summer. Source: Fierce Biotech Link: https://lnkd.in/d8XZHcKs Article: SVB Financial to sell VC business SVB Capital Takeaway: Pinegrove Capital Partners is set to purchase the venture capital enterprise, a newly formed entity supported by both Brookfield Asset Management and Sequoia Capital Heritage. Source: Reuters Link: https://lnkd.in/dDX5gP7F Article: Eye On AI: After Q4 Slowdown, AI-Related M&A Picked Back Up In Q1 Takeaway: In Q1 2024, there was an uptick in M&A exits, with 55 startups being acquired, marking an increase from Q4 2023. Source: Crunchbase Link: https://lnkd.in/dYbcfimk Article: Roark Capital completes $9.6bn Subway deal Takeaway: A private equity firm specializing in leveraged buyout investments within the franchise restaurant and food sectors has finalized the acquisition of Subway. Source: Private Equity Insights Link: https://lnkd.in/d88rwqYZ Article: Bolt closes €220M revolving credit facility: a step towards IPO Takeaway: The company aims to expedite the shift from individual car ownership to shared mobility, providing superior alternatives for various needs as part of its journey towards an IPO. Source: Tech.eu Link: https://lnkd.in/d_q7nCGK Article: The Stock Market Will Rise Nearly 10% More This Year, Money Managers Predict in Barron’s Latest Poll Takeaway: Numerous individuals anticipate that a stable economy will result in a more robust performance in the stock market. Source: Barron's Link: https://lnkd.in/d3tet-GY Article: Private equity firms step up plans to edge banks out of low-risk lending Takeaway: Apollo Global Management, Inc. issued a historic $40 billion in loans, reflecting a similar expansion seen among competitors such as KKR and Blackstone, as they raise long-term projections within this strategy. Source: Financial Times Link: https://lnkd.in/dv6AUJtb Article: Insight Partners’ Hinkle: M&A Is The Key To Unlocking Startup Liquidity This Year Takeaway: Many believe that the existing liquidity shortage stems from a dearth of exits in the startup market, leaving VCs with numerous illiquid assets. Source: Crunchbase Link: https://lnkd.in/dPvFPSMi Article: Rite Aid Closes a Quarter of Stores as It Navigates Bankruptcy Takeaway: At the time it filed for Chapter 11, the store was operational in 17 states and shuttered 520 locations within a span of seven months. Source: Bloomberg Link: https://lnkd.in/dhTUyf78 Article: Mubadala Capital plans $13.5bn biofuels investment in Brazil Takeaway: The fund also supported initiatives to establish a new stock exchange in Brazil and is augmenting its investments in the country. Source: Financial Times Link: https://lnkd.in/d4uUK6Pt #news #research #business

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Eupraxia Pharmaceuticals Inc. ("Eupraxia" or the "Company") (NASDAQ: EPRX) (TSX: EPRX), a clinical-stage biotechnology company, today unveiled new pharmacokinetic ("PK") data from its Phase 2a Eosinophilic Esophagitis ("#EoE") program. Management believes these data underscore the unique capabilities of Eupraxia's proprietary DiffuSphere™ platform technology, which aims to provide precise, localized, safe, and effective long-lasting drug delivery. Most conventional drugs release in a pattern characterized by steep peaks and rapid declines in drug concentration, where peaks often lead to negative side effects, and troughs result in reduced efficacy. In contrast, #DiffuSphere™ is a unique microsphere that is designed to enable #precisedrugrelease into target tissues with a flat, stable, and long-lasting profile, minimizing potential adverse events associated with high-dose systemic delivery. What sets DiffuSphere™ apart is its composition: a pure drug crystal encased in a microns-thick polymer shell. Eupraxia's #proprietary #technology uses this polymer to precisely control a drug's release, ensuring high drug concentrations in the target tissues while minimizing exposure to the rest of the body. This #innovative approach has been observed in Eupraxia's clinical programs, demonstrating precision, tolerability, and extended duration of delivering fluticasone propionate ("FP") directly to the intended tissues for EoE and knee osteoarthritis ("OA") patients. #biotech #pharma #investing #mergers https://lnkd.in/gX8fii4X

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