Abhishek Gupta

Tune in live now or listen later to this pre-JPM #BiotechHangout discussion!

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𝗪𝗵𝗲𝗿𝗲 𝘄𝗶𝗹𝗹 𝗯𝗶𝗼𝘁𝗲𝗰𝗵 𝗶𝗻𝘃𝗲𝘀𝘁𝗺𝗲𝗻𝘁 𝗺𝗼𝗻𝗲𝘆 𝗴𝗼 𝗻𝗲𝘅𝘁 𝘆𝗲𝗮𝗿? According to Pitchbook...... * 𝗣𝗵𝗮𝘀𝗲 𝟮 and later clinical stage companies. Risk aversion is diminished the later the stage, and deeper pockets now prefer conservative bets. * 𝗚𝗟𝗣-𝟭 related obesity drugs. Now an obvious target pathway, many are trying to enter ASAP. Competition is for a $70 Bn market, but who can beat the global manufacturing capacity of Lilly or Novo? * 𝗚𝗲𝗻𝗲𝗿𝗮𝘁𝗶𝘃𝗲 𝗔𝗜. Still buzzy over the past 3 years, even though utility that leads to enhanced clinical success is still not so obvious yet. Application to predictively create potent Biologics is a major theme. I suppose any.asset that is a mid/late stage, GLP-1 obesity biologic, created by generative AI, will be the preferred word-salad for box-checking investors in the year to come. If Pitchbook 2025 prophecy is correct, then for everyone else currently seeking reduced early stage investments - "𝗦𝘂𝗿𝘃𝗶𝘃𝗲 𝟮𝟬𝟮𝟱" might be the motto for next year. Let's hope it does not turn out this way. 𝗙𝗶𝗲𝗿𝗰𝗲 𝗕𝗶𝗼𝘁𝗲𝗰𝗵 𝗿𝗲𝗽𝗼𝗿𝘁𝘀- " Against an economic backdrop of rising interest rates and tighter capital markets, PitchBook doesn’t expect investors’ appetite for companies with “clearer paths to monetization or Big Pharma acquisition” to wane any time soon. "

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RAPT Therapeutics Therapeutics and Shanghai Jemincare Pharmaceutical Announce Exclusive License Agreement for Novel Long-Acting anti-IgE Antibody SOUTH SAN FRANCISCO, Calif., Dec. 23, 2024 (GLOBE NEWSWIRE) -- RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based biopharmaceutical company focused on discovering, developing and commercializing novel therapies for patients with significant unmet needs in inflammatory diseases, and Shanghai Jemincare Pharmaceutical Co., Ltd (“Jemincare”), a subsidiary of Jiangxi Jemincare Group, a leading pharmaceutical company in China, today announced they have entered into an exclusive license agreement for JYB1904 (RAPT designation RPT904), a clinical-stage, half-life extended anti-immunoglobulin E (IgE) monoclonal antibody. Under the terms of the license agreement, RAPT is granted worldwide rights excluding mainland China, Hong Kong, Macau and Taiwan (together, the “Jemincare Territory”) to develop and commercialize RPT904. As consideration for the license, Jemincare receives a $35 million upfront license fee, up to $672.5 million in additional payments upon the achievement of various regulatory and commercial milestones, and royalties on future sales of RPT904 outside the Jemincare Territory. RPT904 is being developed to offer patients a potentially improved therapeutic option compared to omalizumab (marketed as Xolair®), an anti-IgE monoclonal antibody approved for several allergic disorders, including asthma, chronic spontaneous urticaria (CSU), chronic rhinosinusitis with nasal polyps and, most recently, food allergy. RAPT plans to pursue development of RPT904 initially in food allergy. Separately, Jemincare is conducting Phase 2 clinical trials of JYB1904 in China in asthma and CSU. “We’re delighted to partner with Jemincare and excited by RPT904 and its potential to be a novel best-in-class treatment option for patients with food allergy. Omalizumab’s rapid uptake in food allergy since its approval earlier this year confirms the high unmet need and large opportunity in this growing market,” commented Brian Wong M.D., Ph.D., President and CEO of RAPT. “RPT904 targets the same clinically validated epitope as omalizumab and combined with extended half-life, gives this molecule a best-in-class profile. We plan to initiate a Phase 2b clinical trial of RPT904 in food allergy in the second half of 2025.” https://lnkd.in/e92YkAmT

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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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MCO-010 Phase 2b Data Supports BLA Submission for Retinitis Pigmentosa The Phase 2b RESTORE trial (NCT04945772) evaluating MCO-010 (sonpiretigene isteparvovec; Nanoscope Therapeutics Inc. Therapeutics) in patients with retinitis pigmentosa (RP) met its primary and secondary endpoints, significantly improving best-corrected visual acuity (BCVA) with no serious adverse events (AEs).1 “We observed significant vision restoration in many patients with severe vision loss, including those who were completely blind,” investigator David Boyer, MD, Adjunct Clinical Professor of Ophthalmology, Keck School of Medicine, University of Southern California, said in a statement.1 “Many patients treated with MCO-010 derived a clinically meaningful benefit measurable on the primary visual function test, and this effect was confirmed by a parallel improvement in functional vision assessments. If approved, MCO-010 is poised to make a positive, meaningful impact on the lives of patients affected by this debilitating condition.” Nanoscope plans to submit a Biologics License Application (BLA) to FDA in the second half of 2024. The company previously received positive feedback from the FDA that change from baseline in a measure of visual acuity in low vision patients could be an appropriate primary efficacy assessment in an adequate and well-controlled study to support a BLA approval.2 Nanoscope announced the feedback in January along with positive feedback from the Icelandic Medicines Agency similarly endorsing the endpoint. "Consistent input from both the FDA and IMA on the potential for visual acuity to serve as the primary endpoint for the clinical program is a major milestone for Nanoscope and provides MCO-010 with a clear regulatory path in the US and in Europe," Sulagna Bhattacharya, Co-founder, and CEO of Nanoscope, said in a statement at that time.2

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While we still collect presentation details, it's exciting to have the expansive Phacilitate Advanced Therapies Week agenda up for the January 2025 meeting. We're excited to hit the ground running in 2025 and showcase the technical prowess and novel programs in cell and gene therapies. Don't miss out on our: - 🌟 Advanced Therapies Investment Summit, being built by Serena Mirpuri - 🌟 Women in Advanced Therapies Congress, led by Becky Johnson-Kent & Serena Mirpuri - 🌟 Advanced Therapies Awards 6 core tracks, including: - 🌟 Therapeutic Advances - 🌟 Commercializing Future Therapies - 🌟 Gene Therapy Manufacturing - 🌟 Cell Therapy Manufacturing - 🌟 CGT Supply Chain - 🌟 Patient Considerations and Advocacy Not to mention our stellar Innovation Showcase, placing early-stage companies in front of potential investors and partners (Reach out to Cameron Binns if you want to join!) There are still sponsor opportunities across the agenda and show floor, so reach out to David Green, Ayaan Lowry & Becky Johnson-Kent for opportunities (including our new 30-minute briefing format!) If you are a speaker/panelist or sponsor and need any modifications to your agenda segment, reach out directly to myself (Ry Leahy) and Serena Mirpuri! I'm very excited to see you in Dallas this January, and look forward to our conversations about the future of our exciting field. #ATW25 #AdvancedTherapiesWeek #Phacilitate #AdvancedTherapies #Cellandgenetherapy #celltherapy #genetherapy #regenerativemedicine #biotech #biotechnology #lifesciences #genetics

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A few words about the trends and challenges we see in Biotech nowadays, & how we propose to support innovation.

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Interested in seeing what Rocky Balboa, Jeff Spicoli, AI and machine learning have in common, check out this Timmerman Report article on east Coast vs West Coast AI culture clash. We are fortunate to have a network of experienced AI & machine learning professionals available for consulting assignment. Let us know if we can be of help.

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Elevation Oncology Partners with Synaffix BV in $368M Deal to Advance HER3 ADC Development Synaffix announces that it has entered into a licensing agreement with Elevation Oncology to develop EO-1022, a HER3-targeting Antibody-Drug Conjugate (ADC) leveraging Synaffix’s advanced platform technologies. This collaboration aims to create a highly effective and safer therapy for HER3-positive solid tumors. With up to $368 million in milestone payments, this partnership highlights the transformative potential of ADCs in precision oncology and patient care. Cutting-Edge Technology Integration Synaffix provides Elevation Oncology with global access to its clinical-stage, site-specific ADC technology platform, including GlycoConnect® antibody conjugation, HydraSpace® polar spacer, and the toxSYN® linker-payload SYNstatin E™. These advanced technologies enable the creation of a differentiated HER3 ADC candidate with enhanced efficacy and tolerability, holding the potential to set a new benchmark in oncology therapeutics. Strategic and Financial Framework Under the agreement terms, Synaffix will receive up to $368 million, contingent on achieving specific clinical, regulatory, and commercial milestones. Additionally, Elevation Oncology will handle research, development, and commercialization, while Synaffix will manage manufacturing components tied to its proprietary platform. https://lnkd.in/em89DTzB

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Aviceda Therapeutics Raises Upsized $207.5 Million in Series C Financing Round to Advance Lead Program AVD-104 for Geographic Atrophy into Pivotal Trials - Business Wire CAMBRIDGE, Mass.--(BUSINESS WIRE)--Aviceda Therapeutics (“Aviceda”), a private, clinical-stage biotech company focused on developing next-generation immunomodulators with a proprietary High Affinity Ligands of Siglecs (HALOS™) nanotechnology platform to alleviate chronic, non-resolving inflammation, today announced the closing of a $207.5 million Series C financing. Proceeds will support the ongoing Phase 2b/3 and planned pivotal Phase 3 clinical trial for AVD-104 in geographic atrophy (GA), a vision-threatening condition secondary to advanced age-related macular degeneration, and the expansion into other ophthalmic indications. The Series C round was co-led by renowned healthcare funds, Omega Funds and TCGX, with participation from leading investment firms Enavate Sciences, Jeito Capital, Blue Owl Healthcare Opportunities, Longitude Capital, OrbiMed, Logos Capital, Marshall Wace, Catalio Capital Management, funds managed by abrdn Inc., and Digitalis Ventures. “We are proud to support Aviceda and its team of retinal specialists, whose blend of ophthalmic expertise and operational excellence drives Aviceda’s advancement of AVD-104 toward critical milestones,” commented Bernard Davitian, Partner at Omega Funds. AVD-104’s dual mechanism of action targets immune modulation and complement inhibition. By repolarizing overactivated macrophages to their healing stage and stabilizing complement factor H, AVD-104 has shown positive proof-of-concept efficacy in slowing GA lesion growth and preserving/enhancing visual function in a Phase 2a trial completed in early 2024, which underscores its potential for first-in-class efficacy and safety. Aviceda’s ongoing Phase 2b/3 trial evaluating two doses of AVD-104 versus avacincaptad pegol (Izervay) in patients with GA is fully enrolled, with 12-month primary endpoint data anticipated in the second half of 2025. https://lnkd.in/eAy_ChMb

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OMG you would think I would LOVE an announcement that Illumina and NVIDIA were working together but honestly this sounds like this morning's second case of Illumina having to give up a lot to get a little. Deal has two parts: (1) Illumina's "get" is to host a bunch of Nvidia tools for LLMs and data analytics in on the Illumina Connected Analytics platform. Also MONAI for spatial cell imaging workflows though not sure what they have to do with NGS. Anyway, this seems like a nice-to-have, maybe a convenience to customers - but I don't think it opens up any new functionality. Doesn't cost Nvidia anything, and they get to add another logo on their partners slide 🤷‍♂️ (2) Illumina (this is the "give" part) will be "enabling DRAGEN algorithms on NVIDIA GPUs". Not sure why this is good for Illumina, and the PR doesn't explain this benefit at all 🤯 DRAGEN is built on a hardware architecture - FPGA - that has nothing to do with GPU at all, in fact it's kind of the opposite. So Illumina is going to have to make a major investment in porting DRAGEN to Nividia GPU for no stated benefit to Illumina. But the benefit to Nvidia is clear: they get to claim yet another customer for their already vast GPU customer base, and they get to big to displace FPGAs in Illumina instruments. Must be missing something here 🤔🤔🤔 (Don't forget to check out our JMP podcast! https://lnkd.in/gU_qpY3S)

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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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🎉 Huge congratulations to Orum Therapeutics for their deal yesterday! Vertex bolts on Orum's Degrader-Antibody Conjugate(DAC) conditioning agents to ImmunoGen's ADC conditioner, acquired in March 2023. But what drives Vertex to explore novel conditioner candidates?   Traditional conditioning agents are poorly tolerated and have posed significant challenges for Hematopoietic Stem Cell (#HSC) Cell and Gene Therapies (CGT), such as Vertex's Casgevy. These hurdles are now being addressed by novel, less aggressive conditioners, as above. For instance, companies like Actinium Pharmaceuticals are also advancing novel conditioning agents in this space. Thus, improved conditioning agents will play a crucial role in expanding the application of HSC CGT.   Another critical challenge in HSC CGT is effective #mobilization. Emerging CGTs often require a higher yield of HSCs than conventional HSC transplantation in cancer. When it comes to in vivo approaches, having more HSCs available in peripheral blood will be critical since ex vivo collection and engineering are not feasible. Thus, there is a growing demand for enhanced HSC mobilizers in this evolving field.   Here comes #GPC201, a potential best/fastest-in-class HSC mobilizer currently in phase 2 clinical trials in the US. It is demonstrating superior features for HSC transplantation in multiple myeloma. As the universe of various CGT pipelines expands rapidly, GPC201 is poised to showcase its full potential.   I eagerly anticipate the transformative impact of pairing GPC201 with novel conditioning agents to enhance patients' HSC transplantation experiences.   Curious about GPC201? 👉👉👉 GPCR Therapeutics

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By leveraging AI with vast genetic databases, we are not only quickening the pace of identifying potential drug targets but also enhancing the accuracy of clinical diagnostics and predictive modeling. Recently, I had the privilege of discussing the profound impact AI is having on pharmaceutical R&D with Kim Branson, GSK's SVP Global Head of Artificial Intelligence and Machine Learning. Listen on Raising Health: https://lnkd.in/g6QUm2-A

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Tempus is leveraging the power of multimodal data to introduce the next generation of immunotherapy biomarkers into the clinic. In July, we announced a multimodal algorithm called IPS (immune profile score) that uses DNA+RNA to help inform who may or may not respond to immunotherapy. The test is available today for research use by life science companies, and can help accelerate their work in several meaningful ways: 1. Label Expansion: IPS can be used to identify study participants who may have favorable clinical outcomes when treated with ICI outside of your FDA-approved biomarker labels. 2. Novel ICI Combinations: Use IPS to identify study participants who may have less favorable outcomes when treated with standard ICI and may be candidates for more intensive novel combination therapies. 3. Trial Design: With IPS, partners can refine study participant selection and stratification in clinical trials, enabling more targeted and effective study designs. All made possible by Tempus’ AI-enabled platform.

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This article is inspired by comments from Tony Czarnik and Stefan Ivanov regarding my previous blog post at https://lnkd.in/gFPhXfKW. To clarify, I am writing from the perspective of an AI believer or practitioner, acknowledging the critical viewpoint that claims "AI has shown little in terms of actual achievements in drug discovery." However, I believe it is not sufficient to simply agree with this stance without further elaboration. My focus here is on higher-level concepts rather than practical examples. Here are three key takeaways: 1. Need for Comprehensive Data Collection The current drug discovery process often lacks systematic documentation and relies heavily on subjective reporting, which hampers the ability to draw robust conclusions. Scientists, particularly chemists and biologists, should prioritize automating the collection of extensive data and metadata to create a rich database for analysis. 2. Integration of Mathematical and Data-Driven Approaches Two primary strategies for improving drug discovery are mathematical modeling and data-driven methods. While traditional scientific practices lean toward mathematical modeling for deeper understanding, advancements in AI highlight the effectiveness of data-driven approaches in addressing existing challenges and providing new insights into complex biological processes. 3. Transformative Role of AI AI technology plays a crucial role in drug discovery by enabling scientists to embrace data-driven methodologies that enhance problem-solving capabilities and deepen understanding of biological systems. Combining both mathematical models and data-driven techniques can lead to a more robust framework for innovation in the pharmaceutical industry.

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Over the weekend I was thinking (as one does 🤪) about Illumina moats. A clear sign that they're less effective than they've been historically is the traction that Element Biosciences has in the benchtop space, and MGI's success in overcoming Illumina's long held IP moat. I've heard that concern aboutthis problem drove Illumina's focus on vertically integrating via the GRAIL acquisition; I think it's fair to say that didn't work both because of regulators and it wasn't a real thing. So what's a monopoly to do? Obviously try and catch next the week wave - in my opinion that's proteomics sequencing - but that's going to take a lot of time and investment and technology risk. What can be done right now to shore up the moat as it's at immediate risk? One idea would be to license its core SBS technology to other life science companies. Some of the pros and cons: PROS: - Lets OtherCo's do the vertical integration. A great example of this is what Thermo Fisher Scientific has done with Genexus - built a clinical grade sample to answer system around Ion Torrent technology. Illumina has nothing like this because it doesn't have the extraordinary breadth of IVD experience and technologies that Thermo brought to the table. Why not license SBS to companies with these capabilities such as Danaher Corporation, QIAGEN and Roche? - Flexible high margin business model. Sure at one end of the licensing spectrum is royalties with 100% margin but low revenue, but there also the option of selling consumables. The seed for that model can be seen in the new MiSeq: all the complexity/IP is in the consumable (CMOS, fluidics, etc), very little in the instrument. Perfect for partnerships - you guys build the razors and we'll build the razorblades. - Licensing creates amazing moats as it turns potential large-company competitors into customers. It's a win-win for those customers: they get access to a constantly improving low risk NGS technology while being able to focus on their true value added, which is in non-NGS things. - I'm guessing this wouldn't be a problem for regulators - they would see it as a good thing as Illumina is sharing it's IP with others which feels anti-monopolist. CONS: - Potential for cannibalizing Illumina sales with licensee sales. This requires careful choice of licensees and limiting their field of use e.g. licensing SBS to Qiagen to only build a clinical sample to answer. And maybe picking winners here would be an anti-trust issue? Dunno. - Leakage of IP to licensees - Other? Struggling a little for a business analogy there. Kind of like Arm: a true platform with a great moat and scalable business model; customers for sure build vertically integrated solutions around their IP. Any other pro/con suggestions or business analogies?

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I think we're seeing a whole new Illumina strategy for defending the mid-market from Element Biosciences competition. I for one was thinking too narrowly to see this - figured it was all about Aviti vs NextSeq. But I think we're starting to see a clever three-pronged Illumina strategy: (1) Times are hard, why buy a mid instrument for $400K when you can just skip that and send your samples out to someone with an X? (2) High throughput is where it's at, here's a cheap NovaSeq X, why not stretch a little for that and be able to hang with the cool kids? (3) Have you seen our new MiSeq i100? Here take this Lego one. It's just $100K, you can most of your stuff with that, and just send out for the big stuff. Sure, kills NextSeq sales too, but that's easy pain for Illumina to bear, but existential for Element. Here's Illumina CFO Ankur Dhingra spelling it out for Wolfe's Doug Schenkel: "...customers, because of the macro [economic/grant] issues, have been transitioning their sample volume to high throughput. And a lot of our mid throughput customers who are constrained by that macro environment are resorting to sending their samples to either core labs or other sequencing providers." "...the constrained environment is making people take decisions on what are they going to invest in their lab or until money frees up, they're going to take advantage of the lower high throughput [X-based sequencing as a service] pricing that is available externally." "I do think there are additional two factors that are coming our way from an Illumina perspective. We have said we will be launching a slightly lower throughput version of NovaSeq X with a single flow cell. And that is at a price point which is well below 1 million. So, there is a potential that some of our mid throughput customers may see some value there. And also, the higher end of the new MiSeq i100 that we have launched has capabilities that can seep into some of the mid throughput kind of requirements as well. So, it's [Element?😳] going to get squeezed from a little bit from both the directions as well."

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Let me add an informal ‘bet’ - measuring ~9000 proteins on the SOMAmer-based platform in these 50k samples will yield more actionable new discoveries (biomarkers for stratification, pQTLs for drug targets) than measuring far fewer proteins (and very few new proteins*) in 300-600k samples in the parallel PPP study. Any takers? * See https://lnkd.in/dHub6w-D

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