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Chardan’s Viral News in Genetic Medicines is a weekly piece detailing key news and industry research. In this week’s industry note: 1) Sangamo Therapeutics (unrated) announced Pfizer (unrated) has elected to terminate the companies' collaboration for giroctocogene fitelparvovec (giro-vec), an AAV6-based gene therapy for hemophilia A. Pfizer had announced positive phase III data for giro-vec in July and was expected to proceed with BLA and MAA submissions in early 2025. Sangamo will now regain development and commercialization rights and may seek a new partner; however, the therapy faces an uncertain future. Pfizer's decision follows that of Roche (unrated), which removed its own Spark Therapeutics-developed hemophilia A gene therapy from its pipeline in December and withdrew its clinical trial, with both companies prioritizing other modalities in the indication. BioMarin Pharmaceutical's (unrated) Roctavian, the only gene therapy currently approved for hemophilia A, has continued to struggle since its July 2023 FDA approval; while Roche's FVIII-mimicking antibody Hemlibra has continued to thrive. In addition, 2 anti-TFPI antibodies were approved over 2024 for both hemophilia A and B: Pfizer's Hympavzi in October, and Novo Nordisk's (unrated) Alhemo in December. SGMO stock fell 56% following the announcement. 2) Novartis (unrated) announced topline results from the phase III STEER study of OAV101IT, the company's intrathecal formulation of Zolgensma, in patients ages 2 to less than 18 with spinal muscular atrophy (SMA) Type 2. The study met its primary endpoint of an increase from baseline in total HFMSE scores for OAV101IT-treated patients compared to sham controls. The therapy also demonstrated a favorable safety profile. Novartis will provide detailed data at a medical meeting in 2025. Zolgensma was approved in 2019 for IV infusion in SMA patients under 2 years old; however, older patients do not currently have a gene therapy option available to them and instead manage their disease with drugs such as Biogen's (unrated) ASO splicing modifier Spinraza or Roche's small molecule splicing modifier Evrysdi, as well as supportive care. To learn more about Chardan research, info@chardan.com. #geneticmedicines

Chardan Senior Research Analyst, Rudy Li, PhD, has initiated coverage on three names in the CNS space. 1) MindMed (MNMD) he initiated coverage on with a Buy rating and a $20 PT on the potential of its lead asset MM120 (LSD) for the treatment of generalized anxiety disorder (GAD) and major depressive disorder (MDD). 2) Prothena Corporation plc (PRTA) he initiated coverage with a Buy rating and a $40 PT as a diversified play for proteinpathies. 3) Stoke Therapeutics, Inc. (STOK) he initiated coverage with Buy rating and a $24 PT on the potential of its lead asset zorevunersen/STK-001 (ASO) for the treatment of Dravet syndrome. Clients can access reports here: https://lnkd.in/e5JU2fRg or by reaching out to your Chardan sales contact. #ChardanResearch

Chardan is pleased to have acted as Co-Manager on CleanSpark’s convertible bond offering . Cleanspark, America's Bitcoin Miner®, is a market-leading, pure play Bitcoin miner with a proven track record of success. They own and operate a portfolio of mining facilities across the United States powered by globally competitive energy prices. Sitting at the intersection of Bitcoin, energy, operational excellence and capital stewardship, they optimize their mining facilities to deliver superior returns to their shareholders. Monetizing low-cost, high reliability energy by securing the most important finite, global asset – Bitcoin – positions Cleanspark to prosper in an ever-changing world. To read more about this transaction: https://lnkd.in/eC5b2Skt or reach out to info@chardan.com #ChardanTransactions #crypto 

Chardan is pleased to have acted as Sole Placement Agent on Sonnet BioTherapeutics Registered Direct Offering and Concurrent PIPE. Sonnet is an oncology-focused biotechnology company with a proprietary platform for innovating biologic drugs of single or bifunctional action. Known as FHAB (Fully Human Albumin Binding), the technology utilizes a fully human single chain antibody fragment (scFv) that binds to and "hitch-hikes" on human serum albumin (HSA) for transport to target tissues. Sonnet's FHAB was designed to specifically target tumor and lymphatic tissue, with an improved therapeutic window for optimizing the safety and efficacy of immune modulating biologic drugs. FHAB is the foundation of a modular, plug-and-play construct for potentiating a range of large molecule therapeutic classes, including cytokines, peptides, antibodies, and vaccines. To read more about this transaction: https://lnkd.in/etDrgB7N or reach out to info@chardan.com  

Chardan is pleased to have acted as Sole Placement Agent on Eyenovia, Inc.’s Registered Direct Offering. Eyenovia, Inc. is an ophthalmic technology company developing and commercializing advanced products leveraging its proprietary Optejet topical ophthalmic medication dispensing platform. The Optejet is especially useful in chronic front-of-the-eye diseases due to its ease of use, enhanced safety and tolerability, and potential for superior compliance versus standard eye drops. Together, these benefits may combine to produce better treatment options and outcomes for patients and providers. The company’s current commercial portfolio includes clobetasol propionate ophthalmic suspension, 0.05%, for post-surgical pain and inflammation, and Mydcombi® for mydriasis.  Read more about the transaction here: https://lnkd.in/ex-kH4FE or reach out to info@chardan.com #ChardanTransactions #ophthalmology

Congratulations to ACE Green Recycling, Inc. on their announced merger with Athena Technology Acquisition Corp II. Chardan is pleased to have acted as exclusive financial advisor to Ace Green Recycling on this transaction. Ace is advancing electrification by building a global recycling technology to create sustainable supply chain solutions for critical metals that will enable next-generation technologies. Read more about the transactions here: https://lnkd.in/ezjCeEcW or by reaching out to info@chardan.com. 

Chardan’s Viral News in Genetic Medicines is a weekly piece detailing key news and industry research. In this week’s industry note: 1) On 26 November, Sarepta Therapeutics (unrated) announced a global licensing and collaboration agreement with Arrowhead Pharmaceuticals (Buy) for multiple preclinical and clinical stage siRNA programs. Per the deal, Arrowhead will receive an upfront payment of $500 mm, an equity investment of $325 mm, annual payments of $50 mm for 5 years for a total of $250 mm, and potential future milestone payments and royalties. The agreement covers 4 clinical programs: ARO-DUX4 for FSHD, ARO-DM1 for DM1, ARO-MMP7 for IPF, and ARO-ATXN2 for SCA2, and 3 preclinical programs that leverage Arrowhead's TRiM CNS delivery platform: ARO-ATXN1 for SCA1, ARO-ATXN3 for SCA3 and ARO-HTT for Huntington's disease. In addition, the companies have entered into a discovery collaboration for 6 muscle, cardiac and/or CNS targets. 2) On 26 November, Poseida Therapeutics, Inc. Therapeutics (unrated) announced its planned acquisition by Roche Holdings (unrated) for a total equity value of up to ~$1.5 bn. Roche will purchase Poseida shares at $9 per share in cash, plus a non-tradeable CVR of up to $4.00 per share upon the achievement of "specific milestones". The acquisition will include Poseida's full set of non-viral capabilities in designing and manufacturing allogeneic T stem cell memory cell rich CAR-T therapies upon closing of the transaction, which is expected to be completed in 1H25. The acquisition builds upon the companies' 2022 collaboration, which is centered on allogeneic CAR-Ts for blood cancers and has included the development of P-BCMA-ALLO1 in multiple myeloma. To learn more about Chardan research, info@chardan.com. #geneticmedicines  

Chardan’s Viral News in Genetic Medicines is a weekly piece detailing key news and industry research. In this week’s industry note: 1)REGENXBIO (Buy) reported first functional data from the phase I/II AFFINITY DUCHENNE trial for AAV8 gene therapy (GT) RGX-202 in pts with Duchenne muscular dystrophy (DMD). The data revealed positive functional outcomes for the first 5 pts, across 2 dose levels, with all 5 pts showing stable or improved function relative to natural history controls on the NSAA and timed functional tests. The company has initiated a pivotal trial for RGX-202 and dosed its 1st patient. The pivotal trial will have a primary endpoint of proportion of patients with ≥10% microdystrophin levels at 12 weeks. Recall, microdystrophin expression was used as the basis for accelerated approval of Sarepta's (unrated) DMD GT Elevidys. 2) Novartis (unrated) acquired Kate Therapeutics(pvt), a preclinical company developing AAV GTs for DMD, FSHD and DM1 leveraging novel liver de[1]targeted capsids. Novartis will pay up to $1.1bn, comprising an upfront cash payment and potential additional milestone payments. The acquisition further signals NVS's expansion of its GT work, building on Zolgensma and its CNS capsid deal with unrated Voyager.3) Neurogene (unrated) announced it will no longer enroll pts in the high dose cohort (3E15 vg) of the phase I/II clinical trial of its AAV9 GT NGN-401 for Rett syndrome. This update followed the assessment of a treatment-related SAE in this cohort and the subsequent death of the patient. The FDA has allowed the company to proceed with the low dose (1E15 vg), though the company no longer anticipates completing enrollment by 4Q24. 4) Arbutus Biopharma Corporation (Buy) presented follow-up and end-of-treatment data from the IMPROVE I and IM-PROVE II phase IIa clinical trials on imdusiran (AB-729-201) at AASLD. The IMPROVE I open label trial randomized subjects into 4 dosing regimens. Functional cures were demonstrated in 3/12, 2/13 and 1/10 pts in Cohorts A1, A2 and B2 respectively. The study also reported corresponding decreases in HBsAb, HBV DNA and ALT in these pts. 5) Silence Therapeutics plc (Buy) presented data from the ALPACAR-360 phase II study of zerlasiran in ASCVD pts with high Lp(a) at AHA. Zerlasiran produced >80% mean time-average placebo-adjusted reductions in Lp(a) from baseline during 36 weeks of follow-up. Notably, this end point was different from prior trials of nucleic acid–based therapies, which all used either maximal reduction in Lp(a) or lowering at a specific time point. Lp(a) reductions persisted at 60 weeks post initial administration, with largest median reduction of 58.8% seen in the 300mg every 16 week cohort. 6) The American College of Rheumatology Convergence 2024 included several genetic medicines companies sharing preclinical and clinical updates on CAR-T cell therapies for autoimmune indications. To learn more about Chardan research, info@chardan.com. #geneticmedicines

Chardan is pleased to have acted as a Co-Placement Agent on Forte Biosciences, Inc.'s PIPE. Forte Biosciences, Inc. is a clinical-stage biopharmaceutical company that is advancing FB102, which is a proprietary anti-CD122 monoclonal antibody therapeutic candidate with potentially broad autoimmune and autoimmune-related indications. To learn more about this transaction, reach out to info@chardan.com. 

Chardan senior research analyst, Keay Nakae, CFA is quoted in Investor’s Business Daily article “Avidity Biosciences, Inc. Has Surged 416% This Year – And That’s Before Today’s ‘Game-Changing’ News”. Keay comments on Avidity’s plans to test out new treatments for two rare diseases, PLN cardiomyopathy and PRKAG2 syndrome. To read more: https://lnkd.in/e_8iRCMC