Chardan

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 [email protected].?

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

Avidity Biosciences, Inc., a Chardan 2024 top pick (published Feb 2, 2024), announced two new lead candidates for cardiomyopathies using the Company's antibody oligonucleotide conjugate (AOC) technology. The programs announced on Tuesday are wholly-owned, and separate from those being developed under its collaboration with Bristol Myers Squibb (BMY, unrated) for up to 5 AOC therapeutic candidates targeting cardiac tissue. Director of Research, Keay Nakae, CFA maintains a BUY rating and 12-month PT of $65. Clients can access the report here: https://lnkd.in/d3mBiTR or by reaching out to your Chardan sales contact. #ChardanResearch #RNA

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 6 November, as part of its quarterly update, Sarepta Therapeutics (unrated) announced it will discontinue development of its next-generation exon skipping PPMO SRP-5051 for Duchenne muscular dystrophy (DMD), as well as all other PPMOs in its pipeline. The company stated that this decision was based on several factors, including the risk-benefit profile of the program, feedback from the FDA on the non-availability of the accelerated approval pathway based on the current profile to-date, and the evolving landscape for DMD including the approval of Sarepta's own AAV gene therapy Elevidys. 2) On 8 November, Autolus Therapeutics (unrated) announced that it has received FDA approval of its CAR-T therapy Aucatzyl (obe-cel) for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL). Aucatzyl is an autologous CD19-directed CAR-T therapy with a novel CAR designed to display improved binding kinetics compared to standard CARs. Approval followed the PIb/II FELIX trial, in which 42% of patients (27/65) with r/r B-ALL achieved CR within 3 months. 3) Abstracts for the American Society of Hematology Annual Meeting & Exposition (ASH), which will take place on 7-10 December in San Diego, CA, were released this week, with many genetic medicines companies announcing presentations. Additionally, the Society for Immunotherapy of Cancer Meeting took place, with several companies in the space presenting on cell therapy, viral immunotherapy, vaccines and other genetic medicines strategies in oncology. 4) Beam Therapeutics (unrated) released preliminary data for a subset of patients from its phase I/II study of its ex vivo base editing therapy BEAM-101 in sickle cell disease (SCD). The data showed that all 4 efficacy-evaluable patients achieved induction of HbF within 1 month, which was sustained for all time points measured (with 2 patients reaching >60% HbF at 5-6 months). The safety profile was largely consistent with other treatments requiring myeloablative conditioning with busulfan. Notably, one patient died due to respiratory failure resulting from the busulfan conditioning (deemed unrelated to BEAM-101 treatment itself). Beam will release additional data at ASH next month. To learn more about Chardan research, [email protected]. #geneticmedicines

Chardan is pleased to have acted as a Financial Advisor on Ocugen’s credit facility. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. Their breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and they are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. To learn more about this transaction, reach out to [email protected]. #ChardanTransactions?

Chardan’s Viral News in Genetic Medicines is a weekly piece detailing key news and industry research. In this week’s industry note: 1) Lexeo Therapeutics (Buy) presented at the CTAD2024 conference interim results from the phase I/II open label trial of its Alzheimer’s disease (AD) gene therapy program, LX1001, in AD patients homozygous for the high-risk APOE4 allele. The data provide initial clinical proof of concept in terms of safety, APOE2 expression, which was detected in all cohorts at 6 or 12 months, and reductions in AD-associated CSF tau biomarkers and tau PET in a majority of patients. However, we have yet to see signs that the magnitude of effects here can drive cognitive benefit in treated patients. As disclosed previously, the company hopes to advance LX1001 with a partner given the potentially large pivotal study required to advance the program in addition to the significant capital that would likely be required for commercializing LX1001. The company has initiated engagement with the FDA and expects to provide an update on the regulatory interactions and further LX1001 development plans in 2025. 2) Alnylam Pharmaceuticals (Buy) reported initial interim multi-dose data from its phase I study of mivelsiran (ALN-APP), an RNAi-based therapy for early-onset Alzheimer's disease and presented data from the program at CTAD2024. Prior single dose data shown for a 50 mg dose on day 0, mean reductions of approximately 75% in sAPPβ observed at month one, with sustained reduction of approximately 50% still apparent at month 6 and a slow return towards baseline thereafter. Patients administered 50 mg doses at month 0 and 6, saw a similar trajectory in sAPPβ during the first 6 months, and then saw further reductions in sAPPβ at month 7 (1 month after the 2nd dose) achieving about 90% lowering of sAPPβ. The company plans to initiate a phase II trial for mivelsiran by the end of 2024 . 3) Pfizer (unrated) announced 3Q24 earnings and raised its FY24 revenue guidance to a range of $61.0 to $64.0 bn (up from $59.5 to $62.5bn from 2Q24). The company expects to receive $5 bn in revenue from its Comirnaty Covid-19 vaccine. 3Q revenue for Comirnaty were $1.4bn, up $119mm compared to last year. 4) BioMarin Pharmaceutical Inc. Pharmaceutical (unrated) announced 3Q24 earnings, reporting $746 mm in total revenue (+28% YoY), with Roctavian, its AAV5-based gene therapy to deliver a functional Factor VIII to patients with hemophilia A, accounting for only $7 mm in net product revenue. To learn more about Chardan research, [email protected]. #geneticmedicines

Chardan’s recent panel “Getting to the Heart of the Matter: Genetic Medicines for Cardiac Diseases” highlighted that advances in key enabling technologies and a greater understanding of heart disease biology through genomics approaches are converging to drive the advancement of genetic medicines for heart disease. Panelists Faraz Ali (Tenaya Therapeutics), Yann Chong Tan, PhD (Nuevocor), and Nolan Townsend (Lexeo Therapeutics) discussed the scientific, clinical, and commercial outlook for genetic medicines in the cardiac disease space. To view the replay, please reach out to [email protected]. #geneticmedicines #celltherapy #geneediting #cardiacdisease?

At Chardan’s 8th Annual Genetic Medicines Conference, Peter Anastasiou (Capsida Biotherapeutics), Eric Kelsic, PhD (Dyno Therapeutics), Joe La Barge (Apertura Gene Therapy), and Rick Modi (Affinia Therapeutics) discussed the latest advancements in next-gen AAVs. For a replay, please reach out to [email protected]. #geneticmedicines #genetherapy #geneediting #celltherapy?

On September 30 and October 1, Chardan hosted its 8th Annual Genetic Medicines Conference (CGMC 2024). CGMC 2024 featured 80 public and private companies representing in vivo gene therapy, ex vivo gene therapy, gene editing, RNA medicines, and other sub-segments of the genetic medicines space, as well as various panels with preeminent thought leaders. Featured on the panel “Next-Gen Delivery Strategies”, Dave Anderson, PhD (Code Biotherapeutics), David Lockhart, PhD (ReCode Therapeutics), and Joel Schneider, PhD (Carbon Biosciences) discuss the development of next-gen delivery of genetic medicines. To access the replays, please reach out to [email protected]. #geneticmedicines #celltherapy #genetherapy #geneediting

Chardan’s Genetic Medicines Conference is one week away! For more information, [email protected]. #geneticmedicines #celltherapy #ChardanGMConf