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

Chardan has initiated coverage on CervoMed with a Buy rating and 12 month price target of $55 based on the potential of the company's lead asset neflamapimod targeting Dementia with Lewy Bodies (DLB). CervoMed Inc. is a clinical-stage company focused on developing treatments for age-related neurologic disorders. The company is currently developing neflamapimod, an investigational, orally administered small molecule brain penetrant that inhibits p38 mitogen-activated protein kinase alpha. Neflamapimod has the potential to treat synaptic dysfunction, the reversible aspect of the underlying neurodegenerative processes that causes disease in DLB and certain other major neurological disorders. Neflamapimod is currently being evaluated in a Phase 2b study in patients with early-stage DLB. Clients can access the report here: https://lnkd.in/d3mBiTR or by reaching out to your Chardan sales contact. #ChardanResearch

Chardan biotech analyst, Daniil Gataulin, PhD hosted the 2nd annual Virtual Ophthalmology Summit focusing on AMD and Diabetes Related Ocular Disease. Participating companies included: Adverum Biotechnologies, Clearside Biomedical, Inc., EyePoint Pharmaceuticals, Ocugen, Oculis, Outlook Therapeutics, Inc., REGENXBIO Inc., UNITY Biotechnology. View session replays here: https://lnkd.in/es4yjHrq or to read the published takeaways, please reach out to [email protected]. #ChardanResearch #Ophthalmology #biotech

Chardan’s Viral News in Genetic Medicines is a weekly piece detailing key news and industry research. In this week’s industry note: 1) Arrowhead Pharmaceuticals (Buy) presented data from the phase III PALISADE study evaluating RNAi tx plozasiran in patients with familial chylomicronemia syndrome (FCS) at the European Society of Cardiology (ESC) Congress 2024. As previously disclosed, the study met the primary endpoint, change in fasting triglycerides at month 10 compared to baseline, and met all key secondary endpoints. At 10 months, the median change from baseline level in the fasting triglyceride level, declined ?80% in the 25-mg plozasiran group, and ?78% in the 50-mg plozasiran group, compared to ?17% in the placebo group. A key secondary endpoint showed that patients receiving plozasiran achieved an 83% reduction in the risk of developing acute pancreatitis versus placebo. The company expects to file NDA with FDA by year-end and will seek global approvals (see below and research for further commentary). 2) Dyne Therapeutics (Buy) reported additional data from the PI/II DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) patients with mutations in exon 51. The readout included 6-month biomarker and functional data from 8 pts in the 20 mg/kg cohort, and 12-month functional data from patients in the 10 mg/kg cohort. The 20 mg/kg dose demonstrated an increase in mean absolute dystrophin expression (unadjusted for muscle content: 3.71%; adjusted for muscle content: 8.72%), compared to the level observed for the 10 mg/kg dose (3.2% unadjusted, 7.6% adjusted). The company also noted treatment related serious adverse events from 40mg/kg dose. The company will provide further updates on registration process by end 2024. 3) Arsenal Biosciences, Inc. (pvt) announced series C financing of $325 mm from investors including Bristol Myers Squibb (unrated) for the advancement of T-cell engineering based therapeutic assets for solid tumors including ovarian, kidney, and prostate cancers. ArsenalBio had recently announced dosing of the1st patient in the PI/II trial of AB-2100, in renal cell carcinoma. AB-2100 leverages ArsenalBio's CRISPR Integration of Transgenes by Electroporation (CITE) technology to engineer T cells for targeting tumor cells while minimizing off-tumor toxicity, inserting a transgenic DNA cassette into a new safe harbor locus on Chromosome 11. The tumor selectivity is conferred by a logic gate that requires PSMA on tumor endothelium and carbonic anhydrase 9 (CA9) on adjacent tumor cells. Additionally, the entire tumor targeting complex includes shRNA to protect T-cells from TME immunosuppression and synthetic pathway activator (SPA) for enhanced functioning & durability of eng. T-cells. To learn more about Chardan research, [email protected]. #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) The FDA authorized and approved updated mRNA-based Covid-19 vaccines for the 2024-25 fall and winter season from Pfizer/BioNTech SE (unrated) and Moderna (Neutral). The updated vaccines are designed to target the KP.2 strain of the SARS-CoV-2 Omicron JN.1 lineage consistent with the agency's guidance. The vaccines are approved for individuals 12+ years and have EUA for 6 months through 11 years. Pfizer/BioNTech and Moderna both expect the updated vaccines to be made available across care settings in the US in the coming days after approval. 2) Moderna also announced European Commission (EC) approval of mRNA-1345 (mRESVIA), its mRNA-based RSV vaccine, for adults 60 yrs & older. The approval was based on results from PIII ConquerRSV trial demonstrating 83.7% efficacy at 3.7 months of median follow-up. mRESVIA had received approval from the FDA in May this year. Moderna separately received approval of a Covid vaccine targeting JN.1 from the Ministry of Health, Labour and Welfare (MHLW) Japan. 3) Borealis Biosciences, a private company focused on RNA based therapeutics, emerged from stealth with a total of $150 mm series A funding from Versant ventures and Novartis (unrated). In addition to the upfront payment, Novartis will also collaborate in research for xRNA based therapeutic innovation in renal diseases. Per the agreement, Novartis committed up to a $100 mm upfront payment and has an option to acquire two "development-ready" programs in future, for a total consideration of up to $750 mm to Borealis under clinical and regulatory milestone payments. To learn more about Chardan research, [email protected]. #geneticmedicines

Chardan is pleased to have acted as Co-Manager on Avidity Biosciences, Inc.'s follow on. Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs?). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through internal discovery efforts and key partnerships. To learn more about this transaction, reach out to [email protected] #ChardanTransactions

Chardan has initiated coverage on Clearside Biomedical, Inc. Biomedical with a Buy rating and 12 month price target of $6 based on the commercial potential of the company's lead CLS-AX program in wet AMD. Clearside Biomedical, Inc. is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS). Clearside’s SCS injection platform, utilizing the company’s patented SCS Microinjector, enables an in-office, repeatable, non-surgical procedure for the targeted and compartmentalized delivery of a wide variety of therapies to the macula, retina, or choroid to potentially preserve and improve vision in patients with sight-threatening eye diseases. Clearside is developing its own pipeline of small molecule product candidates for administration via its SCS Microinjector. The company’s lead program, CLS-AX (axitinib injectable suspension), for the treatment of neovascular age related macular degeneration (wet AMD), is in Phase 2b clinical testing. Clearside developed and gained approval for its first product, Xipere (triamcinolone acetonide injectable suspension) for suprachoroidal use, which is available in the U.S. through a commercial partner. Clearside also strategically partners its SCS injection platform with companies utilizing other ophthalmic therapeutic innovations. Clients can access the report here: https://lnkd.in/d3mBiTR or by reaching out to your Chardan sales contact. #ChardanResearch

Congratulation to our client iBio, Inc. on their ATM offering. Chardan is pleased to have served as the Sales Agent on this transaction. iBio is an AI-driven innovator that develops next-generation biopharmaceuticals using computational biology and 3D-modeling of subdominant and conformational epitopes, prospectively enabling the discovery of new antibody treatments for hard-to-target cancers, and other diseases. iBio’s mission is to decrease drug failures, shorten drug development timelines, and open up new frontiers against the most promising targets.?To learn more about Chardan’s investment banking practices and recent transactions, visit https://lnkd.in/g2p-a6kB. #Chardantransactions #healthcare #AI

Chardan’s Viral News in Genetic Medicines is a weekly piece detailing key news and industry research. In this week’s industry note: 1) Adaptimmune Therapeutics (unrated) announced receipt of FDA accelerated approval for its MAGE-A4 TCR engineered T cell therapy, Tecelra (afami-cel), for synovial sarcoma. The approval was based on the clinical results from its SPEARHEAD-1 (cohort 1) trial demonstrating overall response rate (ORR) of 43%, complete response rate of 4.5% and a median duration of response (DOR) of 6 months. 39% of patients who were responsive to the treatment had DOR of 12 months and over. The results were published in the journal Lancet in March this year. This is the 1st genetically engineered cell therapy approved for solid tumors, following the earlier approval of Iovance Biotherapeutics, Inc. (Buy) non-engineered TIL therapy lifileucel in melanoma. At an announced list price of $727,000, Tecelra is set to be the most expensive T cell therapy for cancer thus far. 2) BioNTech SE (unrated) announced the PII trial of mRNA immunotherapy BNT111 in combination with anti-PD-1 antibody Libtayo (cemiplimab) met its primary endpoint in unresectable post-anti-PD-(L)1 melanoma. Specifically, the topline readout indicated a stat sig improvement in ORR for patients treated with the BNT111 + cemiplimab combo compared to historical control. While the trial also includes monotherapy arms, details were not provided beyond disclosing that the cemiplimab arm ORR was "in line" with the historical control. BioNTech and unrated Regeneron plan to present these data at a medical conference and the PII trial will continue to allow secondary endpoints to mature. Recall, BNT111 is a fixed antigen cancer vaccine that uses BioNTech's uridine mRNA (uRNA) and lipoplex (LPX) delivery platforms to deliver 4 melanoma-associated antigens to trigger anti-cancer immunity (as part of its FixVac franchise), distinguishing the program from personalized cancer vaccine programs. 3) Pfizer (unrated) reported 2Q24 results and pipeline updates including discontinuation of its gene therapy for Duchenne muscular dystrophy (DMD). The move was not surprising as in June the company had announced failure of the therapy to meet primary and secondary endpoints in PIII trial in DMD patients of 4-7 yrs of age. 4) Passage Bio (Buy) announced out-licensing of its three gene therapies targeting pediatric lysosomal storage disease to private GEMMA Biotherapeutics formed by gene therapy pioneer Dr. Jim Wilson as part of a transition from the Penn Gene Therapy Program. Per agreement, GEMMABio gains exclusive worldwide rights to PBGM01 GM1 gangliosidosis, PBKR03 in Krabbe disease, and PBML04 in metachromatic leukodystrophy (MLD) for an initial aggregate payment to Passage Bio of $10mm for clinical product supply for the programs, a $10mm contingent payment and Passage Bio is eligible for $114mm in development and commercial milestones. To learn more about Chardan research, [email protected].

We hope to see you at Chardan’s 8th Annual Genetic Medicines Conference Sept 30 – Oct 1 in NYC! For more information, [email protected] #ChardanGMConf2024 #GeneticMedicines #CellTherapy?