Chardan

Chardan’s 8th Annual Genetic Medicines Conference is taking place September 30 – October 1, 2024 in New York City. Join us as we address major themes in genetic medicines through featured fireside chats, presentations, panel discussions, and 1x1 meetings. Attendees will hear from nearly 100 public and private companies and groundbreaking thought leaders. To learn more about the conference, please contact CorpAccess@Chardan.com #ChardanGMConf2024 #GeneticMedicines #CellTherapy 

Chardan’s Viral News in Genetic Medicines is a weekly piece detailing key news and industry research. In this week’s industry note: 1) Companies in the genetic medicines space presented updates at the 42nd ASRS Annual Scientific Meeting, notably, i) Adverum Biotechnologies (Buy) presented 26-week interim analysis of the PII LUNA trial of ixo-vec (GT) in wet AMD patients. The study demonstrated favorable safety profile with no or minimal inflammation in 100% of patients treated at 6e10 dose and no additional requirement of corticosteroids "beyond the scheduled prophylaxis". 76% patients remained injection free at 6e10 dose level which also demonstrated 90% reduction in mean annualized anti-VEGF injections. The mean change in best corrected visual acuity (BCVA) was -1.1 from baseline and fluid control was also maintained (-12.6 µm change in CST from baseline); ii) 4D Molecular Therapeutics (Buy) presented 24-week data from the less severe population extension cohort in the PII PRISM trial of intravitreal GT 4D-150 in wet AMD patients. The data demonstrated 89% reduction in annualized injection rate (with 77% of patients injection free) at the planned phase III dose (3E10 vg/eye); the BCVA was +3.5 ETDRS letters from baseline at weeks 20 & 24 for 3e10, showing some dose response vs. the 1e10 group (-2.2 letters). The 3e10 dose also showed improved anatomic control (-9.2 µm change in CST, vs +6.9 µm for the lower dose). FDMT's stock dropped 32% on the day of the news. 2) Vertex Pharmaceuticals (unrated) and Orum Therapeutics (pvt) announced a licensing deal to leverage Orum's dual-precision targeted protein degradation (TPD) technology derived degrader-antibody conjugates (DACs) as a conditioning agent for gene editing therapies. The deal entails a $15 mm upfront payment to Orum along with an eligibility for milestone payments of up to $310 mm per target for up to 3 targets, and additional sales royalties. Separately, Vertex has sued the U.S. Govt. for not allowing access to its fertility preservation program to the SCD/TDT pts treated with gene editing therapy Casgevy. (Current myeloablative conditioning agents such as busulfan carry a risk infertility.) Vertex intends to offer its fertility support and treatment services to the Casgevy eligible patients. While the program is available for pts on commercial insurance, those on govt. insurance cannot avail this service as the Health and Human Services (HHS)'s Office of the Inspector General (OIG) in its initial "oral" decision has stated the fertility program offering as a violation of federal anti-kickback law. Vertex has still not received a written statement in this regard. To learn more about Chardan research, info@chardan.com.  #geneticmedicines #genetherapy #celltherapy

Congratulations to our client, Catcha Group on successfully completing their $685M business combination with Crown LNG, a leading provider of offshore LNG liquefaction and regasification terminal infrastructure for harsh weather locations. Chardan is pleased to have served as the Exclusive Technical Capital Markets Advisor to Catcha. Catcha’s CFO, @Kit Wong: “Chardan provided invaluable assistance with detailed, flexible, and creative solutions at critical moments. They were reliable and consistently delivered on their promises. Throughout, they served as an excellent sounding board for advice.” To read more about this transaction: https://lnkd.in/eVPjuZwe. #ChardanTransactions

Chardan is recruiting for our 2025 banking summer internship program. Eligible candidates must have a graduation date of December 2025 – June 2026. For more information or to apply, careers@chardan.com.

Congratulations to Vyome Therapeutics, Inc. on their announced merger with ReShape Lifesciences Inc. Chardan is pleased to have acted as financial advisor to Vyome Therapeutics on this transaction. The combined company will focus on advancing the development of its immune-inflammatory assets and on identifying additional opportunities between the world class Indian innovation corridor and the U.S. market. To read more about this transaction: https://lnkd.in/e66B4TBV. To learn more about Chardan’s Investment Banking practice and recent transactions, please visit https://lnkd.in/g2p-a6kB. #ChardanTransactions

Chardan has initiated coverage on CARGO Therapeutics with a Buy rating and 12 month price target of $28 based on Phase II CAR-T Asset + Innovative Platform Tech. CARGO Therapeutics, Inc. is a clinical-stage biotechnology company, which engages in the development of cell therapies for cancer patients. It is involved in evaluating its lead program, CRG-022, an autologous CD22 chimeric antigen receptor T-cell therapy candidate, in a potentially pivotal Phase II clinical trial in patients with large B-cell lymphoma whose disease relapsed or was refractory to CD19 CAR T-cell therapy. The company was founded by Crystal Mackall, Nancy Goodman, Louai Labanieh, and Robbie Majzner in December 2019 and is headquartered in San Carlos, CA. Clients can access the report here: https://lnkd.in/d3mBiTR or by reaching out to your Chardan sales contact. #ChardanResearch

Chardan is pleased to have acted as Lead Placement Agent on Eyenovia, Inc.’s Registered Direct Offering. Eyenovia is an ophthalmic technology company commercializing Mydcombi™ (tropicamide and phenylephrine hydrochloride ophthalmic spray) 1%/2.5% for mydriasis, Clobetasol Propionate Ophthalmic Suspension, 0.05% for postsurgical inflammation and pain, and developing the Optejet® device for use both in connection with its own drug-device therapeutic product for pediatric progressive myopia as well as out-licensing for additional indications. Read more about the transaction here: https://lnkd.in/e9sfzZNW or by reaching out to info@chardan.com. #ChardanTransactions

Congratulations to our client ANEW MEDICAL, INC. on the completion of their business combination with Redwoods Acquisition Corp. ANEW Medical is a biopharmaceutical technology company focused on developing disruptive new therapies to treat the central nervous system and neurodegenerative diseases. Chardan is pleased to have acted as exclusive M&A and Capital Markets Advisor to ANEW Medical. Read more about the transaction here https://lnkd.in/eJnzhgcA or by reaching out to info@chardan.com. #ChardanTransactions  

 Chardan 2024 Top Pick (published, Feb 2, 2024), Alnylam Pharmaceuticals has reported positive topline results from the phase III HELIOS-B study evaluating vutrisiran in ATTR-CM patients, with primary and secondary endpoints achieving statistical significance. Senior Research Analyst, Keay Nakae, CFA maintains a BUY rating and increases the 12-month PT to $265. Alnylam was founded in 2002 by world-renowned RNA scientists Phillip Sharp, Paul Schimmel, Dave Bartel, Thomas Tuschl, and Phillip Zamore. Alnylam has developed 5 commercial RNAi products and has an extensive pipeline of internally-developed product candidates in ongoing or completed clinical trials. Alnylam is headquartered in Cambridge, MA. 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)Sarepta Therapeutics(unrated) announced the FDA expanded approval of its AAVrh74 gene therapy, Elevidys, in Duchenne muscular dystrophy patients 4 years of age and above, with traditional approval granted in ambulatory patients and accelerated approval in non-ambulatory patients. The accelerated approval requires the company to submit confirmatory clinical data in non-ambulatory patients for continued approval. Notably, the approval came despite FDA clinical and statistical reviews concluding the additional sBLA data did not demonstrate the benefit of Elevidys in ambulatory or non-ambulatory patients with CBER director Dr. Peter Marks deciding in favor of approval. Sarepta stock closed up 30% following the news. 2)Silence Therapeutics plc(Buy) announced 48-week data from the PII ALPACAR-360 study of zerlasiran(siRNA) in patients at high risk of atherosclerotic cardiovascular disease events and baseline lipoprotein(a) (Lp(a)) levels of 125 nmol/L or more(n=178, follow-up: 48 weeks). Patients were administered 300mg every 16 or 24 weeks and 450 mg of zerlasiran every 24 weeks, respectively. The study reported results that were consistent with 36-week data (primary endpoint) announced previously, with over 90% median reduction in Lp(a) through the dosing period (48 weeks) for both the doses, compared to placebo. No safety concerns were observed. The patients will be followed through end of study at week 60. The company plans to move forward with the 300mg dose in PIII trial. 3)Taysha Gene Therapies(Buy) announced data from the PI/II REVEAL trial of AAV9 GT TSHA-102 in pediatric and adult Rett syndrome patients. The data from 2 adult Rett patients in cohort 1 demonstrated improvement in clinical parameters including motor and communication skills, autonomic function, seizures. Additionally, 2 pediatric patients also demonstrated early development signs in motor & communication skills and other efficacy parameters. The company plans to report initial data from high dose (1e15 vg) cohorts of trials in adults and pediatric patients in 2H24. 4)Ascidian Therapeutics(pvt) announced license agmt. with Roche(unrated) for the development of RNA based therapeutics for neurological diseases. Under the terms of agreement, Ascidian will license its RNA exon editing technology to Roche for undisclosed neurological indications. Ascidian will be responsible for the discovery and some part of preclinical studies while Roche will conduct preclinical, clinical, and commercial activities. Ascidian will receive an initial payment of $42mm and is eligible for up to $1.8bn in milestone payments, as well as sales royalties. Ascidian's lead candidate, ABCA4, is designed on the company's RNA exon editing technology and targets Stargardt disease, while its other candidates are at the initial stage and focus on neuro and neuro-muscular disorders.