Chardan

Chardan biotech analyst Rudy Li, PhD expanded his CNS coverage with three initiations. He initiated coverage of 1) Immunic Therapeutics (IMUX) with a Buy rating and a $3 PT on the potential of its lead asset IMU-838 (vidofludimus calcium) for the treatment of multiple sclerosis (MS). IMU-838 is in Phase 3 development for the treatment of multiple sclerosis (MS). It has a dual MoA targeting both Nurr1 (neuroprotective effect) and DHODH (anti-inflammatory effects), potentially leveraging the success of Aubagio (teriflunomide, DHODH). The company has completed Phase 2 studies in RMS, PMS and UC, demonstrated promising efficacy and clean safety for all tested doses (10mg, 30mg, 45mg). 2) Alto Neuroscience (ANRO) with a Buy rating and a $15 PT as a diversified play in the depression space. The company’s drug development strategy is based on brain-based biomarkers derived from its Precision Psychiatry Platform, with data from neurocognitive assessments, electroencephalography (EEG), wearable devices, as well as genetic and genomic samples. 3) Reviva Pharmaceuticals (RVPH) with a Buy rating and a $2 PT on the potential of its lead asset brilaroxazine as a next-gen antipsychotic product. Brilaroxazine is a serotonin/dopamine modulator in late-stage studies for the treatment of schizophrenia, and the MoA has been validated with multiple products approved. Brilaroxazine has completed 1 Phase 2 and 1 Phase 3 studies in schizophrenia, and 50mg dose has demonstrated consistent treatment effect in 2 studies despite some caveats. Clients can view the full notes here: https://lnkd.in/ejzamzkS (Immunic), https://lnkd.in/e4HTqf9Q (Alto Neuroscience), https://lnkd.in/ee3TccUw (Reviva Pharmaceuticals). To learn more about Chardan research or request access at info@chardan.com.  #ChardanResearch

We’re three weeks out from Chardan’s 9th Annual Genetic Medicines Conference being held October 21, 2025 in New York City. This year’s event features 20 panel discussions, where we bring together industry leaders focused on In-vivo Gene Therapies, RNA Medicines, and Gene Editing to discuss key topics ranging from investment in the space, progress in specific indications, and next wave technologies across each modality. For further information, please contact corpaccess@chardan.com. #ChardanGMConf2025 #CGMC2025 #GeneticMedicines

In this week's Viral News in Genetic Medicines: 1) uniQure (Buy) announced updated data from its pivotal Phase I/II program of AMT-130 in Huntington's disease (HD), including 3-year data for 24 treated patients. The new Phase I/II data indicated that patients who received the high dose of AMT-130 experienced a 75% reduction of disease progression as measured by the composite unified Huntington's disease rating scale (cUHDRS), and a 60% reduction of disease progression as measured by the total functional capacity (TFC) subscale, at 3 years post-treatment compared to matched external control. These data will serve as the basis for a BLA, which uniQure is expected to submit in 1Q26. 2) PepGen (unrated) announced data from its Phase I FREEDOM-DM1 single ascending dose study of PGN-EDODM1 in myotonic dystrophy type 1 (DM1). PGN-EDODM1 is a peptide-conjugated oligonucleotide that binds to mutant DMPK transcripts, thus disrupting its interaction with the splicing protein MBNL1, the sequestration of which is responsible for DM1. The new Phase I data indicated a 53.7% mean splicing correction among the 6 patients in the 15 mg/kg dose cohort as measured by the Thorton 22-gene panel at 28 days post-dosing. 3) Biogen (unrated) announced that it received a complete response letter (CRL) for its sNDA for a high dose regimen of Spinraza (licensed from unrated Ionis Pharmaceuticals) in spinal muscular atrophy. The CRL was related to CMC concerns, and did not cite any deficiencies in the clinical data for the high dose regimen. 4) The FDA issued 3 new Draft Guidance documents for cell and gene therapies, pertaining to i) clinical trial design for programs involving small populations, ii) post-approval studies, and iii) expedited programs for regenerative medicines for serious diseases. Clients can view the full note here: https://lnkd.in/eeJAbPa2. To learn more about Chardan research or request access at info@chardan.com. #ChardanResearch

Congratulations to Kodiak and Ares Acquisition Corporation II (NYSE: AACT) on the successful closing of their business combination. The company has been renamed Kodiak AI, Inc. and is trading on NASDAQ under the symbols “KDK” and “KDKRW”. Kodiak is a leading provider of AI-powered autonomous vehicle technology that is designed to help tackle some of the toughest driving jobs. Kodiak's driverless solution can help address the critical problem of safely transporting goods in the face of unprecedented supply chain challenges. To read more about this transaction visit: https://lnkd.in/gWx-XNNp #ChardanTransactions

In this week's Viral News in Genetic Medicines: 1) VectorY Therapeutics (private) and Shape Therapeutics (private) announced an option and license agreement that grants VectorY the exclusive option to evaluate Shape's deep-brain penetrating AAV5-derived capsid, SHP-DB1, to deliver vectorized antibodies against 3 neurodegenerative disease targets. The agreement will enable IV delivery of VTx-003, a mHTT and TDP-43 dual-targeting antibody for Huntington's disease, and VTx-005, targeting phosphorylated tau in Alzheimer's disease. 2) Biogen (unrated) announced the planned acquisition of Alcyone Therapeutics (private) for an upfront payment of $85 mm plus certain milestones related to the development and regulatory approval of ThecaFlex DRx. ThecaFlex is an implantable subcutaneous port and catheter being developed for the intrathecal delivery of ASOs, which would provide an alternative to the repeated lumbar punctures that are currently standard for intrathecal delivery. 3) Replimune (unrated) announced that it has not yet agreed upon a path forward under accelerated approval for RP1 following a Type A meeting with the FDA on September 16th. In July, the company received a CRL from the Agency as the IGNYTE trial did not provide "substantial evidence of effectiveness", even though the company had aligned with the previous FDA leadership on the trial design. 4) Intellia Therapeutics (Buy) announced it has completed enrollment in the global phase III HAELO trial of lonvoguran ziclumeran (lonvo-z) for hereditary angioedema. The company completed enrollment within 9 months of initiating dosing and expects to report topline data in 1H26 with a BLA filing anticipated in 2H26. Clients can view the full note here: https://lnkd.in/eUVpemDD . To learn more about Chardan research or request access at info@chardan.com. #ChardanResearch

Chardan recently initiated coverage on Ocular Therapeutix, Inc. with a Buy rating and a price target of $21 based on the commercial potential of the company’s lead Axpaxli program in wet AMD and NDPR. Axpaxli (OTX-TKI) is an axitinib intravitreal hydrogel based on the company's proprietary ELUTYX bioresorbable hydrogel formulation technology. The therapy is designed to deliver axitinib, a pan-VEGF receptor inhibitor, intravitreally, where it is slowly released over several months and has a potential to significantly reduce anti-VEGF treatment burden for patients with wet age-related macular degeneration (wet AMD) and non-proliferative diabetic retinopathy (NPDR). Clients can view the full note here: https://lnkd.in/dQAqVXsb. To learn more about Chardan research or request access at info@chardan.com.  #ChardanResearch #Ophthalmology #EquityResearch

Join Chardan Senior Research Analyst Rudy Li, PhD, as he hosts Sleep Disorder Day 2025, a virtual conference exploring the latest in sleep medicine. Together with companies Apnimed, Centessa Pharmaceuticals, and Ognomy Sleep, expert panelists, and a featured KOL, Rudy will lead discussions on the treatment landscape for hypersomnia and OSA, the promise of emerging therapies, and more. Reach out to corpaccess@chardan.com with interest in attending. #Chardan #SleepDisorderDay2025

In this week's Viral News in Genetic Medicines: 1) Avidity Biosciences, Inc. (Buy) announced updated data from the Phase I/II EXPLORE44 and EXPLORE44-OLE trials of del-zota in Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44). The new data show functional benefits at one year among 17 patients (12 ambulatory, 5 non-ambulatory) who were treated with del-zota in EXPLORE44 and continued into EXPLORE44-OLE. These patients improved in various functional metrics such as 4-stair climb, 10-meter walk/run, time-to-rise, and performance of upper limb; while natural history control patients declined in the same metrics. Del-zota-treated patients also maintained stable North Star Ambulatory Assessment scores, while natural history patients declined. 2) Capsida Biotherapeutics (private) announced that the first patient treated in its first-in-human SYNRGY trial of CAP-002 has died. CAP-002 is a next-generation IV-administered gene therapy for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), developed using Capsida's proprietary capsid engineering technology. Capsida announced IND clearance for CAP-002 in May 2025. The company has voluntarily paused the SYNRGY study while it investigates the cause of the patient's death. Clients can view the full note here: https://lnkd.in/gs-8yFTF. To learn more about Chardan research or request access at info@chardan.com.

Chardan Senior Research Analyst, Keay Nakae, CFA, will be hosting a KOL call today at 1pm EST featuring Dr. Jacob Sands, a medical Oncology clinician and clinical researcher specialized in lung cancer. Join them as they discuss current treatment options, a review of results of the phase III IMforte trial, NCCN Category 1 guidelines for evidenced-based cancer treatment recommendations, and more. Register to listen here: https://lnkd.in/ejThCm8t

Chardan is pleased to have acted as a co-manager on Metaplanet Inc. | 株式会社メタプラネット’s ¥212,905,000,000/$1.4 billion international share offering transaction. Metaplanet Inc. (Tokyo Stock Exchange: 3350 / OTCQX: MTPLF) is Japan’s first and only publicly listed Bitcoin Treasury Company. The company leverages Bitcoin as its core treasury reserve asset, employing innovative equity and debt financing strategies to maximize shareholder value. To read more about this transaction visit: https://lnkd.in/e42cmDkQ or reach out to info@chardan.com. #ChardanTransactions #Bitcoin