Skye Bioscience (SKYE) 2025 Conference June 05, 2025 09:20 AM ET Speaker0 Morning, and welcome everyone to the Jefferies Global Healthcare Conference. I'm Jordan Carey with the investment banking team, and it's my pleasure to introduce Puneet Dillon, CEO of Sky Biosciences. Speaker1 Okay. Thank you so much. All right. Good morning, everyone. Yes, thank you again for the invitation and being here at Jefferies. It's always a pleasure seeing the entire Jefferies team and talking about what we're working on at ...

Moleculin Biotech (MBRX) Update / Briefing June 05, 2025 08:30 AM ET Speaker0 Hello, and welcome to the Molecular Biotech Key Opinion Leader Webcast to discuss the company's positive top line efficacy results from its MB-one hundred seven U. S. Phase 1btwo clinical trial evaluating an antiomycin for the treatment of soft tissue sarcoma lung metastases. At this time, I'd like to remind our listeners that remarks made during this webcast may state management's intentions, beliefs, expectations or future proje ...

Core Insights - Autonomix Medical, Inc. has received Ethics Committee authorization to expand its clinical trials for a proprietary ablation technology aimed at treating visceral cancer pain, building on initial success in pancreatic cancer pain [2][3] - The follow-on phase of the clinical program (PoC 2) is set to begin patient enrollment in June 2025, with the potential to double the addressable market by including additional visceral cancers [2][3][4] Group 1: Clinical Development - The PoC 2 phase will evaluate the safety and effectiveness of delivering transvascular energy to ablate problematic nerves, with a focus on interventional cancer pain management applications [2][4] - Initial trials in late-stage pancreatic cancer patients showed promising results, including significant pain reduction and decreased opioid use, indicating the technology's potential to transform cancer pain management [3][4] Group 2: Market Expansion - The expansion into visceral cancer pain aligns with Autonomix's strategy to create long-term value through multi-indication growth, addressing significant unmet needs in cancer treatment [3][4] - The technology platform has the potential to address various indications beyond cancer, including cardiology and chronic pain management, indicating a broad market opportunity [4][6][7] Group 3: Technology Overview - Autonomix's technology includes a catheter-based microchip sensing array designed to detect and differentiate neural signals with greater sensitivity than existing technologies [6][7] - The investigational nature of the technology means it has not yet been cleared for marketing in the United States, highlighting its developmental stage [7]

Core Insights - Tonix Pharmaceuticals Holding Corp. is set to present a poster at the Annual European Congress of Rheumatology (EULAR) 2025, focusing on its product candidate TNX-102 SL for fibromyalgia treatment [1][3] Company Overview - Tonix is a fully-integrated biotechnology company with a focus on pain management therapies and vaccines for public health challenges [4] - The company is advancing TNX-102 SL, which has a PDUFA goal date of August 15, 2025, for marketing authorization based on two significant Phase 3 studies [4] - TNX-102 SL has received Fast Track designation from the FDA for fibromyalgia management [4] - The company is also developing TNX-102 SL for acute stress reaction and acute stress disorder under a study funded by the U.S. Department of Defense [4] Product Development - Tonix's immunology portfolio includes TNX-1500, a monoclonal antibody targeting CD40-ligand for preventing organ transplant rejection and treating autoimmune diseases [4] - The infectious disease portfolio features TNX-801, a vaccine for mpox and smallpox, and TNX-4200, a broad-spectrum antiviral agent with a contract worth up to $34 million from the U.S. DoD [4] Marketed Products - Tonix Medicines, the commercial subsidiary, markets Zembrace® SymTouch® and Tosymra® for acute migraine treatment [4]

Core Insights - Immunic, Inc. has completed enrollment for both Phase 3 ENSURE trials of vidofludimus calcium in patients with relapsing multiple sclerosis (RMS), with top-line data expected by the end of 2026 [1][3] - Additional data from the Phase 2 CALLIPER trial in progressive multiple sclerosis (PMS) supports the positive results previously released, highlighting the neuroprotective potential of vidofludimus calcium [1][4] Group 1: ENSURE Trials - The ENSURE program consists of two identical multicenter, randomized, double-blind Phase 3 trials aimed at evaluating the efficacy, safety, and tolerability of vidofludimus calcium versus placebo in RMS patients [2] - A total of 1,121 patients were enrolled in ENSURE-1 and 1,100 patients in ENSURE-2, with the primary endpoint being the time to first relapse over 72 weeks [2] - Secondary endpoints include time to confirmed disability worsening, volume of new T2 lesions, and MRI-based endpoints [2] Group 2: CALLIPER Trial Data - In the Phase 2 CALLIPER trial, vidofludimus calcium demonstrated a 24% reduction in the hazard ratio for 24-week confirmed disability worsening (24wCDW) compared to placebo [5] - The drug showed a 33% reduction in 24wCDW in primary progressive multiple sclerosis (PPMS) patients, a 19% reduction in non-active secondary progressive multiple sclerosis (naSPMS), and a 34% reduction in active secondary progressive multiple sclerosis (aSPMS) [5] - For patients without evidence of gadolinium-enhancing lesions at baseline, vidofludimus calcium reduced 24wCDW by 34% compared to placebo [5][6] Group 3: Vidofludimus Calcium Overview - Vidofludimus calcium is an investigational oral small molecule drug targeting chronic inflammatory and autoimmune diseases, currently in late-stage clinical trials for multiple sclerosis [7] - The drug acts as a selective immune modulator, activating the neuroprotective transcription factor Nurr1 and inhibiting dihydroorotate dehydrogenase (DHODH) to provide neuroprotective, anti-inflammatory, and anti-viral effects [7] - The drug has been tested in approximately 2,700 individuals, showing an attractive pharmacokinetic, safety, and tolerability profile [7]

Cullinan Therapeutics Licensing of velinotamig (BCMAxCD3) from Genrix Bio June 2025 © CULLINAN THERAPEUTICS, INC. ALL RIGHTS RESERVED. CONFIDENTIAL AND PROPRIETARY Important Notice and Disclaimers This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Cullinan's beliefs and expectations regarding the potential benefits of, and pl ...

Achieve Life Sciences (ACHV) Conference Summary Company Overview - Achieve Life Sciences is focused on addressing nicotine dependence through its drug cytisinicline, which is positioned as a potential blockbuster treatment for smoking and vaping cessation [3][4][5] Industry Context - The U.S. faces a significant public health crisis with approximately 46 million individuals affected by nicotine dependence, including 29 million smokers and 17 million vapers [3][4] - The vaping population has increased by 6 million in the last 24 months, highlighting a growing concern [4] Key Points and Arguments 1. **Market Opportunity**: - The addressable market for nicotine dependence treatment is substantial, with an estimated annual market opportunity of $11 billion based on previous treatments like Chantix [33][35] - There are 15.4 million annual attempts to quit smoking, indicating a high demand for effective cessation tools [33] 2. **Product Development**: - Cytisinicline is expected to be the first new treatment for nicotine dependence in nearly 20 years, with an NDA filing planned for the end of June 2025 and anticipated FDA approval by mid-2026 [5][22] - The drug has shown a differentiated efficacy profile in clinical trials, with odds ratios of 5.3 and 5.8 in two phase three trials, indicating a significantly higher likelihood of quitting compared to existing treatments [19][20] 3. **Clinical Trials**: - Cytisinicline has undergone two large-scale phase three clinical trials involving over 1,600 patients, demonstrating robust efficacy and tolerability [5][25] - A phase two clinical trial for vaping cessation showed an odds ratio of 2.65, with plans for a phase three trial starting in early 2025 [27][23] 4. **Regulatory Pathway**: - The NDA submission is expected to be accepted within 74 days, with a clear regulatory pathway outlined by the FDA [22][43] 5. **Commercial Strategy**: - Achieve Life Sciences plans a targeted digital marketing strategy focusing on primary care physicians and patients, leveraging the lack of competition in the smoking cessation market since Chantix became generic [30][39] - The company aims to change the narrative around nicotine dependence from a lack of willpower to a medical condition, enhancing awareness and acceptance of cytisinicline [35][36] 6. **Safety and Tolerability**: - Cytisinicline has a favorable safety profile, with side effects significantly lower than those of existing treatments like Chantix, particularly in terms of nausea and vomiting [20][21] Additional Insights - The company has a strong intellectual property position and a proven leadership team, which is expected to drive shareholder value [43][44] - There is potential for future indications beyond nicotine dependence, including cannabis use, although the focus remains on the immediate market opportunity [50][52] Conclusion - Achieve Life Sciences is positioned to make a significant impact in the nicotine dependence treatment market with cytisinicline, addressing a critical public health issue while leveraging a well-defined commercial strategy and strong clinical data [44][54]

Summary of BioCryst Pharmaceuticals (BCRX) Conference Call Company Overview - **Company**: BioCryst Pharmaceuticals (BCRX) - **Focus**: Rare diseases, specifically targeting hereditary angioedema (HAE) with its lead drug Orlodayo, which is in its fifth year of launch [4][6] Financial Performance - **Profitability**: Expected to be profitable in 2025, with a strong financial position and debt reduction already achieved this year [5][68] - **Revenue Guidance**: Increased revenue guidance for 2025 to $580 million - $600 million from a previous estimate of $535 million - $550 million, driven by strong demand and patient access to paid therapy [6][7] Product Pipeline Orlodayo - **Market Performance**: Strong demand from both patients and physicians, particularly in the Medicare and commercial populations [7] - **Pediatric Opportunity**: FDA accepted the supplemental NDA for pediatric use with a PDUFA date set for September 12, 2025 [8][9] Netherton Syndrome (Drug: 17725) - **Mechanism of Action**: KLK5 inhibitor targeting a genetic disorder with a clear mechanism involving faulty gene function leading to uncontrolled KLK5 activity [12][13] - **Clinical Trials**: Initial evaluations in healthy volunteers have been positive, allowing progression to patient trials [20][21] - **Study Design**: Phase one study designed to assess drug penetration and skin healing, with a focus on skin outcomes and patient-reported outcomes [31][36] - **Market Opportunity**: Estimated 1,600 Netherton patients in the U.S., with potential growth as targeted therapy becomes available [49][50] Diabetic Macular Edema (DME) - **Drug: Vorlestat**: Lower solubility drug being developed for a suprachoroidal delivery approach, aiming for sustained exposure at the retina [58][60] - **Preclinical Data**: Positive results in animal models showing significant reduction in retinal vascular leakage, supporting the mechanism for DME treatment [63][64] - **Phase One Study**: Small study focusing on safety and retinal thickness changes over time, with a bar for success being measurable changes in retinal thickness [65][66] Financial Strategy - **Debt Management**: Paid down $75 million in Pharmakon debt, with a remaining debt of $249 million and a cash position of approximately $240 million [68] - **Future Investments**: Plans to continue paying down debt while investing in the pipeline and potential business development opportunities [68] Key Takeaways - BioCryst is positioned for growth with a strong financial outlook and promising drug pipeline focused on rare diseases - The company is actively pursuing regulatory approvals for pediatric indications and expanding its clinical trials for Netherton Syndrome and DME - Financial health is robust, allowing for continued investment in research and development while managing debt effectively [5][68]

Scholar Rock Holding (SRRK) 2025 Conference June 04, 2025 03:45 PM ET Speaker0 Fantastic. All right. Well, thank you. Good afternoon everyone. Welcome to the next session here at the Jefferies Healthcare Conference. I'm Michael Yee, a senior biotechnology analyst, and really happy to have a couple members of the ScholarRock management team with us. And importantly, this somewhat of a new team that got introduced over the last month. So I'd love perhaps, although David, you were chairman, an opportunity for ...

BRISBANE, Calif., June 04, 2025 (GLOBE NEWSWIRE) -- Vera Therapeutics, Inc. (Nasdaq: VERA), a late clinical-stage biotechnology company focused on developing and commercializing transformative treatments for patients with serious immunological diseases, today announced that the Company’s management team will participate in a fireside chat at the Goldman Sachs 46th Annual Global Healthcare Conference, which is taking place in Miami Beach, FL from June 9 – 11, 2025. The management team will also participate i ...