– SLE and LN registrational discussions with FDA anticipated in 3Q25; systemic sclerosis registrational discussions with FDA anticipated in 4Q25 – – New clinical data on rese-cel in myositis, SLE / LN and systemic sclerosis to be presented in three oral sessions at the EULAR 2025 Congress in June – – Two subtype specific cohorts with ~15 patients each added to the ongoing RESET-Myositis™ trial – As of March 31, 2025, the Company had cash and cash equivalents of $131.8 million, per our Form 10-Q being filed ...

LEXINGTON, Mass., May 15, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, today announced the first peer-reviewed publication of data demonstrating the ability of alkaline phosphatase (ALPL) to transport a novel AAV capsid across the blood-brain barrier (BBB). The article, titled "Highly conserved brain vascular receptor ALPL mediates transport of engineered AAV vectors across the blood-brain barrier ...

Company Overview - Aura Biosciences, Inc. is a clinical-stage biotechnology company focused on developing precision therapies for solid tumors aimed at preserving organ function [5] - The company's lead candidate, bel-sar (AU-011), is in late-stage development for primary choroidal melanoma and early-stage development for other ocular oncology indications and bladder cancer [5] Public Offering Announcement - Aura announced the launch of an underwritten public offering of shares of its common stock, including pre-funded warrants and accompanying warrants [1] - The offering is subject to market conditions, and there is no assurance regarding its completion or the actual size and terms [1] Management and Underwriters - Leerink Partners and Evercore ISI are acting as joint bookrunning managers for the offering, with LifeSci Capital also serving as a bookrunning manager [2] - Citizens Capital Markets and Scotiabank are co-managers for the offering [2] Regulatory Compliance - The offering is made pursuant to a shelf registration statement filed with the SEC on March 27, 2024, and declared effective on April 5, 2024 [3] - A preliminary prospectus supplement and accompanying prospectus will be filed with the SEC and made available on their website [3] Investor Relations - For inquiries, investors can contact Alex Dasalla, Head of Investor Relations and Corporate Communications at Aura [7]

Core Viewpoint - The Gross Law Firm has issued a notice to shareholders of Sana Biotechnology, Inc. regarding a class action lawsuit due to allegations of misleading statements and financial misrepresentation during a specified class period [1][2]. Group 1: Allegations Against Sana Biotechnology - The complaint alleges that Sana was at significant risk of having insufficient funds to maintain its current operations and advance its product candidates [1]. - It is claimed that product candidates SC291 in oncology, SC379, and SG299 were less promising than previously communicated to investors [1]. - To preserve cash and focus on more promising candidates, Sana is likely to decrease funding for SC291, SC379, and SG299, and significantly reduce its workforce [1]. - The defendants allegedly overstated Sana's financial capacity to maintain operations and advance existing product candidates [1]. - As a result, the public statements made by the defendants were materially false and/or misleading throughout the relevant period [1]. Group 2: Class Action Participation - Shareholders who purchased shares of SANA during the class period from March 17, 2023, to November 4, 2024, are encouraged to register for the class action [2]. - The deadline for shareholders to seek lead plaintiff status is May 20, 2025, and there is no cost or obligation to participate [2]. - Registered shareholders will be enrolled in a portfolio monitoring software to receive updates on the case [2]. Group 3: About The Gross Law Firm - The Gross Law Firm is a nationally recognized class action law firm dedicated to protecting the rights of investors affected by deceit and illegal business practices [3]. - The firm aims to ensure companies adhere to responsible business practices and seeks recovery for investors who suffered losses due to misleading statements [3].

Core Viewpoint - PolTREG S.A. has received a positive opinion from the European Medicines Agency's Paediatric Committee regarding its Pediatric Investigation Plan for PTG-007, a therapy aimed at preventing symptomatic type 1 diabetes in children, which could lead to potential marketing authorization in the EU and EEA [1][2][8] Group 1: Pediatric Investigation Plan and Clinical Trials - The PDCO's positive opinion is based on PolTREG's clinical trial protocol initiated in October 2024, which originally enrolled children aged 6–16 years, with a recommendation to broaden eligibility to ages 3–18 years [3][8] - Achieving the clinical endpoints defined in the PIP may serve as a basis for obtaining marketing authorization for PTG-007 [2][8] Group 2: Product Development and Safety Data - PolTREG combines over 12 years of clinical data confirming the safety and efficacy of polyclonal Treg therapies, making it the only company with such extensive patient-safety and efficacy data for autologous polyclonal Treg therapies [4][9] - In vivo murine studies have shown preliminary safety and efficacy for CAR-TREG lymphocytes, supporting upcoming Phase 1 clinical trials in multiple sclerosis and amyotrophic lateral sclerosis [2][5][8] Group 3: Manufacturing and Expansion Capabilities - PolTREG operates a GMP-certified manufacturing facility, one of Europe's largest, with over 2,100 sqm of laboratory space and 15 production lines, allowing for the production of Treg therapeutics [6][7] - The company has the option to expand its facility to accommodate next-generation engineered therapies and can ship its cellular therapy products across Europe within 24 hours [7]

Summary of Immunovant (IMVT) Conference Call Company Overview - **Company**: Immunovant (IMVT) - **Event**: BofA Annual Healthcare Conference - **Date**: May 14, 2025 Key Points Company Strategy and Management Changes - Immunovant is transitioning its focus from betoklimab to IMG1402, a next-generation program, following late-stage study readouts in myasthenia gravis (MG) and chronic inflammatory demyelinating polyneuropathy (CIDP) [5][6] - Eric Vanker has been appointed as the new CEO, emphasizing clinical execution and patient enrollment in Graves' disease as top priorities [6][7] Focus on Graves' Disease - Graves' disease is identified as a unique opportunity for Immunovant, with a significant patient population and high unmet medical need [8][9] - The company aims to establish a strong presence in Graves' disease, similar to Argenx in MG [9][10] - Approximately 350,000 patients in the U.S. are refractory to current treatments, representing a substantial market opportunity [21][22] Clinical Trial Challenges - Enrollment for clinical trials in Graves' disease is complicated due to a lack of recent novel drug development and the need for operational infrastructure in many physician practices [24][25] - The company is working to address these operational challenges to facilitate patient enrollment [25] Value Proposition of Therapy - The therapy aims to improve patient quality of life by reducing reliance on methimazole, which has been the standard treatment for decades [20][32] - Over 50% of patients in high-dose trials were able to discontinue methimazole within 12 weeks [34][45] - The potential for drug-induced remission is highlighted as a significant benefit, with ongoing studies to assess long-term outcomes [35][46] Regulatory Environment - The FDA has shown enthusiasm for new therapies in Graves' disease, indicating a constructive relationship for regulatory approval [48][49] Myasthenia Gravis (MG) and CIDP - Immunovant acknowledges the challenges of entering the MG market, where Argenx has a strong foothold [50][53] - The company believes that deeper IgG suppression may yield better clinical benefits, positioning itself for potential success despite being a late entrant [52][56] Future Development and Pipeline - Immunovant is focused on executing its current indications before exploring new opportunities, although additional indications may be announced in the future [63][64] - The company is also developing a phase three trial for dermatomyositis, with expectations for regulatory approval based on statistical significance [68][73] Legal Considerations - Immunovant is involved in a legal case against Moderna regarding patent infringement related to lipid nanoparticles used in COVID vaccines, with potential significant financial implications depending on the trial outcome [87][90] Additional Insights - The company is optimistic about the potential for its therapies to address significant unmet needs in autoimmune diseases, particularly in Graves' disease and MG, while navigating the complexities of clinical trials and regulatory pathways [36][56][63]

Summary of Roivant Sciences (ROIV) Conference Call Company Overview - **Company**: Roivant Sciences (ROIV) - **Event**: BofA Annual Healthcare Conference - **Date**: May 14, 2025 Key Points Immunovant and Clinical Focus - Roivant is transitioning its focus to IVD 1402, a next-generation program, after late-stage studies in betoclimab for MG and CIDP [8] - Eric Vanker has taken over as CEO of Immunovant, emphasizing patient enrollment in Graves' disease as the top priority [9] - Graves' disease is identified as a unique opportunity with a large patient population and high unmet need [11] Market Dynamics and Patient Population - Approximately 350,000 Graves' patients in the US are refractory to current treatments, primarily methimazole [25] - The current treatment landscape for Graves' disease has not seen a novel drug in 70 years, creating a significant opportunity for Roivant [29] - The company aims to address the operational challenges of enrolling patients in clinical trials due to the lack of recent novel therapies [30] Value Proposition of Therapy - The therapy aims to improve patient quality of life by reducing reliance on methimazole and potentially achieving drug-free remission [36] - Evidence from phase two data shows that 76% of patients on high doses achieved T3 and T4 normalization, with 56% able to discontinue anti-thyroid drugs [46] - The company is also exploring the potential to reduce the onset of Thyroid Eye Disease (TED) in hyperthyroid patients with proptosis [38] Regulatory and Clinical Trial Insights - The FDA has shown enthusiasm for new therapies in Graves' disease, indicating a supportive regulatory environment [51] - The first GRAVE study will treat patients for six months before randomization to assess remission rates [41] - The company is optimistic about the potential for accelerated approval based on positive clinical outcomes [85] Myasthenia Gravis (MG) and CIDP Challenges - Roivant acknowledges the challenges of entering the MG market, dominated by Argenx, and recognizes the need for deeper clinical data to compete effectively [58] - The company aims to shift the focus in MG from treatment response to deeper remission endpoints, which could favor their drug's profile [61] Dermatomyositis and Other Programs - Roivant is preparing for a phase three trial in dermatomyositis, with expectations for regulatory approval based on statistical significance [78] - The company is also developing a pulmonary hypertension program, with data expected in the second half of next year [87] Legal Matters - Roivant is involved in litigation regarding intellectual property related to lipid nanoparticles used in COVID vaccines, with a jury trial scheduled for September 2025 [90] - Potential damages from the case could be substantial, given the scale of global COVID vaccine sales [93] Additional Insights - The company is focused on executing its current indications before exploring new opportunities, indicating a disciplined approach to R&D investment [66] - Roivant is evaluating its pipeline continuously and may announce new indications as they progress [66] This summary encapsulates the key discussions and insights from the Roivant Sciences conference call, highlighting the company's strategic focus, market opportunities, and ongoing challenges in the biopharmaceutical landscape.

Summary of Relay Therapeutics Conference Call Company Overview - **Company**: Relay Therapeutics (Ticker: RLAY) - **Event**: BofA Annual Healthcare Conference - **Date**: May 14, 2025 Key Industry Insights - **Focus on Precision Oncology**: The company has faced challenges in precision oncology and is now prioritizing its PI3K program for breast cancer, indicating a strategic shift in resource allocation towards this program [2][4] - **Restructuring and Cost Management**: Relay Therapeutics has made significant reductions in its research footprint due to limited access to capital, focusing on maximizing value from its balance sheet over the next three to five years [4][5] Financial Position - **Cash Reserves**: The company currently holds $710 million in cash, which is expected to sustain operations and support studies through 2029 [5] Clinical Development - **PI3K Alpha Program**: The PI3K alpha immune selective molecule (02/1400) is identified as a key driver for the company, with plans to advance through pivotal studies [5][9] - **Upcoming Studies**: The company is preparing for the Rediscover II Phase III study, with expectations of presenting updated data at the upcoming ASCO conference [9][10] - **Vascular Malformation Study**: A new study has been initiated for a PIK3CA mutant-driven genetic disease, with an estimated 170,000 patients in the U.S. [6][53] Competitive Landscape - **Benchmarking Against Competitors**: The company is comparing its clinical data against competitors, noting that its median progression-free survival (PFS) is significantly better than the competitive benchmark of 5.5 months [12][14] - **Differentiation Strategy**: Relay Therapeutics aims to establish a next-generation profile compared to existing therapies, emphasizing the importance of being first to market in a large patient population [19][20] Regulatory Considerations - **Potential Labeling Strategies**: The company is considering the evolving landscape of CDK4/6 therapies and aims to construct studies that could lead to line-agnostic approvals [21][23] Market Opportunity - **Vascular Malformations Market**: The potential market for PIK3CA-driven vascular malformations is significant, with estimates suggesting that 10-40% of patients may seek systemic therapy, translating to a meaningful commercial opportunity [53][54] Future Directions - **Ongoing Research**: The company plans to advance its Fabry and NRAS programs to IND readiness, with decisions on further development contingent on the macro environment and business considerations [57] Additional Notes - **Safety and Efficacy**: The company believes that its experience in oncology has de-risked safety profiles for its new indications, allowing for a more favorable assessment of efficacy in younger patient populations [43][44] - **Strategic Focus**: Relay Therapeutics is maintaining a small research footprint focused on high-value oncology targets while exploring additional preclinical programs as opportunities arise [57]

Novavax (NVAX) 2025 Conference Summary Company Overview - **Company**: Novavax (NVAX) - **Event**: 2025 Bank of America Healthcare Conference - **Date**: May 14, 2025 Key Points Industry and Company Developments - Novavax has transitioned from a small biotech to a more structured organization post-pandemic, focusing on leveraging its technology platform for growth through partnerships and R&D [3][4] - The company has launched four new R&D programs in the last quarter, indicating a robust pipeline for future growth [3] Financial Guidance and Performance - Novavax raised its revenue guidance based on BLA milestones from its partnership with Sanofi, with expectations of breakeven and profitability as early as 2027 [6][7] - The company anticipates receiving $2.25 billion in cash from BLA approvals and license transfers to Sanofi, which includes a $1.75 billion milestone and two $25 million license transfers [39][40] Regulatory and Approval Pathways - Novavax is in discussions with the FDA regarding post-marketing commitments, which are expected to be less stringent than requirements, allowing for potential approval in the near term [9][10][14] - The company has a robust dataset from a placebo-controlled trial involving 30,000 individuals for its COVID vaccine, Nuvaxavid, supporting its approvability [17][18] Market Dynamics and Vaccine Strategy - The company is observing a shift in consumer choice towards vaccines with better tolerability, which could favor Novavax's offerings [58][59] - The COVID vaccine market is expected to grow significantly, with Novavax aiming to capture a substantial share through its innovative technology and partnerships [77] Partnerships and Collaborations - Novavax has expanded its partnership with Sanofi and signed new agreements with top pharmaceutical companies to explore the utility of its Matrix M adjuvant in various vaccine platforms [4][48][50] - The updated agreement with Takeda includes a $20 million upfront payment and a shift to a traditional royalty model, enhancing the partnership's economic viability [44][46] Future Outlook - The company is focused on reducing liabilities and costs, aiming for an 80-85% reduction in R&D and SG&A expenses by 2027, which will enable a stronger focus on value creation [76][77] - Novavax is positioned to capitalize on the growing vaccine market, projected to increase from $50 billion to $75 billion, leveraging its technology across multiple vaccine categories [77] Additional Insights - The company is actively monitoring the evolving landscape of COVID variants and the need for seasonal vaccine updates, which is critical for maintaining market relevance [26][28] - Novavax's strategy includes developing combination vaccines and exploring new markets, such as oncology, to diversify its product offerings [48][52] Conclusion - Novavax is strategically positioned for growth through innovative partnerships, a strong pipeline, and a focus on cost reduction, aiming to capture a significant share of the expanding vaccine market while ensuring product safety and efficacy.

Group 1 - The company MIRXES-B (02629) plans to conduct an initial public offering (IPO) from May 15 to May 20, 2025, with a total of 46.62 million shares being offered globally, where 10% is allocated for Hong Kong and 90% for international markets [1] - The offering price is set at HKD 23.30 per share, with trading expected to commence on May 23, 2025, at 9:00 AM on the Hong Kong Stock Exchange [1] - Founded in 2014 and headquartered in Singapore, the company specializes in microRNA (miRNA) technology, focusing on making disease screening and diagnostic solutions accessible in key Asian markets, including Singapore and China [1] Group 2 - The estimated net proceeds from the global offering, calculated at the offering price of HKD 23.30 per share, will be approximately HKD 880.5 million [2] - The intended use of the net proceeds includes 51% for research and development, regulatory filings, production, and commercialization of the core product GASTROClearTM; 24% for ongoing and planned R&D for pipeline products; 15% to enhance and integrate the company's end-to-end capabilities; and 10% for working capital and general corporate purposes [2] - The company has entered into cornerstone investment agreements with Beijing Xunrui Enterprise Management Partnership (Limited Partnership) and Evergreen Gate Limited, with cornerstone investors agreeing to subscribe for USD 57.92 million under certain conditions [2]