Summary of Savara's Conference Call Company Overview - **Company**: Savara - **Focus**: Clinical stage biopharmaceutical company specializing in rare respiratory diseases - **Product**: Developing MOLBREEVI, an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) for autoimmune pulmonary alveolar proteinosis (APAP) [1][3] Recent Progress - **FDA Submission**: BLA (Biologics License Application) accepted by the FDA with a priority review assigned, PDUFA date set for August 22nd [3][4] - **Clinical Trials**: Positive results from the global phase III study (IMPALA-2) for APAP, supporting confidence in the approval process [3][6] Unmet Need and Treatment Landscape - **Current Situation**: No approved therapies for APAP in the U.S. and Europe; existing treatment involves a mechanical procedure (whole lung lavage) that is not standardized and only available in select centers [8][10] - **MOLBREEVI's Advantage**: Addresses the underlying pathophysiology of APAP by activating macrophages, improving gas exchange and surfactant homeostasis [11] Market Potential - **Patient Population**: Estimated 5,500 diagnosed patients in the U.S., equating to approximately 16 patients per million, which is within the published range of 6-26 patients per million for this rare disease [14][15] - **Treatment Accessibility**: All identified patients could potentially benefit from MOLBREEVI upon approval, with physicians indicating willingness to prescribe it regardless of disease severity [15][19] Commercial Strategy - **Field Force**: Plans to establish a commercial field force of approximately 30 customer-facing personnel, focusing on the top 500 accounts that manage two-thirds of the patient population [20][21] - **Partnerships**: Selected PANTHERx Rare as the exclusive specialty pharmacy, leveraging their experience in rare diseases and existing relationships with pulmonary offices [22][23] Financial Position - **Cash Reserves**: As of the last report, Savara has $264 million in cash, with potential access to an additional $150 million in non-dilutive financing upon FDA approval [26][27] - **Funding Strategy**: Includes a royalty agreement and a debt facility to support operations and commercialization efforts [27] Additional Insights - **Launch Preparations**: Ongoing disease awareness campaigns and educational initiatives are being conducted to prepare for the commercialization of MOLBREEVI [24][25] - **Long-term Outlook**: The company is well-funded and positioned for a successful launch, with a strong focus on addressing the needs of APAP patients [27][28]

Core Viewpoint - Savara Inc. has announced that the FDA has filed for review the Biologics License Application (BLA) for MOLBREEVI, aimed at treating patients with autoimmune pulmonary alveolar proteinosis (PAP), marking a significant milestone for the company and the autoimmune PAP community [1] Group 1 - The FDA has granted Priority Review for the BLA, with a Prescription Drug User Fee Act (PDUFA) action date set for August 22, 2026 [1] - The filing of the BLA is seen as a step closer to potential approval for a new therapy in the treatment of autoimmune PAP [1]

Core Viewpoint - Savara Inc. has announced that the FDA has filed for review the Biologics License Application (BLA) for MOLBREEVI, aimed at treating patients with autoimmune pulmonary alveolar proteinosis (PAP), marking a significant milestone for both the company and the autoimmune PAP community [1] Group 1 - The FDA has granted Priority Review for the BLA, with a Prescription Drug User Fee Act (PDUFA) action date set for August 22, 2026 [1]

Summary of Savara's Conference Call Company Overview - **Company**: Savara - **Focus**: Single asset company specializing in orphan rare pulmonary diseases - **Key Asset**: MOLBREEVI, targeting autoimmune pulmonary alveolar proteinosis (APAP) [3][4] Core Points and Arguments - **Regulatory Progress**: - Resubmitted Biologics License Application (BLA) for MOLBREEVI in December, expecting FDA acceptance soon [3][4] - Anticipates priority review due to Breakthrough Therapy Designation, with PDUFA date projected for August [4] - Plans to file Marketing Authorization Applications (MAAs) in Europe and the UK by the end of Q1 [4] - **Manufacturing Changes**: - Shifted to Fujifilm as the primary drug substance manufacturer after a refusal to file due to manufacturing data issues [5][6] - Alignment achieved with FDA on analytical comparability protocol with Fujifilm [6][8] - **Clinical Data**: - Strong Phase 3 results published in the New England Journal, with DLCO as the primary endpoint showing statistical significance at 24 weeks [9][10] - Key secondary endpoints also demonstrated clinical benefit, including SGRQ and exercise tolerance tests [10][11] - **Market Potential**: - Identified approximately 5,500 diagnosed patients in the U.S., with a goal to confirm 1,000 patients by the end of 2025 [18][19] - Market is concentrated, with top 500 accounts representing about two-thirds of the market [19][20] - **Commercial Strategy**: - Partnering with PANTHERx, a specialty pharmacy, to facilitate product launch and patient access [21] - Ongoing disease awareness campaigns targeting both physicians and patients to increase diagnosis and treatment uptake [24][25] - **Pricing Strategy**: - Pricing corridor established between $400,000 and $500,000 per patient per year, with payers showing willingness to cover under typical prior authorization criteria [32] - **International Plans**: - Plans to commercialize in Europe and the UK independently, with a similar market opportunity as the U.S. [33] - Japan's market strategy remains under evaluation, with potential differences in approach compared to Europe and the UK [35] Additional Important Information - **Patient Awareness**: - Disease awareness campaigns have successfully identified newly diagnosed patients, indicating a potential for higher prevalence than previously estimated [25][27] - **Financial Position**: - Well-funded with $264 million on the balance sheet and potential for $75 million in royalties upon FDA approval [39] - Restructured debt facility allows for up to $150 million in non-dilutive capital upon FDA approval [39] - **Upcoming Catalysts**: - Anticipated updates regarding FDA decision by the end of January, with a focus on maintaining communication with stakeholders [38]

Core Viewpoint - Savara Inc. has amended its loan and security agreement with Hercules Capital, enhancing its financial position and liquidity [1] Financial Strengthening - The amended loan agreement allows for an additional $75 million to be available upon FDA approval of MOLBREEVI, Savara's investigational therapy for autoimmune pulmonary diseases [1]

Core Viewpoint - Bragar Eagel & Squire, P.C. is investigating potential claims against Savara, Inc. due to a class action complaint alleging breaches of fiduciary duties by the company's board of directors during a specified class period [2]. Group 1: Company Overview - Savara, Inc. is facing legal scrutiny following a class action complaint filed on September 8, 2025, concerning events that occurred between March 7, 2024, and May 23, 2025 [2]. - The complaint alleges that Savara's board failed to disclose critical information regarding the MOLBREEVI Biologics License Application (BLA), which lacked sufficient details on chemistry, manufacturing, and controls [8]. Group 2: Financial Impact - On May 27, 2025, Savara announced it received a refusal to file (RTF) letter from the FDA regarding the MOLBREEVI BLA, leading to a significant stock price drop of $0.90 per share, or 31.69%, closing at $1.94 per share [8]. - The delay in regulatory approval for MOLBREEVI increased the likelihood that Savara would need to raise additional capital [8]. Group 3: Legal Actions - Investors who suffered losses during the class period are encouraged to contact Bragar Eagel & Squire for discussions regarding their legal rights and options [1][4]. - The law firm represents both individual and institutional investors in various litigation matters, indicating a broad scope of legal expertise [5].

Company Overview - Savara is focused on building a world-class experienced orphan rare disease company with a team that has extensive experience in the field [3] - The company operates as a single-asset entity with one late-stage product, MOLBREEVI, which is a novel inhaled biologic [3] Product Development - Savara has submitted a Biologics License Application (BLA) in the U.S. for MOLBREEVI's application for use in autoimmune conditions [3]

Summary of Savara's Conference Call Company Overview - **Company**: Savara - **Focus**: Orphan-rare disease company with a single late-stage product, Mobrevi, a novel inhaled biologic for autoimmune pulmonary alveolar proteinosis (autoimmune PAP) [2][3] Industry Context - **Disease**: Autoimmune PAP is a severe, rare lung disease with no approved therapeutic options in the U.S., Europe, or the U.K. [3] - **Current Treatment**: Patients undergo a lengthy diagnostic journey, often requiring a lung lavage procedure, which is not a sustainable long-term solution [5][6][7] Product Details - **Product**: Mobrevi (molgramostim inhalation solution) - **Administration**: Delivered via nebulization, once daily at 300 micrograms using a proprietary eFlow nebulizer system [10] - **Clinical Trials**: - BLA submitted in December based on positive results from the IMPALA-2 trial, which enrolled 164 subjects [11][15] - Primary endpoint (DLCO) showed statistically significant improvement at 24 weeks compared to placebo [12] - Secondary endpoints included SGRQ and exercise capacity, also showing significant improvement [12][13] Market Opportunity - **Patient Population**: Estimated 5,500 diagnosed autoimmune PAP patients in the U.S., equating to approximately 16 patients per million [17][31] - **Market Dynamics**: The U.S. PAP market is concentrated, with the top 500 accounts managing about 65% of identified patients [17] - **Pricing Strategy**: Anticipated pricing corridor of $400,000-$500,000 per patient per year, with expected coverage under typical prior authorization criteria [22][35] Regulatory and Commercial Strategy - **Regulatory Designations**: Mobrevi has received breakthrough designation in the U.S. and orphan drug designation in Europe, providing 12 and 10 years of exclusivity, respectively [16] - **Commercial Preparation**: Active recruitment of a market development team to prepare for potential approval and launch later this year [18][20] - **Diagnostic Test**: Development of a free blood test for diagnosing autoimmune PAP, with 100% sensitivity and specificity [19] Financial Position - **Capitalization**: Company is well-capitalized with approximately $264 million in cash [24] - **Investment Thesis**: Significant unmet need in the market, with potential for blockbuster revenue given the pricing and patient population [24] Additional Insights - **Patient Experience**: The journey to diagnosis can take years, with significant physical and emotional impacts on patients [9] - **Commercial Strategy**: Focus on both moderate and severe patients, emphasizing the importance of early intervention [32] Conclusion - Savara is positioned to address a significant unmet need in the treatment of autoimmune PAP with Mobrevi, supported by strong clinical data and a robust commercial strategy aimed at improving patient outcomes and accelerating diagnosis [24][36]

Core Viewpoint - Savara Inc. has resubmitted the MOLBREEVI Biologics License Application (BLA) to the FDA for the treatment of autoimmune PAP, a rare lung disease [1] Group 1: Company Developments - The resubmission of the MOLBREEVI BLA is in collaboration with Fujifilm as the drug substance manufacturer [1] - The treatment targets autoimmune PAP, which is characterized by the abnormal build-up of surfactant in the alveoli [1]

Core Viewpoint - Savara Inc. is a clinical stage biopharmaceutical company focused on rare respiratory diseases and will present at the 44th Annual J.P. Morgan Healthcare Conference [1] Group 1: Company Presentation Details - The presentation will take place on Wednesday, January 14, at 5:15 PM PT / 8:15 PM ET [1] - Matt Pauls, the Chair and Chief Executive Officer of Savara, will be the presenter [1] - The location of the presentation is the Westin St. Francis, San Francisco, Elizabethan B [1]