Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Savara Inc. and its officers or directors, following a significant stock price drop after negative FDA news [1][3]. Group 1: Investigation Details - Pomerantz LLP is representing investors of Savara Inc. and is looking into claims of securities fraud or other unlawful business practices [1]. - Investors are encouraged to contact Pomerantz LLP for more information regarding the investigation [1]. Group 2: Stock Performance - On May 27, 2025, Savara announced it received a refusal to file letter from the FDA regarding its Biologics License Application for MOLBREEVI, leading to a stock price decline of $0.90 per share, or 31.69%, closing at $1.94 per share [3].

Savara Inc (SVRA) 2025 Conference Summary Company Overview - Savara Inc is a biotech company focused on orphan rare diseases, specifically in the rare pulmonary space with a single asset, Molgrimostum inhalation solution, branded as Molbrevi [6][7] Key Points and Arguments Clinical Development - Positive phase three data for IMPALA two trial was reported, involving 164 patients with autoimmune pulmonary alveolar proteinosis (APAP) [7] - The trial showed positive results on the primary endpoint of DLCO and all key secondary endpoints at 24 and 48 weeks [7] - A Biologics License Application (BLA) resubmission is planned for Q4 2025, with a potential PDUFA date in mid-2026 [8][48] Market Opportunity - Estimated 3,600 diagnosed APAP patients in the U.S., with a potential market size of approximately 7,300 when including undiagnosed patients [10][12] - Pricing research indicates a price range of $300,000 to $500,000 per patient per year, suggesting significant pricing power [13] Treatment Insights - Molbrevi is expected to be a first-line chronic therapy for APAP, addressing the pathophysiology of the disease [14][16] - The drug is administered once daily using a proprietary nebulizer, contrasting with alternatives that require twice-daily administration [50] Regulatory Challenges - The recent refusal to file (RTF) was due to the need for additional Chemistry, Manufacturing, and Controls (CMC) data, not related to clinical trials or safety concerns [21][22] - The company is working with two drug substance partners, GEMA and Fujifilm, to ensure compliance and readiness for the BLA resubmission [31][39] Future Plans - A type A meeting with the FDA is scheduled within 30 days of the RTF, with meeting minutes expected within 90 days [30][31] - European and UK regulatory filings are on track for year-end 2025 [46] Additional Important Information - The company is preparing for an Advisory Committee (AdCom) meeting, considering it the first therapeutic for this orphan rare disease [45] - There is an ongoing Early Access Program (EAP) for patients awaiting approval of Molbrevi [57] - Approximately 10% of patients in previous trials underwent lung lavages, indicating a need for chronic therapeutic options [60] - The company is scaling up its ILD clinic pilot to identify undiagnosed APAP patients using an antibody test [61] This summary encapsulates the critical insights and developments regarding Savara Inc and its lead product, Molbrevi, as discussed in the conference.

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Savara Inc. and its officers or directors following a significant stock price drop after an FDA refusal letter [1][3]. Group 1: Company Overview - Savara Inc. is a publicly traded company on NASDAQ under the ticker SVRA [1]. - The company recently announced it received a refusal to file letter from the FDA regarding its Biologics License Application for MOLBREEVI, a therapy aimed at treating autoimmune PAP [3]. Group 2: Stock Performance - Following the FDA news, Savara's stock price decreased by $0.90 per share, representing a decline of 31.69%, closing at $1.94 per share on May 27, 2025 [3]. Group 3: Legal Investigation - Pomerantz LLP, a prominent firm in corporate and securities class litigation, is leading the investigation into Savara's potential misconduct [1][4]. - The firm has a history of recovering significant damages for victims of securities fraud and corporate misconduct [4].

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Savara Inc. following a significant stock price drop after the FDA's refusal to file letter regarding its drug application [1][3]. Company Summary - Savara Inc. received a refusal to file letter from the FDA for its Biologics License Application for MOLBREEVI, intended to treat autoimmune PAP [3]. - Following this announcement, Savara's stock price decreased by $0.90, representing a 31.69% decline, closing at $1.94 per share on May 27, 2025 [3]. Legal Investigation - Pomerantz LLP is representing investors of Savara and is looking into claims of securities fraud or other unlawful practices by the company and its officers or directors [1].

Core Points - Savara Inc. received a refusal to file letter from the FDA regarding its Biologics License Application for MOLBREEVI, intended for treating autoimmune pulmonary alveolar proteinosis [1] - Following the FDA's decision, Savara's stock price experienced a decline [1] Company Information - Holzer & Holzer, LLC is investigating whether Savara complied with federal securities laws in light of the recent FDA decision [1] - The law firm specializes in representing shareholders and investors in litigation, including class action and derivative litigation, and has recovered hundreds of millions of dollars for affected shareholders since its founding in 2000 [3]

Core Viewpoint - Savara Inc. received a U.S. FDA refusal to file (RTF) letter regarding its Biologic License Application (BLA) for Molbreevi, a treatment for autoimmune Pulmonary Alveolar Proteinosis (PAP) [1][3]. Group 1: FDA Response - The FDA determined that the BLA submitted in March 2025 was not sufficiently complete for substantive review and requested additional data related to Chemistry, Manufacturing, and Controls (CMC) [3]. - The RTF did not arise from safety concerns, and the FDA did not request additional efficacy studies [3]. Group 2: Company Response and Future Plans - The company plans to request a Type A meeting with the FDA within 30 days to discuss the next steps [3]. - The requested CMC data outlined in the RTF letter are currently being generated, and the company is confident in addressing the FDA's request [4]. - The company expects to resubmit the BLA in the fourth quarter of 2025 and remains confident in the clinical data supporting Molbreevi's efficacy [4]. Group 3: Operational Updates - The company is working to establish a redundant supply chain and is on track to complete technology transfer with a second-source drug substance contract manufacturer in the fall [4]. - The company has completed three upstream process performance qualification (PPQ) batches and is in the process of completing the downstream PPQ campaign [4]. Group 4: Market Reaction - Following the news, SVRA stock declined by 28.9%, trading at $2.02 [4].

Group 1 - Savara Inc. shares fell over 30% in intraday trading on May 27, 2025, following the FDA's issuance of a Refusal to File letter for its Biologics License Application for Molbreevi, its lead drug candidate for autoimmune PAP [2] - The FDA cited that the submission was "not sufficiently complete" for review, specifically pointing out issues with the CMC section of the application, and Savara was unable to specify what data was missing or why deficiencies were not addressed prior to submission [2] Group 2 - Block & Leviton is investigating Savara Inc. for potential securities law violations and may file an action to recover losses for investors [4] - Investors who have lost money in Savara Inc. investments are encouraged to contact Block & Leviton to learn more about recovery options [5][3] Group 3 - Block & Leviton is recognized as a leading securities class action firm, having recovered billions of dollars for defrauded investors and representing many top institutional investors [7]

Financial Data and Key Metrics Changes - At the end of Q1 2025, the company had over $172 million in cash and short-term investments [23] - A $200 million debt facility was executed with Hercules, providing a cash runway into the second half of 2027 [23] Business Line Data and Key Metrics Changes - The company is focused on a single development program for a rare disease called autoimmune pulmonary alveolar proteinosis (APAP) [2] - The Phase III pivotal clinical trial IMPALA two showed statistically significant improvement in DLCO compared to placebo at week 24, with sustained improvement at week 48 [7][8] - 97% of patients completed the double-blind treatment period, with no withdrawals due to drug-related adverse events [8] Market Data and Key Metrics Changes - The diagnosed prevalence of APAP in the U.S. is estimated at approximately 3,600 patients, with an additional 3,700 likely undiagnosed patients [13][14] - The potential market opportunity in the U.S. is significant, with a total of over 7,000 patients identified [15] Company Strategy and Development Direction - The company aims to establish relationships with pulmonologists and treatment centers to gain visibility into the patient population before the launch of Molbrevi [17] - A U.S. commercial team is being built prior to approval, consisting of 25 to 30 individuals responsible for patient profiling and disease awareness campaigns [22] - The pricing power for Molbrevi is projected between $300,000 and $500,000 per patient per year, aligning with other orphan drug analogues [24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of Molbrevi, highlighting the lack of approved medicines for APAP in the U.S. and Europe [4][11] - The company anticipates a potential PDUFA date around November if priority review is granted by the FDA [11] - There is a strong interest from U.S. pulmonologists and payers regarding Molbrevi, with 83% of pulmonologists likely to prescribe it [18][19] Other Important Information - The company launched a free blood antibody test called APAP ClearPath to facilitate quicker diagnosis of APAP [20] - The test has been piloted at an interstitial lung disease clinic, aiming to identify undiagnosed APAP patients [21] Q&A Session Summary Question: What is the current status of the regulatory submission for Molbrevi? - The company completed the submission of the BLA to the FDA and is awaiting feedback within a 60-day window [11] Question: How many patients does the company aim to reach by launch? - The company aims to have line of sight to 1,000 known APAP patients by launch, with a goal to confirm the total of 3,600 patients [15][17] Question: What are the expectations regarding payer coverage for Molbrevi? - 87% of payers indicated they intend to provide coverage with simple pre-authorization criteria, recognizing the disease burden of APAP [19]

Financial Performance - Savara reported a net loss of $26.6 million for the three months ended March 31, 2025, compared to a net loss of $20.3 million for the same period in 2024, representing an increase of 30.9%[93]. - The total operating expenses for the three months ended March 31, 2025, were $28.4 million, compared to $22.5 million for the same period in 2024, reflecting an increase of $5.9 million[108]. - Cash used in operating activities for Q1 2025 was $27.2 million, an increase from $20.6 million in Q1 2024[116]. - Other income decreased by $0.3 million to $1.8 million for the three months ended March 31, 2025, primarily due to a loss on extinguishment of debt[112]. - The company expects to incur significant additional expenses and continue operating losses for at least the next several years as it advances the clinical development of MOLBREEVI[94]. Research and Development - Research and development expenses increased by $2.4 million, or 14.0%, to $19.2 million for the three months ended March 31, 2025, primarily due to activities related to the MOLBREEVI program[110]. - The company has not generated any revenue from product sales and does not expect to until regulatory approval is obtained[120]. Cash and Investments - As of March 31, 2025, Savara had cash and cash equivalents of $19.6 million and short-term investments of $152.9 million, with an accumulated deficit of approximately $515.9 million[93][113]. - As of March 31, 2025, the company had cash, cash equivalents, and short-term investments totaling approximately $172.5 million[121]. - Cash provided by investing activities was $29.3 million in Q1 2025, compared to $10.8 million in Q1 2024[118]. - Cash provided by financing activities was $2.3 million in Q1 2025, primarily from net proceeds of the Hercules Loan Agreement[119]. Financing Activities - Savara entered into a Hercules Loan Agreement on March 26, 2025, allowing for borrowing up to $200 million, with an initial advance of $30 million used to repay previous obligations[98][113]. - The Hercules Loan Agreement bears interest at 8.95% as of March 31, 2025, with a potential impact from changes in the prime rate[125]. - Future funding requirements may necessitate raising additional capital to support ongoing development and commercialization efforts[122]. - Savara has raised approximately $597.9 million in net cash proceeds since inception, primarily from stock offerings and debt financings[92]. Operational Impact - General and administrative expenses rose by $3.6 million, or 64.1%, to $9.2 million for the three months ended March 31, 2025, driven by personnel costs and commercial activities[111]. - The company has not recognized significant exchange rate losses during Q1 2025 and does not anticipate material effects from currency fluctuations[126]. - Inflation has not materially affected the company's results of operations during the periods presented[127]. Regulatory Developments - The company completed its submission of the Biological License Application (BLA) to the FDA for MOLBREEVI on March 26, 2025, following positive results from the Phase 3 clinical trial[97].

Savara Inc (SVRA) 2025 Conference Summary Company Overview - Savara Inc is a rare orphan respiratory pulmonology-focused company with a single asset, a novel inhaled biologic called molgermostim inhalation solution [4][5] - The company is actively pursuing FDA approval for autoimmune pulmonary alveolar proteinosis (aPAP) and plans to file for MAA in Europe and the UK by the end of the year [5][11] Disease Background - Autoimmune PAP is a chronic rare autoimmune disease characterized by the development of autoantibodies to GM-CSF, leading to impaired gas exchange in the lungs [8][10] - The disease manifests with symptoms such as cough, shortness of breath, and fatigue [10] Patient Population - Estimated prevalence of aPAP ranges from 6-26 per million, with approximately 3,600 diagnosed patients in the U.S. [11][12] - The company believes there are many undiagnosed patients, indicating a robust addressable market for commercialization [12][13] Clinical Program and Results - The IMPALA 2 trial is the largest and longest randomized clinical trial for aPAP, enrolling 64 patients across 43 centers in 16 countries [22] - Primary endpoint was the change in DLCO (diffusion capacity of the lung for carbon monoxide) at 24 weeks, which was statistically significant [23][24] - The trial also showed significant clinical benefits, including improvements in respiratory quality of life and exercise capacity [26][28] Regulatory Process - Savara completed a rolling BLA submission in March, with a 60-day review period expected to conclude in May [32] - The company anticipates priority review due to breakthrough designation, with a potential PDUFA date in November [32][33] Market Preparation and Payer Research - Current treatments for aPAP include lung lavage, which is a rescue procedure, and off-label drugs [40] - Savara is guiding a price range of $300,000 to $500,000 per patient per year, with 87% of payers indicating they would cover it within this range [42] - The company expects 50-70% commercial coverage for patients affected by aPAP [43] Launch Strategy - Savara is implementing a dry blood spot antibody testing program and has initiated a pilot clinic at the University of Florida to identify undiagnosed patients [46][48] - The company aims to educate pulmonologists about aPAP and the availability of testing to facilitate earlier diagnosis [49] Financial Position - As of the last report, Savara has $196 million in cash and has structured a non-dilutive financing option of up to $170 million [50] - The current cash position is expected to last through the second quarter of 2027, with various options available for additional funding [51] Key Takeaways - Savara is on track for potential FDA approval of molgermostim for aPAP, with significant clinical trial success and a robust market strategy [53] - The company is preparing for a launch that could significantly impact the treatment landscape for aPAP, a rare and underserved patient population [53]