Acquisition and Partnerships - The company acquired Lowell Therapeutics, Inc. for approximately $32.5 million, with up to $26.0 million in contingent consideration based on regulatory and sales milestones[25]. - The divestment of DSUVIA to Alora Pharmaceuticals in April 2023 includes royalties and sales-based milestones, while the company continues to market DSUVIA to the Department of Defense[43]. - The company has entered into an agreement with XOMA to sell rights to payments related to DSUVIA sales, providing funding for Niyad's development[44]. Product Development and Regulatory Approvals - Niyad™ has received an Investigational Device Exemption (IDE) and Breakthrough Device Designation from the FDA, with an ongoing registrational trial[26]. - The NEPHRO CRRT Study aims to enroll 70 adult patients and evaluate the efficacy of Niyad compared to placebo, focusing on mean post-filter activated clotting time[28]. - LTX-608 is being explored for indications including antiviral treatment and acute respiratory distress syndrome (ARDS), with initial studies showing potential benefits in COVID patients[29]. - The company is focusing on the registrational trial for Niyad while deprioritizing the development of pre-filled syringe product candidates due to recent FDA approvals of competing products[36]. - Niyad, a nafamostat product, is classified as a Class III medical device and is undergoing the PMA application process, which is generally more costly and time-consuming than the 510(k) process[64]. - The FDA has 180 days to review a filed PMA application, although the review often takes significantly longer, typically between one and three years[67][68]. - The Breakthrough Devices Program allows for expedited review of devices that provide significant advantages over existing treatments, but does not change the standards for approval[71]. - Post-approval requirements include conducting Phase 4 clinical trials to gain additional experience and ensuring compliance with cGMP requirements[61]. - The FDA may withdraw product approval if compliance with regulatory standards is not maintained, which could lead to severe consequences including product recalls[63]. - Manufacturers must establish validated systems to ensure products meet specifications and regulatory standards, and test each product batch prior to release[62]. - The FDA may require post-approval studies or surveillance to monitor the safety and effectiveness of devices after market release[70]. - The approval process for an NDA is lengthy, and the FDA may issue a Complete Response Letter if the submission does not meet approval criteria[59]. Financial Performance and Concerns - The company has not yet generated significant product revenue and anticipates continued losses in the future[17]. - There is substantial doubt regarding the company's ability to continue as a going concern without raising additional capital[17]. - The market price of the company's common stock has historically been highly volatile, which may affect investor confidence and capital raising efforts[19]. - The company faces significant penalties for violations of health regulatory laws, which could adversely affect operations and financial results[88]. - Sales of product candidates depend on third-party payer coverage and reimbursement, which is increasingly challenging due to cost containment efforts[89]. - Legislative changes, such as the Affordable Care Act and the Inflation Reduction Act, may impact the company's ability to commercialize products profitably[94]. - The company anticipates ongoing pressure to provide larger discounts or rebates to maintain acceptable reimbursement levels[89]. - The pricing of prescription drugs in foreign markets is subject to government control, which may affect profitability[91]. - Future healthcare reforms could limit or eliminate spending on development projects, affecting profitability[93]. - The company may need to conduct expensive pharmacoeconomic studies to demonstrate cost-effectiveness for product approval[89]. Compliance and Legal Matters - Compliance with federal and state healthcare laws, including the Anti-Kickback Statute, is critical to avoid legal scrutiny and potential penalties[78]. - The company is subject to stringent FDA regulations, including quality assurance procedures and medical device reporting regulations[79]. - The company is subject to ongoing legal challenges regarding the Medicare Drug Price Negotiation Program[97]. - There is increasing legislative interest in drug pricing practices, including the Medicare Drug Price Negotiation Program, which began in fiscal year 2023[97]. - The company may face adverse impacts from potential future healthcare reforms that could limit product pricing and reimbursement[98]. Intellectual Property and Market Potential - The company has filed an international patent application for Niyad's use as an anticoagulant, which could provide six years of data exclusivity upon FDA approval[27]. - The company is seeking patent protection for its product candidates, with recent filings expected to extend patent coverage into 2045[47]. - The peak sales potential for Niyad could exceed $200 million annually in the U.S., based on an addressable population of 500,000 patients undergoing CRRT and 350,000 patients undergoing IHD, representing a total market opportunity of $4.075 billion[32]. - Niyad would be the first FDA-approved product for regional anticoagulation in the extracorporeal circuit, targeting the 57% of the market currently using citrate or no anticoagulant[34]. Operational and Employee Matters - The company relies on third-party manufacturers and suppliers for its product candidates, which may pose risks to development timelines[17]. - The company relies on contract manufacturers for the production of Niyad, ensuring a backup manufacturer to avoid supply chain risks[41]. - As of December 31, 2024, the company employed 13 full-time employees, with approximately 85% based in San Mateo, CA[99]. - The company is committed to pay equity and conducts annual pay equity analyses[99]. - The company offers competitive salaries, stock options, and comprehensive benefits to foster employee ownership and development[100]. - There are no collective bargaining agreements in place, and the company maintains a good relationship with its employees[101]. Company Background - The company was incorporated as SuRx, Inc. in 2005 and changed its name to Talphera, Inc. in January 2024[102]. - The company files annual and quarterly reports with the SEC, making them available on its website[102]. - The company is classified as a smaller reporting company and is not required to provide certain financial disclosures[321].

Financial Data and Key Metrics Changes - The combined R&D and SG&A expenses for Q4 2024 totaled $3 million, a decrease from $4.6 million in Q4 2023 [49] - Excluding non-cash, stock-based compensation, these amounts were $2.8 million for Q4 2024, compared to $4.3 million for Q4 2023 [50] - Cash operating expenses for 2025 are expected to be in the range of $18-19 million [50] Business Line Data and Key Metrics Changes - The NEPHRO CRRT study has been reduced in size from 166 patients to 70, maintaining a primary endpoint power of 90% [13][30] - The company has added three new clinical study sites, bringing the total to eight enrollment-ready sites [22][29] - The most productive current site has about 20 CRRT patients per month, while new sites are expected to have higher volumes [42][101] Market Data and Key Metrics Changes - The company anticipates a private placement investment of up to $14.8 million, contingent on achieving certain milestones [9] - The financing is structured in three equal tranches of $4.925 million, with the first tranche expected shortly [47] Company Strategy and Development Direction - The company is focused on completing the NEPHRO study by the end of 2025, with a strategic emphasis on reducing operating expenses [46] - The FDA has approved changes to the NEPHRO study protocol, which are expected to enhance enrollment speed and site efficiency [15][28] - The company is actively pursuing additional clinical study sites to meet its enrollment goals [43] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the timely development of NIAID and the planned submission of a PMA in Q1 of the following year [8] - The FDA's responsiveness has been highlighted as a key benefit of NIAID's breakthrough designation [14] - Management believes that clinical, regulatory, and commercial risks for the NEPHRO program are low due to extensive prior use of the product in other markets [24] Other Important Information - The company has identified an ideal clinical site profile based on ICU type and nephrologist involvement, which is expected to improve enrollment rates [39][41] - The company has experienced a much shorter activation time for new sites, with the most recent site taking less than four months to activate [42] Q&A Session Summary Question: What were the original thoughts on the movement to exclude certain patients in the previous year? - The original protocol likely excluded patients based on concerns about clinical endpoints related to time on CRRT, which was deemed irrelevant for the current study [60][62] Question: Why have institutions decided not to use heparin? - Institutions cited risks associated with heparin, including its long half-life and systemic anticoagulation effects, which increase bleeding risks in high-risk patients [70][72] Question: Is there a time limit tied to the milestones for the private placement? - There is no specific time limit tied to the milestones for patient enrollment [76] Question: Are there plans to shift PIs to nephrologists at existing sites? - Discussions are ongoing to shift primary responsibilities to nephrologists or broaden patient populations at existing sites [91][92] Question: What gives confidence that phase three data can be achieved by the end of the year? - The company has already engaged with additional sites and believes that the enrollment of patients can be accelerated based on the new site profiles and engagement [112][113]

Financial Performance - The net loss from continuing operations for Q4 2024 was $1.9 million, compared to a net loss of $4.5 million in Q4 2023, reflecting improved financial performance[10] - The net loss attributable to common shareholders for Q4 2024 was $1.9 million, or $0.07 per share, compared to a net loss of $4.5 million, or $0.25 per share, in Q4 2023[10] - Talphera reported cash and investments of $8.9 million as of December 31, 2024, and announced a private placement financing of up to $14.8 million[10] - The company recognized no revenue for Q4 2024, consistent with Q4 2023, while total revenue for the year was $0 compared to $651,000 in 2023[18] Expenses and Cost Management - Combined R&D and SG&A expenses for Q4 2024 totaled $3.0 million, down from $4.6 million in Q4 2023, primarily due to reductions in personnel and administrative expenses[10] - Talphera expects cash operating expenses in 2025 to be between $18 million and $19 million, including costs related to the NEPHRO CRRT trial[7] - Research and development expenses for Q4 2024 were $1.323 billion, a decrease from $1.769 billion in Q4 2023, representing a decline of about 25.2%[21] - Selling, general and administrative expenses for Q4 2024 were $1.673 billion, down from $2.795 billion in Q4 2023, reflecting a decrease of approximately 40.2%[21] - Total operating expenses for Q4 2024 were $2.996 billion, a decrease from $4.564 billion in Q4 2023, indicating a decline of about 34.4%[21] - Non-GAAP operating expenses for the year ended December 31, 2024, were $14.263 billion, down from $15.830 billion in 2023, representing a decrease of approximately 9.9%[21] - Stock-based compensation expense for the year ended December 31, 2024, was $989 million, down from $1.710 billion in 2023, indicating a decrease of about 42.0%[21] - The company is focusing on cost reduction strategies, as evidenced by the significant decrease in both operating expenses and stock-based compensation[21] Assets and Liabilities - Cash, cash equivalents, and investments decreased to $8.863 billion in 2024 from $9.381 billion in 2023, representing a decline of approximately 5.5%[20] - Total assets decreased to $18.236 billion in 2024, down from $20.395 billion in 2023, reflecting a decrease of about 10.6%[20] - Total liabilities increased significantly to $10.235 billion in 2024, compared to $6.290 billion in 2023, marking an increase of approximately 62.5%[20] - Total stockholders' equity fell to $8.001 billion in 2024, down from $14.105 billion in 2023, indicating a decrease of around 43.4%[20] Clinical Trials and Product Development - The FDA approved a reduction in the NEPHRO CRRT study size from 166 to 70 patients, with a primary endpoint powered at 90%[4] - The company plans to complete the NEPHRO CRRT study by the end of 2025, supported by new site additions and increased enrollment potential[3] - Talphera's lead product candidate, Niyad™, is being studied as an anticoagulant and has received Breakthrough Device Designation from the FDA[11]

The U.S. Food and Drug Administration (FDA) agreed to reduce the number of patients in the NEPHRO CRRT study from 166 to 70 Talphera expects the registrational NEPHRO CRRT study to be completed by the end of 2025 Cash and investments at December 31, 2024 of $8.9 million, together with the private placement financing recently announced of up to $14.8 million, expected to provide capital through target completion of the NEPHRO study in the fourth quarter of 2025 Conference call and webcast to be held Monday, ...

The U.S. Food and Drug Administration (FDA) agreed to reduce the number of patients in the NEPHRO CRRT study from 166 to 70 patients, of which 6 patients are already enrolled Talphera believes the NEPHRO CRRT study should be completed by the end of 2025 Existing investors, Nantahala Capital and Rosalind Advisors led the capital commitment priced at-the-market as defined by Nasdaq rules $4.925 million in gross proceeds at first closing with the potential to receive an additional $9.85 million across two $4.9 ...

Core Viewpoint - Talphera, Inc. is set to release its fourth quarter and full-year 2024 financial results on March 31, 2025, followed by a live webcast and conference call to discuss the results and provide a business update [1]. Group 1: Financial Results Announcement - The financial results will be released after market close on March 31, 2025 [1]. - A live webcast and conference call will take place at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Standard Time [1]. Group 2: Webcast and Conference Call Details - The webcast can be accessed through the Investors section of Talphera's website, with a slide presentation included [2]. - A replay of the webcast will be available for 90 days following the event [2]. - Investors can participate in the conference call by dialing specific numbers for North American and international callers, with a conference ID provided [3]. Group 3: Company Overview - Talphera, Inc. focuses on developing and commercializing innovative therapies for medically supervised settings [4]. - The lead product candidate, Niyad™, is a lyophilized formulation of nafamostat, currently studied as an anticoagulant and has received Breakthrough Device Designation from the FDA [4].

Core Viewpoint - Talphera, Inc. is advancing its NEPHRO CRRT clinical study with FDA's agreement on protocol changes aimed at accelerating patient enrollment and reducing the number of patients required for the study [1][2][3] Group 1: FDA Interactions and Protocol Changes - The FDA has agreed to review a Prior Approval Supplement (PAS) for a reduction in the number of patients in the NEPHRO CRRT study, which is expected to be submitted within the coming week [1] - Two additional protocol changes have been approved by the FDA to broaden inclusion criteria, allowing enrollment of patients on continuous renal replacement therapy (CRRT) beyond 48 hours and heparin-tolerant patients at certain institutions [2] - The PAS review by the FDA will take up to 30 days, and the company is optimistic about completing the study by the end of the year [3] Group 2: Product and Study Details - Talphera's lead product candidate, Niyad™, is a lyophilized formulation of nafamostat, currently under investigation as an anticoagulant for the extracorporeal circuit and has received Breakthrough Device Designation from the FDA [4][6] - The NEPHRO CRRT study aims to enroll 166 adult patients undergoing renal replacement therapy who cannot tolerate heparin or are at risk for bleeding, with primary and secondary endpoints focused on clotting time and dialysis efficacy [7]

Financial Data and Key Metrics Changes - Cash operating expenses for Q3 2024 totaled $3.5 million, an increase from $3 million in the same period last year, attributed to the NEPHRO CRRT clinical study [20] - Year-to-date cash operating expenses reached $11.5 million, with full-year 2024 expenses expected to be in the range of $15 million to $17 million [21] - Cash and investments at the end of Q3 2024 amounted to $11.1 million, with an additional capital injection likely required before the completion of the NEPHRO study [21] Business Line Data and Key Metrics Changes - The NEPHRO CRRT study has onboarded five active clinical trial sites, with two additional sites expected to begin screening, totaling seven by the end of the year [7][8] - The primary endpoint of the NEPHRO trial is the mean activated clotting time measured over the first 24 hours, with patient participation lasting 72 hours [9] Market Data and Key Metrics Changes - The FDA has expanded the maximum number of trial sites from 10 to 14, indicating a proactive approach to enhance patient enrollment [15] - The company is focusing on high-volume CRRT sites with broader ICU populations to optimize patient recruitment [13] Company Strategy and Development Direction - The company is committed to improving enrollment rates at existing sites and onboarding new productive sites to expedite the NEPHRO trial [8][14] - There is a strong emphasis on leveraging breakthrough designation from the FDA to facilitate study enrollment and completion [18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in nafamostat's potential clinical utility, citing a long track record of efficacy and safety in Japan and South Korea [17] - The company anticipates having more information on the clinical trial's progress early next year, indicating a positive outlook for future developments [16] Other Important Information - The company is consulting regulatory experts to implement potential changes to study design elements to facilitate enrollment [14] - Management highlighted the need for an alternative anticoagulant in CRRT, reinforcing the market opportunity for nafamostat [15] Q&A Session Summary Question: Can you walk through the optimization on the screening to drive enrollment? - Management discussed the need for improved site engagement and automated notifications to capture patients before CRRT begins, addressing previous enrollment challenges [26][27] Question: What percentage of patients are screened and successfully enrolled? - Management noted that screening percentages fluctuate by site, and feedback from physicians has been positive regarding patient experiences in the trial [40][41] Question: Will data from the NEPHRO study be available next year? - Management confirmed that the goal is to have data available next year, with a short timeline from study completion to PMA submission [44] Question: What measures are planned to accelerate enrollment? - Management mentioned ongoing evaluations of study design and the implementation of new site strategies to enhance patient identification and enrollment [46][47]

Financial Performance - Total revenue for the three months ended September 30, 2024, was $117,000, compared to $370,000 for the same period in 2023, representing a decrease of approximately 68.4%[13] - The net loss for the three months ended September 30, 2024, was $3,353,000, compared to a net loss of $1,357,000 for the same period in 2023, indicating a worsening of 146.5%[13] - Basic and diluted loss per share for continuing operations was $(0.13) for the three months ended September 30, 2024, compared to $(0.08) for the same period in 2023[13] - As of September 30, 2023, the company reported a net loss of $1,357,000, with total equity amounting to $18,307,000[15] - For the nine months ended September 30, 2023, the net loss was $13,880,000, with cash used in operating activities totaling $10,429,000[16] - The company recognized a net loss from discontinued operations of $8.098 million for the nine months ended September 30, 2023, with total revenues of $501,000 during the same period[47] Expenses and Costs - Research and development expenses for the nine months ended September 30, 2024, were $5,395,000, an increase of 42.8% from $3,777,000 for the same period in 2023[13] - Total operating costs and expenses for the three months ended September 30, 2024, were $3,749,000, compared to $3,426,000 for the same period in 2023, reflecting an increase of 9.4%[13] - The Company recorded total stock-based compensation expense of $234,000 for the three months ended September 30, 2024, compared to $378,000 for the same period in 2023[79] Equity and Liabilities - Total stockholders' equity decreased to $9,641,000 as of September 30, 2024, down from $14,105,000 as of December 31, 2023[10] - The accumulated deficit increased to $(455,360,000) as of September 30, 2024, compared to $(444,226,000) as of December 31, 2023[10] - The company reported total liabilities and stockholders' equity of $21,014,000 as of September 30, 2024, compared to $20,395,000 as of December 31, 2023[10] Cash and Financing Activities - The company had cash and cash equivalents of $11,117,000 at the end of the reporting period, down from $20,275,000 at the beginning of the period[16] - The company raised $8,924,000 from a private placement offering in July 2023, contributing to net cash provided by financing activities of $12,044,000 for the nine months ended September 30, 2023[16] - The Company entered into a private placement on January 17, 2024, resulting in aggregate gross proceeds of $6.0 million upfront, with an additional $10.0 million contingent upon positive clinical trial results for the NEPHRO CRRT study of Niyad[65] - The first tranche of the private placement closed on January 22, 2024, generating approximately $6.0 million for pre-funded warrants to purchase up to 7,792,208 shares of common stock[66] Product Development and Strategy - The company is focused on advancing the development of its lead product candidate, Niyad™, and aims to secure regulatory approval and commercialization[6] - The company is focused on developing Niyad™, a regional anticoagulant, and LTX-608, a nafamostat formulation, for various medical indications[18][21] - The company completed an asset acquisition agreement for the DSUVIA product on April 3, 2023, which included the transfer of certain assets and liabilities[20] Risks and Concerns - The company faces significant risks including market volatility, supply chain disruptions, and the ability to obtain necessary financing for ongoing operations[6] - The company has incurred recurring operating losses and negative cash flows since inception, raising concerns about its ability to continue as a going concern[23] - The Company may need to reduce its workforce or delay clinical trials if adequate funds are not available[25] Accounting and Financial Reporting - The Company is evaluating the impact of recently issued accounting standards but does not expect them to materially affect its consolidated financial statements[36] - Management's significant accounting estimates include fair value of warrants and impairment of long-lived assets, which could differ from actual results[28] - The effective interest rate for the liability related to the sale of future payments will be reassessed at each reporting date[33] Stock and Equity Compensation - The Company amended its 2020 Equity Incentive Plan to increase the number of authorized shares by 1,171,395, bringing the total to 3,161,395 shares[72] - The Company granted stock-based awards totaling 217,000 shares to a new employee as an inducement for employment in May 2024[73] - The Company amended its 2011 Employee Stock Purchase Plan to increase the number of authorized shares by 100,000, resulting in a total of 345,000 shares available for issuance[74] - As of September 30, 2024, the total stock options outstanding increased to 1,879,806, with a weighted average remaining contractual life of 7.7 years[81] Litigation - The Company believes ongoing litigation is without merit and intends to defend against it, but cannot estimate potential losses due to the uncertainty of litigation outcomes[64]

Financial Performance - Net loss from continuing operations for Q3 2024 was $3.4 million, compared to a net loss of $1.4 million in Q3 2023[7]. - Net loss attributable to common shareholders for Q3 2024 was $3.4 million, or $0.13 per share, compared to a net loss of $1.4 million, or $0.08 per share, in Q3 2023[8]. - Total operating costs and expenses for Q3 2024 were $3.7 million, compared to $3.4 million in Q3 2023[6]. - Total operating expenses for the three months ended September 2024 were $3,749,000, slightly up from $3,426,000, an increase of 9.4%[21]. - Non-GAAP operating expenses for the three months ended September 2024 were $3,515,000, compared to $3,048,000 for the same period in 2023, an increase of 15.3%[21]. - Total operating expenses for the nine months ended September 2024 were $12,256,000, slightly down from $12,976,000 for the same period in 2023, a decrease of 5.5%[21]. Cash and Investments - Cash and investments totaled $11.1 million as of September 30, 2024[5]. - Cash, cash equivalents, and investments increased to $11,117,000 from $9,381,000, representing a growth of 18.5%[20]. - Total assets rose to $21,014,000, up from $20,395,000, indicating an increase of 3.0%[20]. - Total liabilities increased significantly to $11,373,000 from $6,290,000, reflecting an increase of 80.5%[20]. - Total stockholders' equity decreased to $9,641,000 from $14,105,000, a decline of 31.7%[20]. Research and Development - Combined R&D and SG&A expenses for Q3 2024 were $3.7 million, up from $3.4 million in Q3 2023, primarily due to NEPHRO study costs[6]. - Research and development expenses for the three months ended September 2024 were $2,053,000, up 74.4% from $1,178,000 for the same period in 2023[21]. - The NEPHRO CRRT study aims to enroll 166 patients across up to 14 clinical sites in the U.S.[4]. - The first patient was enrolled in the NEPHRO CRRT study in August 2024[3]. - The study's primary endpoint is the mean post-filter activated clotting time for circuits infused with nafamostat compared to placebo over the first 24 hours[4]. - Talphera anticipates completing the NEPHRO study by the end of 2025 with improved enrollment rates[2]. Operating Expenses - Selling, general, and administrative expenses decreased to $1,696,000 from $2,248,000, a reduction of 24.5%[21]. - Stock-based compensation expense for the three months ended September 2024 was $234,000, down from $378,000, a decrease of 38.3%[21]. Regulatory Status - The company has received Breakthrough Device Designation status from the FDA for its lead product candidate, Niyad™[11].