Acquisition and Product Development - 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[17]. - Niyad™, a product candidate, has received FDA's Breakthrough Device Designation and is expected to begin patient enrollment in a registrational trial in Q1 2024, with a PMA application planned by the end of 2024[20][23]. - The NEPHRO CRRT Study will enroll 166 adult patients and aims to evaluate the efficacy of Niyad compared to placebo, with top-line data expected by Q3 2024[23]. - LTX-608, another product candidate, is being explored for indications such as antiviral treatment and acute respiratory distress syndrome, with initial studies showing potential benefits in COVID patients[30]. - The company plans to submit a PMA application for Niyad by the end of 2024 and expects to enroll the first patient in its registrational study in Q1 2024[38]. - The company is evaluating the second targeted indication for its nafamostat product development candidate, LTX-608, focusing on DIC and acute pancreatitis for initial development[50]. Financial Performance and Capital Needs - The company has not yet generated significant product revenue and anticipates continued losses in the future[9]. - The company has incurred significant losses since inception and may need to raise additional capital, which could dilute existing shareholders[9]. - The company will need to raise additional funds through equity sales, asset monetization, or debt issuance to sustain operations and development programs[338]. Regulatory and Approval Processes - The FDA approval process for product candidates is time-consuming and may involve unexpected delays and costs[9]. - Niyad is classified as a Class III medical device and is subject to the PMA application process, which is generally more costly and time-consuming than the 510(k) process[69]. - The FDA has 180 days to review a filed PMA application, although the review often takes significantly longer, and may request additional information during this period[72]. - The approval process for drug products requires substantial time and financial resources, and the company cannot be certain that approval will be granted on a timely basis[57]. - The Breakthrough Devices Program allows for expedited review of medical devices that address life-threatening conditions, with priority review for designated devices[76]. Market Potential and Sales Projections - The peak sales potential for Niyad, if approved for use in CRRT and IHD, may exceed $200 million annually in the United States, based on an estimated addressable population of 500,000 patients undergoing CRRT and 350,000 patients undergoing IHD[35]. - The company believes its two pre-filled syringe product candidates could have a peak sales potential of over $100 million, with a focus on eliminating the need for on-the-spot calculations and additional dilution steps[37]. - Nafamostat, if approved for regional anticoagulation in CRRT, may address safety concerns associated with current anticoagulants, as 29% of cases do not use any anticoagulant due to safety concerns[33]. - The incidence of bleeding during continuous hemofiltration with heparin was 66.7%, compared to only 4.3% with nafamostat, indicating a significant safety advantage[34]. Manufacturing and Supply Chain Risks - The company relies on third-party manufacturers and suppliers for its product candidates, which poses risks to the supply chain[10]. - The company relies on contract manufacturers for the production of its Niyad product, with a single contract manufacturer currently producing the nafamostat API and a separate one for the finished product[52]. - The company is in discussions with a back-up manufacturer for Niyad to ensure there is not a single source of supply[52]. - Unforeseen delays in drug manufacturing and supply chains may arise from regulatory compliance issues, potentially impacting product availability[81]. Compliance and Regulatory Challenges - The company must comply with extensive cGMP requirements throughout the manufacturing process, which require considerable ongoing investment[66]. - Manufacturers must comply with stringent FDA regulations, including quality assurance procedures and medical device reporting regulations[77]. - The Drug Supply Chain Security Act mandates tracking and tracing obligations for pharmaceutical manufacturers, including reporting counterfeit products[80]. - The company faces significant penalties for violations of health regulatory laws, which could adversely affect operations and financial results[91]. Market and Legislative Environment - Sales of approved products depend on third-party payer coverage and reimbursement, which are increasingly scrutinized for cost-effectiveness[92]. - The company may need to conduct pharmacoeconomic studies to demonstrate the cost-effectiveness of products for reimbursement approval[92]. - The company anticipates ongoing legislative and regulatory changes in the healthcare system that may impact product commercialization and profitability[93]. - In the U.S., the Affordable Care Act and subsequent reforms aim to broaden insurance access and reduce healthcare spending, with potential implications for drug pricing and reimbursement rates[98]. - The Inflation Reduction Act of 2022 extends enhanced subsidies for health insurance coverage through 2025 and introduces measures to lower out-of-pocket costs for Medicare beneficiaries[98]. - The company faces competition from lower-priced products in foreign markets due to varying drug pricing regulations, particularly in the European Union[95]. - Legislative proposals are being made to increase transparency in drug pricing and reform reimbursement methodologies, which could affect the company's pricing strategies[102]. Workforce and Employment - As of December 31, 2023, the company employed 15 full-time employees, with a commitment to pay equity and competitive benefits[104]. - The company is not currently required to provide certain financial disclosures as a smaller reporting company under the Securities Exchange Act[339].

Core Viewpoint - AcelRx Pharmaceuticals, Inc. has rebranded to Talphera, Inc. to reflect its new mission of developing innovative therapies for medically supervised settings, moving beyond its original focus on acute pain [1][2]. Company Overview - Talphera, Inc. is a specialty pharmaceutical company focused on innovative therapies for medically supervised settings, with its lead product candidate being Niyad, a formulation of nafamostat [6][7]. - The name "Talphera" is derived from "Talisman," symbolizing a strong leader and a new pharmaceutical era for the company [2]. Product Development - Talphera's lead product candidate, Niyad, has received FDA Breakthrough Designation and is being studied as an anticoagulant for the extracorporeal circuit [3][6]. - The company plans to initiate the NEPHRO CRRT (Nafamostat Efficacy in Phase 3 Registrational Continuous Renal Replacement Therapy) Study, which has received IRB approval and will evaluate 166 adult patients [4][5]. Regulatory and Market Strategy - A PMA submission for Niyad is expected to be filed with the FDA in the second half of 2024 [5]. - The first patient for the NEPHRO study is anticipated to be enrolled in the first quarter of 2024 [5].

Financial Performance - Total current assets decreased from $24,566,000 as of December 31, 2022 to $14,420,000 as of September 30, 2023, representing a decline of approximately 41.3%[22] - Royalty revenue for the three months ended September 30, 2023 was $117,000, compared to $0 for the same period in 2022, indicating a significant increase[25] - Research and development expenses increased to $1,178,000 for the three months ended September 30, 2023, up from $799,000 in the same period of 2022, reflecting a rise of approximately 47.5%[25] - Selling, general and administrative expenses decreased from $3,724,000 in Q3 2022 to $2,248,000 in Q3 2023, a reduction of about 39.6%[25] - The net loss attributable to common shareholders for the three months ended September 30, 2023 was $1,357,000, compared to a net loss of $6,936,000 for the same period in 2022, showing an improvement of approximately 80.5%[25] - The company reported a net income from discontinued operations of $61,000 for the three months ended September 30, 2023, compared to a loss of $2,111,000 in the same period in 2022[25] - The total stockholders' equity decreased from $21,814,000 as of December 31, 2022 to $18,307,000 as of September 30, 2023, a decline of approximately 16.5%[22] - The company incurred a net loss of $13,880,000 for the nine months ended September 30, 2023, compared to a net income of $55,239,000 in the same period of the previous year[33] - The company reported a balance of 10,994,102 shares as of June 30, 2023, increasing to 16,952,269 shares by September 30, 2023[30] - The total stock-based compensation expense for the nine months ended September 30, 2023, was $1.418 million, compared to $2.237 million for the same period in 2022[106] - The net loss attributable to common shareholders for the three months ended September 30, 2023, was $1.357 million, resulting in a basic net loss per share of $0.08[115] - For the nine months ended September 30, 2023, the net loss attributable to common shareholders was $13,880,000, compared to a net income of $49,073,000 for the same period in 2022[116] - The basic net income (loss) per share from continuing operations was $(0.45) for 2023, down from $8.03 in 2022[116] Assets and Liabilities - Total liabilities decreased from $25,673,000 as of December 31, 2022 to $4,954,000 as of September 30, 2023, a reduction of approximately 80.7%[22] - As of September 30, 2023, total cash and cash equivalents amounted to $13,389,000, a decrease from $20,770,000 as of December 31, 2022[58] - The accrued balance due under the Loan Agreement with Oxford was $0, down from $5.4 million at the end of 2022[85] - The total liabilities of discontinued operations as of September 30, 2023, were $8,615,000, compared to $7,252,000 in net assets[78] - As of September 30, 2023, AcelRx had 21,682,049 outstanding warrants with a weighted average exercise price of $1.40 per share[97] - The fair value of the warrant liability was estimated at $1,380,000, down from $7,098,000 as of December 31, 2022[61] Cash Flow and Financing - The company reported a net cash used in operating activities of $13,543,000 for the three months ended September 30, 2023[33] - Cash provided by investing activities was $3.1 million for the nine months ended September 30, 2023, primarily from the sale of DSUVIA to Alora[188] - Cash provided by financing activities was $3.5 million for the nine months ended September 30, 2023, mainly due to $8.9 million in net proceeds from equity financing[189] - The company raised $10.0 million in July 2023 through a private placement of common stock and warrants, with an additional potential $16.3 million upon the exercise of common warrants[41] - The company closed a private placement of common stock on July 20, 2023, raising gross proceeds of $10.0 million, with an additional potential $16.3 million upon the exercise of common warrants[150] - The company has a liquidity concern, expecting to need additional capital to fund operations within the next twelve months[41] - The company expects to need additional capital to fund operations for at least the next twelve months due to incurred losses and negative cash flows[177] - Current capital levels are insufficient to fund operations for the next twelve months without raising additional capital[212] Business Operations and Strategy - The company is focused on advancing the development of its product candidates, including Niyad™, and aims to secure regulatory approval for commercialization[16] - The company has ongoing development for Niyad™, a regional anticoagulant, which has received Breakthrough Device Designation status from the FDA[39] - The company plans to commercialize an ephedrine pre-filled syringe and a phenylephrine pre-filled syringe, pending FDA approval[40] - The company is preparing New Drug Applications for its ephedrine and phenylephrine pre-filled syringe product candidates, which are already approved in the European Union[134][136] - The company plans to initiate a registrational trial for its product candidate Niyad in 2023 and submit a Premarket Approval application to the FDA in the second half of 2024[121] - The company has pending patent applications for nafamostat in various indications, including ARDS and DIC, which are already approved in Japan and South Korea[137] - The company has no plans to further develop any sufentanil sublingual product candidates following the divestment of DSUVIA[143] - The company is focused on reducing operating costs while maximizing the value of its remaining product candidates[143] Risks and Compliance - The company has identified risks including the ability to manage operating costs and reduce cash burn, which could impact future performance[16] - The company faces risks related to delays in clinical trials, which could increase costs and jeopardize regulatory approvals[205] - The company is dependent on the successful commercialization of DSUVIA by Alora Pharmaceuticals to receive royalties, which poses a risk to financial performance[205] - A material weakness in internal control over financial reporting was previously identified, leading to a restatement of financial statements for certain periods[198] - The company has implemented remediation measures to address the identified material weakness, resulting in an improved internal control environment as of June 30, 2023[200] - The company received a notice from Nasdaq regarding non-compliance with the Minimum Bid Price Rule, which may affect future capital raising efforts[177] Discontinued Operations - The DSUVIA business was classified as discontinued operations as of March 31, 2023, affecting the financial statements for all periods presented[58] - The company closed a transaction with Alora Pharmaceuticals on April 3, 2023, for the divestment of its DSUVIA product, which includes a 15% royalty on net product sales[139] - The impairment of net assets held for sale was $6,853,000 for the nine months ended September 30, 2023, compared to $0 in 2022[72] - The net loss from discontinued operations for the nine months ended September 30, 2023, was $8,098,000, an improvement from a loss of $9,822,000 in 2022[72] - The company recognized a net income from discontinued operations of $0.1 million for Q3 2023, compared to a net loss of $2.1 million in Q3 2022[176] - The company continues to act as an agent in relation to DSUVIA sales to the DoD, recognizing revenue when the DoD obtains control of the product[55] Market and Future Outlook - The company has not yet generated significant product revenue and may never achieve profitability, raising concerns about its ability to continue as a going concern[208] - The company expects to continue incurring substantial losses in 2023 and may face negative cash flows from operations in the future[208] - Future capital requirements may vary significantly due to factors such as regulatory submissions, commercialization expenditures, and business development costs[191] - The company may require additional capital to sustain operations, which could delay or eliminate commercialization efforts and product development programs[210] - There is substantial doubt about the company's ability to continue as a going concern based on current operating expenses[212] - The unaudited financial statements for the quarter ended September 30, 2023, were prepared on the basis of a going concern[212] - Financial statements do not include adjustments that may be necessary if the company cannot continue as a going concern[212]

Financial Data and Key Metrics Changes - The company ended Q3 2023 with $13.4 million in cash and short-term investments, including $26 million from a financing round [16] - Revenues for Q3 were $0.1 million, primarily from royalties on DSUVIA sales, with a 75% royalty from the Department of Defense [16] - Combined R&D and SG&A expenses totaled $3.4 million in Q3, down from $4.5 million the previous year, with expectations for an increase in Q4 due to the upcoming clinical study [17] Business Line Data and Key Metrics Changes - The lead product candidate, Niyad, is a novel anticoagulant for dialysis, with a peak annual sales potential of over $200 million in the US, based on modest market penetration estimates [6][11] - The transition of DSUVIA to Alora Pharmaceuticals is ongoing, with AcelRx supporting Alora during this process [13] - DSUVIA demand from the Department of Defense remained stable, although revenue recognition was affected by shipment timing [14] Market Data and Key Metrics Changes - The market opportunity for Niyad is significant, with estimates suggesting a 20% share of the in-hospital CRRT market and 6% of the outpatient dialysis market [6] - The company is engaging with key opinion leaders in nephrology to discuss the NEPHRO study protocol, reinforcing the urgent medical need for alternatives in anticoagulation during dialysis [7][8] Company Strategy and Development Direction - AcelRx is focusing on unmet medical needs in dialysis, transitioning from acute pain management to developing Niyad for use in continuous renal replacement therapy [4] - The company is preparing for a PMA submission for Niyad in the second half of 2024, with potential launch in the first half of 2025 [11] - AcelRx is also evaluating the timing for NDA submission for its pre-filled syringe candidate, Fedsyra [12] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the success of the Niyad study, citing the favorable safety profile of nafamostat, which has been used in other markets [11] - The company anticipates robust enrollment for the NEPHRO CRRT study, with top-line data expected by mid-2024 [10][21] - Management highlighted the importance of the upcoming KOL panel discussion to further engage with the nephrology community [8] Other Important Information - The company received an ICD-10 CMS procedural code to facilitate reimbursement for Niyad [11] - AcelRx continues to lead its relationship with the Department of Defense, which is a significant customer for DSUVIA [14] Q&A Session Summary Question: Timeline for NEPHRO CRRT study enrollment and potential holiday impact - Management indicated that holiday periods do not significantly affect enrollment due to the nature of ICU patients [21] Question: Attributes for site selection in the NEPHRO CRRT study - Sites selected are major academic centers with expertise in nephrology and critical care, many of whom are advisors for the study [23] Question: Components of the PMA for Niyad beyond the registrational study data - Additional components include preclinical toxicology studies and stability data, which are being generated concurrently with the clinical trials [26] Question: Market opportunity against heparin and citrate - Management noted that while heparin is standard care, there is potential to capture market share from both citrate and patients receiving no anticoagulation [30][34] Question: Opportunities in intermittent hemodialysis - Management acknowledged that intermittent hemodialysis presents a larger market opportunity, although patients are generally less sick and may tolerate heparin better [36] Question: Future development opportunities beyond pre-filled syringes - The company is exploring development opportunities with LTX-608 for indications such as acute pancreatitis and disseminated intravascular coagulation [40]

AcelRx Pharmaceuticals, Inc. (NASDAQ:ACRX) Q2 2023 Earnings Conference Call August 10, 2023 4:30 PM ET Company Participants Raffi Asadorian – Chief Financial Officer Vince Angotti – Chief Executive Officer Pam Palmer – Founder and Chief Medical Officer Conference Call Participants Thomas Yip – H.C. Wainwright Laura Suriel – Alliance Global Operator Welcome to the AcelRx Second Quarter 2023 Financial Results Conference Call. This call is being webcast live via the Events page of the Investors section of Acel ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the quarterly period ended June 30, 2023 or ☐ TRANSITION REPORTS PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the transition period from to Commission File Number: 001-35068 ACELRX PHARMACEUTICALS, INC. (Exact name of registrant as specified in its charter) Delaware 41-2193603 (State or other jurisdiction ...

AcelRx Pharmaceuticals, Inc. (NASDAQ:ACRX) Q1 2023 Earnings Conference Call May 10, 2023 4:30 PM ET Company Participants Raffi Asadorian – Chief Financial Officer Vincent Angotti – Chief Executive Officer Pam Palmer – Co-Founder and Chief Medical Officer Conference Call Participants Brandon Folkes – Cantor Fitzgerald Thomas Yip – H.C. Wainwright James Molloy – Alliance Global Partners Operator Hello, welcome to the AcelRx First Quarter 2023 Financial Results Conference Call. This call is being webcast live ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the quarterly period ended March 31, 2023 or ☐ TRANSITION REPORTS PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the transition period from to Commission File Number: 001-35068 ACELRX PHARMACEUTICALS, INC. (Exact name of registrant as specified in its charter) Delaware 41-2193603 (State or other jurisdictio ...

Financial Data and Key Metrics Changes - AcelRx reported full year 2022 DSUVIA sales of $1.8 million, a 76% increase over 2021 despite reduced commercial investment [14] - The company ended 2022 with $20.8 million in cash and investments, and a debt balance of $5.4 million [15] - Combined R&D and SG&A expenses for Q4 2022 totaled $7.3 million, up from $6.9 million in Q4 2021 [15] Business Line Data and Key Metrics Changes - The divestment of DSUVIA to Alora Pharmaceuticals is expected to enhance sales through a 15% royalty on net commercial sales and $116.5 million in sales-based milestones [3] - The focus has shifted to the lead program, Niyad, which has FDA breakthrough designation and is being developed as an anticoagulant for extracorporeal circuits [6][9] Market Data and Key Metrics Changes - Market research indicates that 60% of CRRT patients currently receive no anticoagulant or use citrate, highlighting a significant opportunity for Niyad [8][29] - Niyad is estimated to have a peak sales potential of $200 million, specifically in the inpatient and outpatient dialysis markets [9][12] Company Strategy and Development Direction - AcelRx aims to leverage its expertise in drug development to focus on high-value assets, particularly the Niyad program and prefilled syringes [13] - The company plans to submit an EUA for Niyad in Q2 2023 and initiate a registrational trial in the second half of 2023 [10][49] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term value creation from the DSUVIA transaction and the potential for increased sales under Alora's management [14] - The company is focused on efficient cash management while advancing its late-stage pipeline, including Niyad and Fed Sera [13][16] Other Important Information - AcelRx has completed the production of the initial development batch of Niyad and is preparing for stability product testing [9] - The company has received an ICD-10 CMS procedural code for reimbursement related to Niyad [9] Q&A Session Summary Question: Can you discuss who drives military procurement for DSUVIA and current DoD orders? - The military procurement process is decentralized across different branches, with relationships fostered over time to ensure supply during potential escalations [20][21] Question: What are the steps remaining for submitting the EUA for Niyad? - The company is confident in the EUA submission process, with most of the package ready and focusing on CMC [23][24] Question: What are the expectations for commercial traction under the EUA for Niyad? - The target market for Niyad includes patients receiving no anticoagulant and those using citrate, representing a significant opportunity [28][29] Question: When is the registrational trial for Niyad expected to commence? - The registrational trial is expected to start in the second half of 2023, with endpoints already agreed upon with the FDA [49][48]

Acquisition and Divestment - 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[17]. - The company has divested its sufentanil sublingual products (DSUVIA and DZUVEO) to Alora Pharmaceuticals, expecting to close the deal in April 2023[15]. - The company plans to divest DSUVIA to Alora in April 2023, which will continue to commercialize the product and pay sales-based milestone payments[40]. - Alora will assume all manufacturing and supply chain activities for DSUVIA upon closing the divestment[54]. Product Development and Regulatory Approvals - Niyad™ has received Breakthrough Device Designation from the FDA and is being developed as a regional anticoagulant for injection into the extracorporeal circuit, with an EUA submission planned for Q2 2023[18][19]. - The FDA has provided guidance for a single registrational study for Niyad, which will involve 160 adult patients across 10 clinical sites[19]. - The PMA application process for Class III devices typically takes between one and three years, but can be significantly longer due to additional information requests or clinical trials[75]. - The FDA's review of a PMA application generally takes 180 days, but often extends beyond this period due to the complexity of the application[74]. - Niyad, a Class III medical device, is under FDA's Breakthrough Device Designation, which aims to expedite the review process for devices that offer significant advantages over existing treatments[71][78]. Financial Performance and Capital Needs - The company has incurred significant losses since inception and anticipates continued losses, requiring additional capital to fund operations[8]. - The company has not yet generated significant product revenue and may never achieve profitability[8]. - The market price of the company's common stock has historically been volatile, and significant sales of shares could impact stock price negatively[9]. - The company anticipates continued pressure from third-party payers to offer larger discounts or rebates to maintain acceptable reimbursement levels for its products[94]. Market Opportunities and Sales Potential - The peak sales potential for Niyad, an anticoagulant product, is estimated to exceed $200 million annually in the U.S. for CRRT and IHD applications[34]. - The peak sales potential for nafamostat in treating ARDS and DIC could range from nearly $700 million to up to $1.4 billion, depending on clinical study outcomes[35]. - The market opportunity for the pre-filled syringe products is estimated to exceed $100 million if approved, addressing the need for ready-to-use formulations in hospitals[36]. - DSUVIA, a sufentanil product, is approved for acute pain management in medically supervised settings, with a target patient population of over 90 million in the U.S.[37]. Compliance and Regulatory Risks - Manufacturers must comply with cGMP requirements throughout the manufacturing process, which includes extensive documentation and validation systems to ensure product safety and efficacy[69]. - The FDA can withdraw product approval if compliance with regulatory standards is not maintained, which may result in administrative actions such as fines or product recalls[70]. - Compliance with federal and state healthcare laws, including the Anti-Kickback Statute, is critical for pharmaceutical and medical device manufacturers to avoid legal repercussions[85][86]. - The company may be subject to civil penalties if found in violation of health regulatory laws, which could adversely affect operations and financial results[92]. Employee and Organizational Structure - As of December 31, 2022, the company employed 19 full-time employees, with approximately 80% working from the corporate office in Hayward, CA[105]. - The company conducts annual pay equity analyses and offers competitive salaries, stock options, and comprehensive benefits to its employees[105]. - The company has no employees under collective bargaining agreements and maintains a good relationship with its workforce[106]. Healthcare Policy and Market Environment - Legislative and regulatory changes in the healthcare system may impact the company's ability to commercialize products profitably, including potential controls on government-funded reimbursement for drugs[95]. - The Affordable Care Act and subsequent reforms, such as the Inflation Reduction Act of 2022, may lead to lower negotiated prices for the company's products compared to non-governmental payers[100]. - The Biden administration's executive order in July 2021 aims to promote competition in the American economy, specifically targeting prescription drug pricing[103]. - Future healthcare reform proposals may impose direct governmental price controls, potentially adversely impacting the company's business[104].