BRIDGEWATER, NJ - Amneal Pharmaceuticals, Inc. (NYSE: AMRX) (“Amneal” or the “Company”) today announced the addition of two denosumab biosimilars referencing both Prolia^® and XGEVA^® to its biosimilar pipeline.

Denosumab is a monoclonal antibody drug that inhibits bone reabsorption. It is indicated for two major categories of therapy: bone metastasis from various forms of cancer and prevention of bone pain and fractures, including osteoporosis-related injuries.

The two denosumab products are being developed by mAbxience, a global biotech company with over a decade of experience in the development, manufacture, and commercialization of biopharmaceuticals. Under the terms of the agreement, mAbxience will fully develop the biosimilar molecule and manufacture it in its state-of-the-art, Good Manufacturing Practice (GMP)-approved facilities, while Amneal will guide the product through regulatory approval and have exclusive commercialization rights in the United States. Amneal and mAbxience also currently partner on ALYMSYS^®, a bevacizumab biosimilar.

“Our goal is to be a top five player in the U.S. biosimilar space, similar to our leadership position in U.S. retail generics. Biosimilars represent the next wave of affordable medicines and these new product opportunities are aligned with our strategy to provide high quality, essential therapies,” said Harsher Singh, Senior Vice President, Amneal Biosciences. “Our first three commercial U.S. biosimilars are doing very well as our excellent commercial team drives uptake in these competitive categories. We are pleased to partner again with mAbxience on these next two biosimilar candidates, which deepens our pipeline and expands our presence in oncology.”

“We are thrilled to strengthen our partnership with Amneal through this second agreement, marking a significant step forward in our shared mission to enhance global health. This collaboration will bring two world-class biosimilars for the treatment of bone diseases and oncology to patients across the U.S., reinforcing our commitment to ensuring worldwide access to high-quality, life-enhancing treatments. Together with Amneal, we continue to make strides in offering affordable and accessible healthcare solutions, contributing positively to public health and solidifying our presence in the global biosimilar space,” said Emmanuelle Lepine, Chief Executive Officer, mAbxience.

According to IQVIA^®, U.S. annual sales for Prolia^® and XGEVA^® for the 12 months ended August 2023 were approximately $4.4 billion.

The financial terms of the transaction were not disclosed, and any incremental expenses associated with these products are contemplated within Amneal’s guidance.

UVA Health has promoted Danielle Griggs, PharmD, MBA, MS, to serve as the health system’s next chief pharmacy officer. A UVA Health team member since 2016, Griggs will oversee pharmacy services across all four of the health system’s medical centers in Charlottesville, Culpeper, Haymarket and Prince William.

Selected for her new role after a national search, Griggs will lead more than 500 frontline pharmacy team members in providing critical medications for patients across Virginia.

“Our pharmacy team is critical to the goal in our 10-year strategic plan of delivering care close to home for all Virginians with complex health needs,” said Min Lee, chief operating officer for UVA Health University Medical Center. “Whether being a persistent advocate for our pharmacy team in support of stellar operations or working tirelessly in pursuit of our mission to achieve outstanding outcomes in research, education and clinical care, Danielle is committed to inspiring ideas from our frontline team that will ensure the best service for our patients and communities, while remaining a premier pharmacy operation.”

Griggs has held a variety of roles at UVA Health, beginning as a pharmacy manager, where she was responsible for health system medication procurement and drug expense management. She went on to oversee the medication formulary, clinical and operational programs, drug shortages, and 340B operations and compliance. In 2019, Griggs was promoted to serve as senior director of pharmacy, where she has been responsible for pharmacy business operations across the health system.

“Over the past seven years, I have seen firsthand what a tremendous pharmacy team we have here at UVA Health, and I am honored to have been chosen to lead it,” Griggs said. “I look forward to continuing to work alongside our excellent team to provide high-quality care and service for all of our patients.”

Griggs received her doctor of pharmacy and master’s in business administration from the University of Kentucky. She also completed a residency in health system pharmacy administration at the University of North Carolina at Chapel Hill, where she also earned her master of science degree from the Eshelman School of Pharmacy.

BERKELEY, CA - Bayer AG announced today the opening of its first Cell Therapy Launch Facility in Berkeley, California to create the capacity to bring cell therapies to patients on a global scale. The $250 million (USD), 100,000-square-foot facility will supply the material required for late-stage clinical trials and potential commercial launch of BlueRock Therapeutics’ bemdaneprocel (BRT-DA01), an investigational cell therapy currently in evaluation for treating Parkinson’s disease. In addition, it includes space for a second module of production suites to support additional cell therapies as Bayer’s portfolio advances. BlueRock Therapeutics LP is a clinical stage, cell therapy company and wholly owned, independently operated subsidiary of Bayer AG.

“Cell therapy represent a groundbreaking class of medicines and is an area where Bayer is making a significant investment to research potentially transformative treatment approaches for people with unmet medical needs,” said Sebastian Guth, President of Bayer U.S.A. and Pharmaceuticals North America, and member of the Pharmaceutical Executive Committee. “Our new cell therapy facility represents true innovation in product development and manufacturing in addition to contributing to Bayer’s sustainability goal as our first fully electric pharmaceutical manufacturing plant.”

The new Cell Therapy Launch Facility is part of a transformation at the company’s dedicated biotechnology site in Berkeley, where Bayer has invested nearly USD 500 million in infrastructure over the past five years.

“Our teams are driving innovation in late-stage development and manufacturing with a goal of bringing transformational cell and gene therapies to patients on a global scale, and this facility will enable us to make it real,” said Jens Vogel, Sr. Vice President and Global Head of Biotech for Bayer’s Pharmaceutical Division. “Bayer is collaborating with biotech innovators, academia, and equipment and automation suppliers to establish platforms that would help bring more therapies to patients faster.”

Bayer’s global biotech organization recognizes the importance of helping innovators transfer their product candidates from the laboratory bench to the clinical study and commercial launch settings. The Biotech team provides its biologic development and manufacturing capabilities for Bayer’s larger biotherapeutics portfolio, including commercial products and late-stage protein and cell therapies in development. As part of Bayer’s larger mission of “Health for All,” the company is now also helping early-stage U.S. and European companies to enable patient trials and commercial launches through its BioPartnering Solutions offerings.

“Having access to this Cell Therapy Launch Facility is central to our goal to deliver impactful cell therapies from our pipeline to patients in need,” said Seth Ettenberg, President & CEO of BlueRock Therapeutics. “Our team is excited to be working shoulder to shoulder with Bayer’s biotech scientists and manufacturing experts as we look to scale up manufacturing for our first investigational therapy, bemdaneprocel for Parkinson’s disease, as it advances through clinical trials.”

The new Cell Therapy Launch Facility, launched in conjunction with manufacturing day in the United States, is among several recent investments to advance Bayer’s biologic pipeline of protein therapeutics, cell and gene therapies including a new Cell Culture Technology Center and Cell Therapy Labs. The new Cell Therapy Launch Facility features flexible, modular space for cell culture, viral transduction and automated filling of cell therapies leveraging Biotech@Bayer expertise in iPSC and CAR-T characterization, process development, analytics and clinical to commercial production.

Beyond Berkeley, the company’s global biotech network includes biologic development, manufacturing science, industrialization and advanced manufacturing engineering teams in Wuppertal and Leverkusen, Germany; and Basel, Switzerland; with a full complement of labs and clinical production suites.

About bemdaneprocel and Parkinson’s Disease

Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine producing neurons that are lost in Parkinson’s disease. These dopaminergic neuron precursors are derived from pluripotent stem cells (PSC) that are human embryonic stem cells. In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson’s disease. When transplanted, they have the potential to reform neural networks that have been severely affected by Parkinson’s and restore motor and non-motor function to patients. Planning is underway for BlueRock Therapeutics’ Phase II study that is expected to begin enrolling participants in H1 (first half) 2024.

Parkinson’s disease is a progressive neurodegenerative disorder caused by the death of nerve cells in the brain, leading to decreased dopamine levels. At diagnosis, it is estimated that patients have already lost 50-80% of their dopaminergic neurons. The loss of these neurons leads to a progressive loss of motor function and symptoms such as tremors, muscle rigidity, and slowness of movement. Even with medication, the symptoms of Parkinson’s disease can fluctuate during the course of the day. According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease, with approximately one million living in the United States. There is no cure, and the effectiveness of current treatments decreases over time.

About BioPartnering Solutions for Biotech Innovators in U.S. and Europe

Through Bayer’s BioPartnering Solutions, innovators can leverage industry-leading biotech process development and biomanufacturing capabilities to make their therapeutic candidates a reality. Bayer’s highly skilled Biotech teams and infrastructure – which includes preclinical, clinical and commercial launch scale manufacturing – are available to advance promising cell therapy, monoclonal antibody and protein therapeutic candidates from the discovery bench to patients. Through BioPartnering Solutions, Bayer provides early-stage companies with a single source of IND- and BLA-enabling development; bioprocess and biochemical engineering; clinical and commercial manufacturing. A range of complementing support service functions such as supply chain management, procurement, quality, and CMC strategy support for regulatory filings are also available. For more information visit: https://www.bayer.com/en/us/BioPartneringSolutions.

NEW HAVEN, CT. - BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience and immuno-oncology, today announced positive overall survival (OS) data from its Phase 2 trial of BXCL701, the Company's investigational oral innate immune activator, in combination with KEYTRUDA^® (pembrolizumab) in patients with small cell neuroendocrine prostate cancer (SCNC). As of a data cutoff of September 6, 2023, evaluable patients with SCNC (n=28) showed a median OS of 13.6 months (95% CI 10.9–NR), and a 12-month survival rate of 56.5%.

“OS is the most meaningful measure by which the effectiveness of an oncology treatment is evaluated. Though these results are based on a non-randomized cohort of patients, observing a median OS of this duration including patients with long-term survival at 12 months and beyond shows exceptional promise, bearing in mind historic data with checkpoint inhibitor monotherapy in this high-risk subset of prostate cancer,” said Rahul Aggarwal, M.D., Principal Investigator, Associate Director for Clinical Sciences, Helen Diller Family Comprehensive Cancer Center, and Professor of Medicine at the University of California San Francisco (UCSF). “SCNC represents a major unmet medical need, with the majority of patients unfortunately succumbing to their disease in less than one year following chemotherapy. The results of this trial suggest that BXCL701 has the potential to extend the lives of patients, and I look forward to its continued clinical development.”

SCNC, classified as a “cold” tumor, represents an underserved, growing patient population, with cases increasing due to earlier and more widespread use of androgen receptor inhibitors. In 2023, there will be an estimated 288,300² new patients with prostate cancer in the United States, with approximately 11,532 patients progressing to SCNC.

“We believe our trial results are highly encouraging for patients with this disease and have potential implications for our evaluation of BXCL701 for the treatment of other high-grade neuroendocrine tumors, such as small cell lung cancer, where effective therapies are lacking,” said Vincent J. O’Neill, M.D., Chief R&D Officer, OnkosXcel Therapeutics, a wholly owned subsidiary of BioXcel Therapeutics. “We intend to discuss these data with the FDA to help determine our next steps with clinical development.”

The Phase 2 trial is an open-label, multicenter study to evaluate the safety and efficacy of BXCL701 in combination with pembrolizumab in men with SCNC. Twenty-eight (28) evaluable SCNC patients received 0.3 mg of BXCL701 twice daily (BID) on days 1 through 14 of a 21-day cycle (0.2 mg BID the first week of cycle 1) plus 200 mg of pembrolizumab administered intravenously on day 1 and every subsequent 21 days. The primary objective of the trial is a composite response rate defined as either objective response by RECIST 1.1 criteria and/or PSA₅₀ and/or CTC count conversion.
Secondary objectives include duration of response, progression-free survival, overall survival, and biomarker evaluation as measured by changes in circulating cytokines and correlation of outcome with baseline tumor characteristics.

The Company is continuing to actively evaluate strategic options for OnkosXcel Therapeutics, including potential financing, strategic partnership, or M&A.

Conference Call
BioXcel Therapeutics will host a conference call and webcast on October 10, 2023 at 8:00 a.m. ET to discuss the data results from the Phase 2 trial of BXCL701. To access the call, please dial 877-407-5795 (domestic) or 201-689-8722 (international). A live webcast and presentation materials will be available on the Investors section of the corporate website, bioxceltherapeutics.com, and a webcast replay will be available through January 10, 2024.

NORTH CHICAGO, IL - AbbVie (NYSE: ABBV) announced today that it has exercised its exclusive right and completed the acquisition of Mitokinin, a discovery-stage biotechnology company developing a potentially first-in-class disease-modifying treatment for Parkinson's Disease (PD). Mitokinin's lead compound, a selective PINK1 activator, is designed to address mitochondrial dysfunction that is believed to be a major contributing factor to Parkinson's disease pathogenesis and progression.

PINK1 plays a key role in maintaining a healthy mitochondrial system by facilitating the turnover of dysfunctional mitochondria. Mutations in PINK1 are associated with a loss of PINK1 function and cause familial forms of PD. Activation of PINK1 offers a potentially disease-modifying treatment approach beyond familial PD, by addressing mitochondrial dysfunction and improving mitochondrial health in sporadic PD.

"Parkinson's disease continues to be a major unmet medical need, impacting patients, caregivers and society. With this acquisition, we are excited to grow our neuroscience portfolio and explore a potential new treatment option for PD," said Jonathon Sedgwick, Ph.D., vice president and global head of discovery research, AbbVie. "While current PD treatments may alleviate the symptoms of parkinsonism, there are currently no available therapies that prevent progression of the disease. Targeting PINK1 offers a novel approach that may alter disease pathogenesis."

"Collaboration with AbbVie's world-class Neuroscience and External Innovation teams added significant value and resources to help accelerate the program to investigational new drug (IND) enabling studies," said Daniel de Roulet, co-founder and chief executive officer, Mitokinin.

The role of PINK1 in addressing mitochondrial dysfunction has long been recognized; however, developing selective, brain penetrant PINK1 activators for therapeutic use in PD has been challenging.

"Our pre-clinical data demonstrate that our PINK1 activator compound can selectively enhance the active-form of PINK1, which is found on damaged mitochondria, without impacting PINK-1 regulation broadly," said Nicholas Hertz, Ph.D., co-founder and chief scientific officer, Mitokinin. "We believe this approach could potentially deliver significant clinical benefit to Parkinson's patients."

Under the terms of the agreement, AbbVie will pay Mitokinin shareholders $110 million at closing for the acquisition of Mitokinin. Mitokinin shareholders remain eligible for potential additional payments of up to $545 million upon the achievement of certain development and commercial milestones related to the success of the PINK1 program, plus tiered royalties based on net sales.

In a historic move, 75,000 healthcare workers at Kaiser Permanente facilities across the United States have gone on strike, demanding fair wages, better working conditions, and improved patient care. The strike is one of the largest labor actions in recent memory and has far-reaching implications for both healthcare workers and patients.

Reasons for the Strike

The Kaiser Permanente workers, including pharmacist, technicians, and support staff, have cited several key reasons for their strike action. At the forefront of their demands is a call for fair compensation. Workers argue that their wages have not kept pace with the rising cost of living, leaving many struggling to make ends meet. Union representatives assert that the strike is a last resort after failed negotiations with Kaiser Permanente management.

Additionally, healthcare workers are seeking improved staffing levels and working conditions. Overworked and understaffed hospitals and clinics have taken a toll on their ability to provide quality care to patients. The COVID-19 pandemic has only exacerbated these issues, pushing many healthcare professionals to their limits.

Impact on Patients

The strike has had a significant impact on patient care. With tens of thousands of healthcare workers walking off the job, Kaiser Permanente has been forced to cancel non-emergency procedures and reduce services in some areas. This has left many patients in need of vital medical care facing delays and uncertainty.

Kaiser Permanente has implemented contingency plans to minimize disruptions to patient care during the strike. They have brought in temporary replacement workers, but the situation remains challenging. Patients are facing longer wait times, and some have had to seek care at other facilities, putting additional strain on the healthcare system.

Potential Outcome of the Dispute

The outcome of this labor dispute could have far-reaching consequences for both the healthcare industry and the labor movement. As the strike continues, both sides are under pressure to reach a resolution that addresses the workers' concerns while ensuring the continuity of patient care.

If a fair agreement is reached, it could set a precedent for better wages and working conditions for healthcare workers across the country. The strike has already garnered significant public support, with many Americans recognizing the vital role these workers play in the healthcare system.

On the other hand, if the strike drags on without resolution, it could lead to further disruptions in patient care and could have negative implications for Kaiser Permanente's reputation. The longer the strike persists, the more challenging it will be for the healthcare provider to maintain its commitment to patient well-being.

The Kaiser Permanente workers' strike is a significant moment in the ongoing struggle for fair wages and better working conditions in the healthcare industry. As the strike continues, the eyes of the nation are on the negotiations between labor unions and management. The outcome of this dispute will not only impact the lives of the 75,000 workers involved but also the millions of patients who depend on Kaiser Permanente for their healthcare needs.

Paris, France - Sanofi announces today that it has entered into an agreement with Janssen Pharmaceuticals, Inc. (Janssen), a Johnson & Johnson company, to develop and commercialize the vaccine candidate for extraintestinal pathogenic E.coli (9-valent) developed by Janssen, currently in Phase 3. The agreement brings together Janssen’s robust science behind this potential first-in-class product and Sanofi’s worldwide manufacturing footprint and recognized world-class expertise in launching innovative vaccines.

Today, the U.S. Food and Drug Administration amended the emergency use authorization (EUA) of the Novavax COVID-19 Vaccine, Adjuvanted for use in individuals 12 years of age and older to include the 2023-2024 formula. Individuals 12 years of age and older previously vaccinated with a COVID-19 vaccine (and who have not already been vaccinated with a recently updated mRNA COVID-19 vaccine) are eligible to receive one dose and unvaccinated individuals receive two doses.

The updated vaccine addresses currently circulating variants to provide better protection against serious consequences of COVID-19, including hospitalization and death. Consistent with the totality of the evidence and input from the FDA’s expert advisors, the Novavax COVID-19 Vaccine, Adjuvanted, a monovalent vaccine, has been updated to include the spike protein from the SARS-CoV-2 omicron variant lineage XBB.1.5 (2023-2024 formula).

This authorization follows the FDA’s recent approvals and authorizations of updated mRNA COVID-19 vaccines for 2023-2024 manufactured by ModernaTX Inc. and Pfizer Inc.

“The COVID-19 vaccines have saved countless lives and have prevented serious outcomes of COVID-19 caused by the SARS-CoV-2 virus,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “Today’s authorization provides an additional COVID-19 vaccine option that meets the FDA’s standards for safety, effectiveness and manufacturing quality needed to support emergency use authorization. As we head into the fall season and transition into 2024, we strongly encourage those who are eligible to consider receiving an updated COVID-19 vaccine to provide better protection against currently circulating variants.”

The FDA evaluated manufacturing data to support the change to the 2023-2024 formula of the Novavax COVID-19 Vaccine, Adjuvanted. Additionally, the FDA evaluated non-clinical immune response data suggesting that the vaccine provides protection against the currently circulating COVID-19 variants. The agency also relied on its evaluation of safety and effectiveness data from clinical trials of Novavax COVID-19, Vaccine, Adjuvanted (Original monovalent) and investigational monovalent and bivalent Novavax COVID-19 adjuvanted vaccines, as well as postmarketing data. The data accrued with these Novavax COVID-19 vaccines are relevant to Novavax COVID-19 Vaccine, Adjuvanted (2023-2024 Formula) as the vaccines are manufactured using a similar process.

The FDA has determined that the Novavax COVID-19 Vaccine, Adjuvanted (2023-2024 Formula) has met the statutory criteria for issuance of an EUA, and that the known and potential benefits of the vaccine outweigh its known and potential risks in individuals 12 years of age and older.

As part of today’s action, the Novavax COVID-19 Vaccine, Adjuvanted (Original monovalent) is no longer authorized for use in the United States.

The FDA granted the emergency use authorization of the Novavax COVID-19 Vaccine, Adjuvanted (2023-2024 Formula) to Novavax Inc. of Gaithersburg, Maryland.

INDIANAPOLIS, IN - Eli Lilly and Company (NYSE: LLY) and POINT Biopharma Global, Inc. (NASDAQ: PNT) today announced a definitive agreement for Lilly to acquire POINT, a radiopharmaceutical company with a pipeline of clinical and preclinical-stage radioligand therapies in development for the treatment of cancer. Radioligand therapy can enable the precise targeting of cancer by linking a radioisotope to a targeting molecule that delivers radiation directly to cancer cells, enabling significant anti-tumor efficacy while limiting the impact to healthy tissue.

POINT's lead programs are in late-phase development. PNT2002¹ is a prostate-specific membrane antigen (PSMA) targeted radioligand therapy in development for patients with metastatic castration-resistant prostate cancer (mCRPC) after progression on hormonal treatment. Topline data from this study are expected in the fourth quarter of 2023. PNT2003¹ is a somatostatin receptor (SSTR) targeted radioligand therapy in development for the treatment of patients with gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Beyond the late-stage clinical pipeline, POINT has several additional programs in earlier stages of clinical and preclinical development. Additionally, POINT operates a 180,000-square-foot radiopharmaceutical manufacturing campus in Indianapolis, as well as a radiopharmaceutical research and development center in Toronto. These facilities will be utilized alongside POINT's extensive network of supply chain partners for sourcing radioisotopes and their precursors.

"Over the past few years, we have seen how well-designed radiopharmaceuticals can demonstrate meaningful results for patients with cancer and rapidly integrate into standards of care, yet the field remains in the early days of the impact it may ultimately deliver," said Jacob Van Naarden, President of Loxo@Lilly, the oncology unit of Eli Lilly and Company. "We are excited by the potential of this emerging modality and see the acquisition of POINT as the beginning of our investment in developing multiple meaningful radioligand medicines for hard-to-treat cancers, as we have done in small molecule and biologic oncology drug discovery and development. We look forward to welcoming POINT colleagues to Lilly and working together to build upon their achievements as we develop a pipeline of meaningful new radioligand treatments for patients."

Joe McCann, Ph.D., CEO of POINT added: "The combination of POINT's team, infrastructure and capabilities with Lilly's global resources and experience could significantly accelerate the discovery, development and global access to radiopharmaceuticals. I look forward to a future where patients all over the world can benefit from the new cancer treatment options made possible by the joining of our two companies today."

Terms of the Agreement

Lilly will commence a tender offer to acquire all outstanding shares of POINT for a purchase price of $12.50 per share in cash (an aggregate of approximately $1.4 billion) payable at closing. The transaction has been approved by the boards of directors of both companies.

The transaction is not subject to any financing condition and is expected to close near the end of 2023, subject to customary closing conditions, including the tender of a majority of the outstanding shares of POINT's common stock, and license transfer approval from the U.S. Nuclear Regulatory Commission. Following the successful closing of the tender offer, Lilly will acquire any shares of POINT that are not tendered in the tender offer through a second-step merger at the same consideration as paid in the tender offer.

The purchase price payable at closing represents a premium of approximately 87% to POINT's closing stock price on Oct. 2, 2023, the last trading day before the announcement of the transaction, and 68% to the 30-day volume-weighted average price. POINT's board of directors unanimously recommends that POINT's stockholders tender their shares in the tender offer.

Lilly will determine the accounting treatment of this transaction as a business combination or an asset acquisition, including any related acquired in-process research and development charges, according to Generally Accepted Accounting Principles (GAAP) upon closing. This transaction will thereafter be reflected in Lilly's financial results and financial guidance.

For Lilly, Goldman Sachs & Co. LLC is acting as exclusive financial advisor and Kirkland & Ellis LLP is acting as legal counsel. For POINT, Centerview Partners LLC is acting as exclusive financial advisor and Skadden, Arps, Slate, Meagher & Flom LLP is acting as legal counsel.

SAN MATEO, Calif.SAN MATEO, Calif.SAN MATEO, CA - AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX), (AcelRx), a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for use in medically supervised settings, today announced that it is advancing Niyad™ (a lyophilized formulation of nafamostat) into a registrational study following the recent approval of an Investigational Device Exemption (IDE) submission to the United States Food and Drug Administration (FDA).  This clinical trial will evaluate the safety and efficacy of Niyad to support a premarket approval application (PMA) expected to be submitted in the second half of 2024. The single study will consist of 166 adult patients undergoing renal replacement therapy (RRT) who cannot tolerate heparin or are at risk for bleeding.  If approved, Niyad would be the first-ever FDA-approved regional anticoagulant for the extracorporeal circuit in the U.S.

"We are thankful that the FDA has reviewed and approved our IDE application so we can now proceed with our registrational study to evaluate our lead product candidate, Niyad, which we believe to be a paradigm-shifting product candidate for the medically supervised setting," said Vince Angotti, Chief Executive Officer of AcelRx.  "We are on track to start this study later this year, with top-line data expected by mid-2024 and submission of a PMA planned in the second half of 2024."

The registration study is designed as a prospective, double-blinded trial to be conducted at approximately 10 U.S. hospital intensive care units that will enroll and evaluate 166 patients that will be randomized into either active or placebo arm.  The primary endpoint of the study is mean post-filter activated clotting time (ACT) over the first 24 hours versus placebo.  Key secondary endpoints include filter lifespan, number of filter changes over 72 hours, number of transfusions over 72 hours and dialysis efficacy (based on urea concentration) over the first 24 hours.

Pamela P. PalmerPamela P. Palmer, M.D., Ph.D., Chief Medical Officer and Co-Founder of AcelRx, commented, "Importantly, we are encouraged by our interactions with the FDA - not only by being granted Breakthrough Device Designation status for Niyad, but also in terms of the Agency's acceptance of a straightforward registrational trial design to evaluate Niyad.  Based on the feedback from the  Emergency Use Authorization request, the FDA stated that AcelRx should proceed with the clinical study. We're looking forward to our upcoming Investigator's Meeting just prior to starting enrollment.  We remain steadfast in our commitment to advance Niyad for use as a regional anticoagulant during renal replacement therapy, as current options are limited and patients often receive care below the recommended international standards.  We also look forward to publishing our independent qualitative market research related to these findings later this year."