Talphera Cuts NEPHRO CRRT Study Size, Secures $14.8 Million Private Placement
The trial size adjustment, along with protocol modifications and the addition of higher-enrollment sites, is expected to facilitate completion of the NEPHRO CRRT study of Niyad in patients undergoing renal replacement therapy by the end of 2025.
Image Credit: Adobe Stock Images/Crystal light
The FDA has approved Talphera’s request to streamline its NEPHRO CRRT study of Niyad (nafamostat) in patients undergoing renal replacement therapy unable to tolerate heparin or who are at risk for bleeding. As a result, patient enrollment will be reduced from 166 to 70 while maintaining a 90% statistical power for the primary endpoint. This adjustment is expected to accelerate trial completion and support the company’s timeline for regulatory submission.1
"We are pleased that the FDA agreed with our assessment to reduce the study size to 70 patients, which still keeps the study's primary endpoint powered at 90%," said Vince Angotti, CEO, Talphera, in a press release. "This change, along with the other protocol changes to the study inclusion criteria and the addition of new sites with higher enrollment potential will support a timely completion of the study, which we are targeting by the end of the year. We are also thankful for the continued support from our existing investors, Nantahala and Rosalind, which led the private placement financing. The additional capital should support our objective of completing the study by the end of the year as we look forward to submitting our PMA early next year and a potential approval in the second half of 2026."
NEPHRO CRRT is a prospective, double-blind, Phase III trial. The primary endpoint is mean post-filter activated clotting time in patients treated with Niyad compared to placebo over the first 24 hours. Secondary endpoints include mean post-filter activated clotting time over 72 hours, filter lifespan, number of filter changes over 72 hours, number of transfusions over 72 hours, and dialysis efficacy over the first 24 hours.
Additionally, the company secured a private placement financing of up to $14.8 million, led by Nantahala Capital and Rosalind Advisors, structured in three tranches based on enrollment milestones. The first closing, which is expected on April 2, 2025, is for $4.925 million.
An additional $4.925 million is expected at the second closing, pending the enrollment of a minimum of 17 patients in the NEPHRO CRRT study, with a minimum stock price of $0.7325 per share for five consecutive days. Finally, an additional $4.925 million is expected in the third closing, amid the enrollment of 35 patients in the study and a minimum stock price of $0.7325 per share for five consecutive days. The first closing will involve issuing 3,405,118 shares of common stock and 4,999,316 pre-funded warrants at $0.586 and $0.585 per unit, respectively, with subsequent closings expected to issue the same amounts upon meeting enrollment milestones and closing conditions.1
Alongside this announcement, Talphera reported its fourth-quarter and full-year 2024 financial results. At the end of the year, the company had a total of $8.9 million in cash and cash equivalence. Additionally, expenses were approximately $2.8 million in Q4, down from $4.3 million in Q4 of 2023. Talphera reported a net loss of $1.9 million, a significant improvement from the $4.5 million loss in Q4 2023, primarily driven by changes in the fair value of its warrant liability. In 2025, the total budget is estimated to be between $18 million and $19 million.2
"The recent announcements of the FDA's approval to reduce the study size to 70 patients, the private placement financing, and FDA-agreed study protocol changes, provide strong momentum to support the NEPHRO CRRT study execution," Angotti said in a press release. "These developments, coupled with the addition of new sites with higher enrollment potential, two of which have just started screening patients with six more sites expected in the first half of this year, support our belief that we will complete the study by the end of 2025. In addition, clinicians' continued enthusiasm for Niyad's profile reinforces our confidence in its potential to fill a significant unmet need in the CRRT market."2
References
1. Talphera Announces Agreement with the FDA to Reduce the NEPHRO CRRT Study Size to 70 Patients From 166 and a Private Placement Financing Priced At-the Market of up to $14.8 Million. PR Newswire. March 31, 2025. Accessed April 1, 2025. https://prnmedia.prnewswire.com/news-releases/talphera-announces-agreement-with-the-fda-to-reduce-the-nephro-crrt-study-size-to-70-patients-from-166-and-a-private-placement-financing-priced-at-the-market-of-up-to-14-8-million-302415309.html
2. Talphera Announces Fourth Quarter and Full Year 2024 Financial Results and Provides Corporate Update. PR Newswire. March 31, 2025. Accessed March 31, 2025. https://www.prnewswire.com/news-releases/talphera-announces-fourth-quarter-and-full-year-2024-financial-results-and-provides-corporate-update-302416091.html
Key Findings of the NIAGARA and HIMALAYA Trials
November 8th 2024In this episode of the Pharmaceutical Executive podcast, Shubh Goel, head of immuno-oncology, gastrointestinal tumors, US oncology business unit, AstraZeneca, discusses the findings of the NIAGARA trial in bladder cancer and the significance of the five-year overall survival data from the HIMALAYA trial, particularly the long-term efficacy of the STRIDE regimen for unresectable liver cancer.
Cell and Gene Therapy Check-in 2024
January 18th 2024Fran Gregory, VP of Emerging Therapies, Cardinal Health discusses her career, how both CAR-T therapies and personalization have been gaining momentum and what kind of progress we expect to see from them, some of the biggest hurdles facing their section of the industry, the importance of patient advocacy and so much more.
