Saol Therapeutics announces FDA acceptance of New Drug Application for SL1009 for treatment of Pyruvate Dehydrogenase Complex Deficiency

ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, January 28, 2025 – Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for SL1009, Sodium Dichloroacetate Oral Solution (DCA). DCA will be used with a proprietary genetic test to treat an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD)1. The FDA granted Priority Review for the NDA and has set a goal date of May 27, 2025, under the Prescription Drug User Fee Act (PDUFA). Priority review is designated to applications for drugs that, if approved, would provide a significant improvement in safety or effectiveness of the treatment, prevention or diagnosis of a serious medical condition. SL1009 has previously been granted Orphan Drug and Fast Track Designations. SL1009 has also been granted a Rare Pediatric Disease Designation by the FDA and thus is eligible for a Priority Review Voucher (PRV).


PDCD is a rare and life-threatening mitochondrial disorder of carbohydrate oxidation that mostly affects the nervous system and skeletal muscle and leads to decreased ATP production and energy failure. DCA is a targeted therapy that inhibits Pyruvate Dehydrogenase Kinase (PDK) to stimulate residual Pyruvate Dehydrogenase Complex (PDC) activity and increase energy (ATP) production by mitochondria.

"This NDA acceptance brings us one step closer to addressing the critical health challenges faced by these children and their families, where no approved treatment is currently available," said Dave Penake, Chief Executive Officer of Saol Therapeutics. “Reaching this regulatory milestone is both a significant scientific achievement and a deeply meaningful moment for everyone who has dedicated themselves to advancing this therapy. Further, designating our application for priority review reaffirms that the FDA views PDCD as a serious condition”


The NDA is supported by results from a Phase 3 double-blind placebo-controlled cross-over study (SL1009-01) and a survival study (SL1009-02). The totality of evidence submitted for review in the NDA includes mechanistic characterization, along with nonclinical and clinical evidence to demonstrate the safety and clinical benefit of DCA in PDCD patients.

DCA is not currently approved for any indication in the United States.

 

About Pyruvate Dehydrogenase Complex Deficiency (PDCD)

PDCD is a mitochondrial disorder of carbohydrate oxidation that mostly affects the nervous system and skeletal muscle and leads to decreased ATP production and energy failure. It is estimated that 300-500 patients are treated in expert centers in the US, and the overall prevalence is thought to be as high as 2,000. PDCD is the most common cause of congenital lactic acidosis, a life-threatening condition that may occur as early as the neonatal period. Patients suffering from PDCD may also exhibit extreme tiredness (lethargy), poor feeding, rapid breathing (tachypnea), and other signs of neurological and neuromuscular dysfunction such as developmental delay, low muscle tone (hypotonia), abnormal eye movements and seizures. Signs and symptoms usually begin soon after birth but may appear later in childhood2.

There are currently no FDA-approved therapies for PDCD.

 

About Saol Therapeutics

Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda. Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases. Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations. For more information, visit www.saolrx.com.

 

Saol Therapeutics Contact
Brian Nappi, Senior Vice President, Strategy
bnappi@saolrx.com

Source: Saol Therapeutics announces FDA acceptance of New Drug Application for SL1009 for treatment of Pyruvate Dehydrogenase Complex Deficiency – Saolrx

Showcase Your Startup at the 2026 Georgia Life Sciences Summit

June 17, 2026
As part of the Georgia Life Sciences Summit (August 25–26), applications are now open for the Startup Showcase , sponsored by Johnson & Johnson , highlighting early-stage innovation across MedTech and Therapeutics . The Showcase will feature some of Georgia’s most innovative life science startups, giving emerging companies the opportunity to present groundbreaking technologies, products, and solutions to a distinguished audience of industry leaders, investors, researchers, and partners. Selected startups will present during the August 26 luncheon program and participate in a live investor Q&A session with John Gutierrez (Ascenta Capital), Emma Heckenberg, Ph.D. (Solas BioVentures), Patrick Jordan (NovaQuest Capital Management), and Emily Dinu (Numinous Capital) Members of the Johnson & Johnson external innovation team will also be onsite for the program and select companies will be scheduled for a 1:1 meeting to discuss strategic alignment and potential collaboration opportunities. If you or companies in your network are building in MedTech or Therapeutics, this is a strong opportunity to gain visibility, receive feedback on commercialization and growth strategy, and connect with key stakeholders. All applicants receive a complimentary Summit registration, and one selected company will also be invited to participate in the NewYorkBIO / New York Stock Exchange Life Sciences Showcase on December 10. If this is relevant to your work, it would be a strong opportunity to consider applying. If not, it may be worth sharing with companies in your network who are building in this space.

Georgia Life Sciences Educator Connection | June Newsletter

June 15, 2026
Workforce & Education Impact: Building Georgia's Future  Life Sciences Workforce - One Teacher at a Time June 16, 2026 - As Georgia Life Sciences concludes the 2025-2026 cycle of the Biotech Teacher Training Initiative (BTTI), the results reinforce the critical role educators play in building the state's future life sciences workforce. Since July of 2025, BTTI engaged 98 educators representing 40 schools across 23 school systems, reaching an estimated 5,375 students through hands-on biotechnology instruction, career-connected learning, and industry-relevant classroom experiences. The Georgia Life Sciences Equipment Depot further expanded the program's impact by supporting 238 teachers with access to laboratory equipment and materials that make authentic life sciences learning possible. Georgia Life Sciences is also pleased to report that funding for BTTI was maintained in the Georgia House version of the FY 2027 budget approved earlier this year. The upcoming 2026-2027 program cycle will mark an important evolution for BTTI. Building on feedback from industry partners and workforce trends across the life sciences sector, Georgia Life Sciences will expand the program's scope to better reflect the skills and competencies employers increasingly need. In addition to foundational biotechnology concepts, future programming will be organized around key industry domains, including Quality Assurance and Quality Control (QA/QC), Research & Development (R&D), MedTech, Artificial Intelligence, Automation, and Advanced Manufacturing. This expanded approach will provide educators with greater insight into the breadth of career opportunities available across Georgia's life sciences ecosystem while helping students develop awareness of the technologies and disciplines shaping the industry's future. The impact extends far beyond individual workshops, creating lasting connections between Georgia classrooms and the state's growing life sciences ecosystem while helping build the talent pipeline that will power Georgia's future innovation economy. "Before BTTI, I was hesitant to do biotech labs because they seemed too complex and expensive," shared Josephine Jeganathan of Stockbridge High School. "The program showed me how simplified it can be and provided the equipment and materials needed to successfully implement the Central Dogma Lab with all my classes." Teachers are also seeing increased student engagement through hands-on learning experiences. Tonie Curry of North Clayton High School used a chromatography lab to connect environmental science concepts to water pollution and sustainability. "Students were highly interested in seeing how substances separated and made strong connections to water pollution and environmental sustainability," Curry noted. "The hands-on nature of the lab encouraged curiosity and deeper understanding." For many educators, one of the most valuable aspects of the program is the connection between classroom learning and real-world careers. As Marshai Waiters of Marietta Middle School reflected: "Exposure is key. There are so many avenues to work in STEM, and they are all accessible with opportunity and knowledge. The insights gained from industry speakers will inform my teaching and create new opportunities for student exposure." When teachers are trained, equipped, and connected to industry, students gain more than a science lesson—they gain a window into Georgia's life sciences future.

PULSE Urges FTC, DOJ to Support Pro-Competitive Life Sciences Collaborations and M&A

June 10, 2026
In recent comments to the Department of Justice (DOJ) and Federal Trade Commission (FTC), PULSE highlighted critical reforms to ensure that U.S. competition policies support – and do not impede – the pro-competitive mergers and acquisitions (M&A) and other collaborations that drive American life sciences innovation. PULSE submitted comments in response to two joint DOJ/FTC Requests related to Guidance on Collaborations Among Competitors and Improvements to the Premerger Notification and Report Form (HSR Form). Read below for key takeaways from PULSE’s comment letters: 1. Life Sciences Innovation Depends on Collaboration “At its core, life sciences innovation is overwhelmingly collaborative. The cutting-edge medicines and cures developed by America’s life sciences industry are rarely the result of just one sole actor. Instead, they more often emerge from a calibrated sequence of partnerships… that collectively usher a biomedical breakthrough from early-stage discovery to FDA approval and delivery to patients.” – PULSE, Comments on Guidance on Collaborations Among Competitors 2. Policies that Ignore the Fundamental Role of Life Sciences M&A Risk Chilling Innovation for Patients “Against the significant challenges and pressures inherent to life sciences innovation, such policies that needlessly delay pro-competitive transactions have significant ripple effects: eroded investment incentives, disruptions in the path to launch and, ultimately, slowed or stalled development of new treatments and cures for patients.” – PULSE, Comments on Improvements to the HSR Form 3. Clear, Predictable Standards Can Support Competition and Innovation in America’s Life Sciences Ecosystem “Preserving clear and workable pathways for collaboration is therefore essential to sustain the broader ecosystem that delivers innovation and sustains America’s status as the world leader in life sciences innovation.” – PULSE, Comments on Guidance on Collaborations Among Competitors Leading business and industry organizations echoed and reinforced these priorities. Their comments highlight the unique market dynamics of life sciences innovation and the importance of collaboration and M&A – particularly with respect to early-stage R&D. “Because drug development typically takes over a decade, and the vast majority of drugs in development never make it to market, the antitrust risks associated with R&D collaborations in this space may be less than with other R&D collaborations.” – ABA Antitrust Law Section, Comments on Guidance on Collaborations Among Competitors “In the biopharmaceutical sector, for instance, R&D ventures in the pre-clinical or Phase I stages should be presumed lawful. At these early stages, about 90% of drugs never make it to market, so these collaborations are far too distant from commercialization to pose a meaningful threat to competition.” – U.S. Chamber of Commerce, Comments on Guidance for Collaborations Among Competitors “Healthy M&A activity also aids company formation and capital raising earlier in a business’s life cycle, as entrepreneurs and early-stage investors often depend on M&A for an exit opportunity. Conversely, discouraging business combinations by imposing burdensome one-size-fits-all standards on all transactions, like those imposed by the 2024 amendments, would disincentivize early-stage innovation and slow down economic growth.” – National Association of Manufacturers, Comments on Improvements to the HSR Form The bottom line: A balanced approach to antitrust enforcement policy should support life sciences M&A and other collaborations, ensuring new medicines continue to reach patients, while preserving a diverse and collaborative ecosystem. PULSE urges the agencies to adopt clear, workable and predictable standards that companies can apply with confidence. That includes preserving enforcement safety zones for low-risk, pro-competitive collaborations, as well as ensuring the HSR Form facilitates a timely, focused and fit-for-purpose screening process for life sciences M&A deals. Click below to read PULSE’s full comments: PULSE Comments on Guidelines on Collaborations Among Competitors PULSE Comments on Improvements to the HSR Form Source: PULSE Urges FTC, DOJ to Support Pro-Competitive Life Sciences Collaborations and M&A - Partnership for the U.S. Life Science Ecosystem (PULSE)
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