Tranquis Therapeutics, Inc.

BETHESDA, Md., April 01, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation allosteric small molecule therapies, today announces the appointment of Jonas Hannestad, M.D., Ph.D. as its chief medical officer, effective as of March 25, 2024. “I am pleased to welcome Jonas to Gain and look forward to his contributions. His extensive development experience and in-depth scientific background in Parkinson’s and other neurodegenerative diseases will be invaluable as we advance our GT-02287 program through the ongoing Phase 1 clinical trial into further clinical development,” commented Gain CEO Matthias Alder. Dr. Hannestad has 25 years of translational and early clinical development experience in CNS. His academic work focused on immuno-neurology, particularly on the effect of systemic inflammation on myeloid cells in the brain. His drug development expertise spans multiple indications, including Parkinson’s, Alzheimer’s, and ALS. Previously, he served as Chief Medical Officer at Tranquis Therapeutics and Capacity Bio, SVP of Clinical Development at Alkahest, Medical Director at Denali Therapeutics, and Director of Neuroscience Discovery Medicine at UCB Pharma. Prior to his industry career, Dr. Hannestad led the Clinical Neuroscience Research Unit at the Yale School of Medicine. He did postdoctoral work and residency training at Bristol-Myers Squibb, Duke School of Medicine, and the UCLA Neuropsychiatric Institute. He received his Ph.D. in Cell Biology from the University of Messina, Italy, and his M.D. from the University of Oviedo, Spain. “Looking at the extensive data package that the Gain team has generated during preclinical development, Gain’s lead drug candidate GT-02287 has a first and best-in-class profile. I look forward to working with this team on advancing this exciting and potentially disease-modifying therapy for patients with Parkinson’s disease,” added Dr. Hannestad. Inducement Grant In connection with his appointment, the Company granted Dr. Hannestad an option to purchase 200,000 shares of the Company’s common stock at an exercise price equal to $4.06 per share, the closing price of the Company’s common stock on March 22, 2024, the last trading day preceding Dr. Hannestad’s employment start date and date of grant. This award was approved by the Compensation Committee of Gain’s Board of Directors and granted under the Gain Therapeutics, Inc. 2021 Inducement Equity Incentive Plan, as an inducement material to Dr. Hannestad entering into employment with Gain, in accordance with Nasdaq Listing Rule 5635(c)(4). The stock option vests over four years, with one-fourth (1/4th) of the shares subject to the option vesting on the first anniversary of Dr. Hannestad’s employment start date (the “Initial Vesting Date”) and the remaining three-fourths (3/4ths) of the shares subject to the option vesting in a series of thirty-six (36) successive equal monthly installments on each subsequent monthly anniversary of the Initial Vesting Date, subject to Dr. Hannestad being continuously employed by the Company as of such vesting dates. The Company is providing this information in accordance with Nasdaq Listing Rule 5635(c)(4). About GT-02287 Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of GBA1 Parkinson’s disease (GBA1-PD). The orally administered, brain-penetrant small molecule is an allosteric protein modulator that restores the function of the lysosomal protein enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to a GBA1 gene mutation, the most common genetic abnormality associated with PD. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced aggregated α-synuclein, neuroinflammation and neuronal death, increased dopamine levels and improved motor function. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker for neurodegeneration. Gain’s lead program in Parkinson’s disease has been awarded funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse – Swiss Innovation Agency. About GBA1 Parkinson’s Disease GBA1 Parkinson’s disease is caused by mutations in the GBA1 gene, found in up to 15% of patients with Parkinson’s disease and making it the primary genetic risk factor. The mutation causes dysfunctional misfolding of the lysosomal enzyme glucocerebrosidase (GCase), reducing its activity in the brain and leading to the subsequent accumulation of α-synuclein and degeneration of dopamine-containing nerve cells. Patients with GBA1-PD tend to have earlier onset and faster symptom progression than those with sporadic PD, a progressive neurodegenerative disease characterized by a motor syndrome consisting of bradykinesia (slowness of movement), rigidity, resting tremors, and postural instability. With current therapies treating only the symptoms of Parkinson’s disease without affecting the underlying progression of the disease, there is an unmet need to develop novel disease-modifying therapies such as GT-02287 that have the potential to slow or stop disease progression and help improve outcomes in this patient population. About Gain Therapeutics, Inc. Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate GT-02287 for the treatment of GBA1 Parkinson’s disease, is currently being evaluated in a Phase 1 clinical trial. Leveraging AI-supported structural biology, proprietary algorithms and supercomputer-powered physics-based models, the company’s Magellan™ drug discovery platform can identify novel allosteric binding sites on disease-implicated proteins, pinpointing pockets that cannot be found or drugged with current technologies. Magellan™ is the next generation of Gain’s original SEE-Tx® (Site-Directed Enzyme Enhancement Therapy) platform, which was enhanced and expanded with new AI and machine-learning tools and virtual screening capabilities to access the emerging on-demand compound libraries covering vast chemical spaces of over 50 billion compounds. Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders and oncology. For more information, please visit GainTherapeutics.com and follow us on LinkedIn. Cautionary Note Regarding Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. All statements in this press release other than statements of historical facts are “forward-looking statements”. In some cases, you can identify these statements by forward-looking words such as "may," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "predict," "goal, " "intend," "seek, " "potential" or "continue," the negative of these terms and variations of these words or similar expressions that are intended to identify forward-looking statements, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements regarding: the development of the Company’s current or future product candidates including GT-02287; expectations regarding the timing of results from a Phase 1 clinical study for GT-02287; and the potential therapeutic and clinical benefits of the Company’s product candidates. These forward-looking statements are based on the Company’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause the Company’s preclinical and future clinical development programs, future results or performance to differ materially from those expressed or implied by the forward-looking statements. These statements are not historical facts but instead represent the Company's belief regarding future results, many of which, by their nature, are inherently uncertain and outside the Company's control. Many factors may cause differences between current expectations and actual results, including the impacts of the post-COVID-19 environment and other global and macroeconomic conditions on the Company’s business; clinical trials and financial position; unexpected safety or efficacy data observed during preclinical studies or clinical trials, clinical trial site activation or enrollment rates that are lower than expected; changes in expected or existing competition; changes in the regulatory environment; the uncertainties and timing of the regulatory approval process; and unexpected litigation or other disputes. Other factors that may cause the Company’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are identified in the section titled “Risk Factors,” in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 26, 2024 and its other documents subsequently filed with or furnished to the Securities and Exchange Commission from time to time. All forward-looking statements contained in this press release speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Investor Contact: CORE IR (516) 222-2560 ir@gaintherapeutics.com Media Contacts: Russo Partners Nic Johnson and Elio Ambrosio nic.johnson@russopartnersllc.com elio.ambrosio@russopartnersllc.com (212) 845-4242

Aug. 29, 2022 12:00 UTC -TQS-168 demonstrated robust geroprotective effects in preclinical studies- REDWOOD CITY, Calif.--(BUSINESS WIRE)-- Tranquis Therapeutics, Inc., a clinical stage immuno-neurology company developing innovative medicines with the potential to revolutionize the management of neurodegenerative and aging-related diseases, today announced it will present preclinical data on the anti-aging effects of TQS-168, a small molecule modulator of PGC-1a, at ARDD 2022, the 9th Aging Research and Drug Discovery meeting, taking place August 29 to September 2, 2022 at the University of Copenhagen, Denmark. In the preclinical study conducted in 18-month-old mice, it was shown that treatment for four months with TQS-168 resulted in a 66% improvement in the 31-point Frailty Index, a commonly used measurement to evaluate aging-related changes in mice. Treatment with TQS-168 also resulted in improvements in grip strength and treadmill running endurance, and a reduction in fat mass with no change in lean mass. These results indicate that TQS-168 has robust geroprotective effects in mice. TQS-168 was found to be well tolerated in healthy adult male subjects in a Phase 1 clinical trial. Adverse events were mild and transient, and no abnormalities were observed in laboratory parameters, ECG, or vital signs. No serious adverse events occurred. TQS-168 is ready for clinical trials to evaluate its potential to impact human aging and quality of life in the elderly. Tranquis is also advancing TQS-168 into clinical studies in other indications. About TQS-168 and PGC-1a TQS-168 is a PGC-1a-enhancing small molecule in development for the treatment of neurodegenerative, metabolic, and aging-related diseases in which PGC-1a modulation plays a critical role. PGC-1a is a transcriptional coactivator that regulates genes involved in cell energy metabolism, acting as a key regulator of mitochondrial biogenesis, giving TQS-168 potential as a target in a broad spectrum of diseases. TQS-168 showed survival and functional benefits in various animal models of neurodegenerative disease, including ALS and Parkinson’s disease. TQS-168 also demonstrated compelling biomarker data, including increased PGC-1a gene expression, reduction of inflammatory monocytes and cytokines, and reduced levels of neurofilament light in animal models. In vitro, TQS-168 also shows specific immunomodulatory effects in myeloid cell lines and in white blood cells from people living with ALS. About Tranquis Tranquis is a clinical-stage biopharmaceutical company developing a portfolio of small molecule therapeutics based on its unique approach of modulating PGC-1a to restore normal cell homeostasis and function. Tranquis’ platform has the potential to revolutionize the management of an entire range of indications in the fields of neurodegeneration, mitochondrial diseases, immuno-inflammation, and immunosenescence. In addition, the Company’s approach may promote healthy aging and longevity.

June 21, 2022 12:00 UTC -Tranquis plans to start a Phase 2 trial in people living with ALS by the end of 2022- REDWOOD CITY, Calif.--(BUSINESS WIRE)-- Tranquis Therapeutics, Inc., a clinical stage immuno-neurology company developing innovative medicines with the potential to revolutionize the management of neurodegenerative and aging-related diseases, today announced the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for TQS-168, a small molecule modulator of PGC-1a, for the treatment of amyotrophic lateral sclerosis (ALS). ALS is a devastating disease with a high unmet need. Tranquis expects to begin a Phase 2 study of TQS-168 in people living with ALS by the end of 2022. The FDA’s Office of Orphan Drug Products grants orphan status to drugs and biologics that demonstrate promise for the treatment of diseases or conditions affecting fewer than 200,000 people in the United States. Orphan drug designation provides Tranquis with certain development incentives, including tax credits for qualified clinical trials, exemptions from FDA user fees, and the potential for seven years of market exclusivity, if TQS-168 is approved. “The orphan drug designation is an important regulatory milestone for Tranquis, further validating our efforts to develop TQS-168, an orally administered compound that targets PGC-1a in myeloid cells, as a novel therapeutic option for people living with ALS,” said Jonas Hannestad, MD, PhD, Chief Medical Officer at Tranquis. “Earlier this month, we reported Phase 1 data on TQS-168 in 78 healthy volunteers demonstrating excellent pharmacokinetic properties and blood plasma exposures associated with clinical benefit in preclinical models, along with a favorable safety and tolerability profile. We look forward to rapidly advancing our Phase 2 program in ALS.” About TQS-168 and PGC-1a TQS-168 is a PGC-1a-targeting small molecule in development for the treatment of ALS and other neurodegenerative diseases in which dysfunction of myeloid cells plays a critical role. PGC-1a is a transcriptional coactivator that regulates genes involved in cell energy metabolism, acting as a key regulator of mitochondrial biogenesis. When upregulated in dysfunctional myeloid cells, PGC-1a normalizes cell energy metabolism and immune function, giving it potential as a target in a variety of neurodegenerative diseases. TQS-168 showed survival and functional benefits in various animal models of neurodegenerative disease, including ALS and Parkinson’s disease. TQS-168 also demonstrated compelling biomarker data, including increased PGC-1a gene expression, reduction of inflammatory monocytes and cytokines, and reduced levels of neurofilament light in animal models. In vitro, TQS-168 also shows specific immunomodulatory effects in myeloid cell lines and in white blood cells from people living with ALS. About Tranquis Tranquis is a clinical-stage biopharmaceutical company developing a portfolio of small molecule therapeutics based on its unique approach of reprogramming dysfunctional myeloid immune cells to restore normal cell homeostasis and function. Tranquis’ therapeutic candidates can “switch” dysfunctional microglia and monocytes back to a homeostatic functional state by targeting master regulators of cell energy metabolism, such as PGC-1a. Tranquis’ platform has the potential to revolutionize the management of an entire range of indications in the fields of neurodegeneration, mitochondrial diseases, immuno-inflammation, and immunosenescence. In addition, the Company’s approach may promote healthy aging and longevity.