GBA

The capsule is intended for patients with Type 1 Gaucher disease. Credit: Sirikarn Rinruesee/Shutterstock.com. Edenbridge Pharmaceuticals has selected PANTHERx Rare to distribute Yargesa (miglustat) capsules for the treatment of Type 1 Gaucher disease. The medication represents the first oral treatment option for adults with mild to moderate disease who are unable to use enzyme replacement therapy. The glucosylceramide synthase inhibitor Yargesa exerts its effects through the substrate reduction therapy process. This treatment method lessens the accumulation of harmful glycosphingolipids, thereby reducing symptoms. Gaucher disease is a rare genetic disorder characterised by a deficiency in the glucocerebrosidase enzyme, which leads to the build-up of a lipid known as glucosylceramide. See Also: Lonza Group gets grant for patent granted for capsule composition with pullulan, setting system, surfactant MediciNova gets grant for ibudilast higher dosage oral tablet or capsule formulations PANTHERx Rare Pharmacy CEO Rob Snyder stated: “We appreciate Edenbridge Pharmaceuticals’ commitment to making a difference in the lives of patients with Type 1 Gaucher disease. “We are looking forward to serving the needs of the patients dealing with this illness, by streamlining the process associated with getting rare medications from the people who create them to the people who need them most, ultimately delivering better health outcomes to people living with rare diseases.” In February 2024, Chiesi Global Rare Diseases, a Chiesi Group business unit, selected PANTHERx Rare for the distribution of Filsuvez topical gel. Filsuvez topical gel treats wounds associated with dystrophic as well as junctional epidermolysis bullosa in adults and paediatric patients aged six months and above.

- Announced private placement of up to $149.4 million led by RA Capital Management with participation from Armistice Capital, Special Situations Funds and Soleus Capital; pro forma cash and cash equivalents from upfront proceeds expected to provide runway through the end of 2025 - CervoMed on track to complete enrollment in 2Q 2024 in its RewinD-LB Phase 2b clinical trial evaluating neflamapimod in patients with dementia with Lewy bodies (DLB); topline data expected in 4Q 2024 - Integrated summary of results from AscenD-LB Phase 2a trial published in peer-reviewed journal and presentations at a major scientific conference further inform on the potential of neflamapimod in DLB and probability of success in RewinD-LB - Appointed Joshua Boger, Ph.D., as Chair of the Board of Directors; Dr. Boger is the founder and former CEO of Vertex Pharmaceuticals BOSTON, April 01, 2024 (GLOBE NEWSWIRE) -- CervoMed Inc. (NASDAQ: CRVO), a clinical stage company focused on developing treatments for age-related neurologic disorders, today reported its financial results for the fourth quarter and full year ended December 31, 2023. “In 2023, we made transformative advancements to build our business, including successfully listing on NASDAQ and have begun 2024 by strengthening our leadership with the appointment of industry veteran, Joshua Boger, Ph.D., as non-executive Chair of the Board,” said John Alam, MD, Chief Executive Officer of CervoMed. “With this momentum, we look forward to building on recent publications that position our lead clinical program, neflamapimod, as a highly differentiated, first-to-market treatment option for patients with DLB. Our RewinD-LB Phase 2b trial is well powered, designed to include patients most likely to benefit from neflamapimod, and is expected to provide a path to market in this high value indication. We remain on track to fully enroll the RewinD-LB trial within the second quarter of this year, followed by availability of topline efficacy results in the fourth quarter of 2024.” Recent Highlights and Anticipated Milestones Enrollment in the randomized, controlled Phase 2b RewinD-LB trial evaluating oral neflamapimod in patients with DLB continues to progress and the Company remains on track to complete enrollment in the second quarter of 2024.At the International Conference on Alzheimer’s & Parkinson’s Diseases (AD/PD) in March 2024, the Company presented data from the AscenD-LB Phase 2a trial in a poster demonstrating that, neflamapimod treatment led to significant improvement compared to placebo in the change in plasma levels of glial fibrillary acidic protein (GFAP) in patients with pure DLB. Moreover, the neflamapimod treatment effects on GFAP were correlated to clinical outcomes, assessed by the CDR-SB. In a separate presentation at AD/PD, scientific collaborators from University College London presented data demonstrating neflamapimod improves axonal transport in a transgenic mouse model of frontotemporal dementia.CervoMed’s CEO, John Alam, MD, participated in a panel discussion at AD/PD titled, “New Insights in the Development of Biomarkers, Imaging, and Therapy of Alpha-synuclein, LRKK2, and GBA Pathologies.” Integrated summary of results from the AscenD-LB Phase 2a trial evaluating treatment with neflamapimod in patients with DLB were published in the Journal of Prevention of Alzheimer’s Disease (JPAD) in February 2024. The manuscript is available online and along with the integrated summary, includes the first peer reviewed publication of positive electroencephalogram and MRI data with neflamapimod treatment. The ongoing RewinD-LB study was designed based on key learnings from the Phase 2a AscenD-LB trial of neflamapimod, including the use of a single dose regimen of neflamapimod 40mg three-times-a-day (TID), enrolling patients with pure DLB (with pre-treatment plasma ptau181 below cutoff) and selecting the Clinical Dementia Rating Sum of Boxes (CDR-SB) scale as the primary endpoint. The RewinD-LB trial also includes the use of structural and functional MRI in a 40-patient subgroup to assess treatment effects on atrophy of the basal forebrain, as well its functional connectivity. Corporate Update On March 28, 2024, the Company announced entry into a securities purchase agreement pursuant to which it agreed to sell an aggregate of 2,532,285 units (the “Units”), each Unit comprised of (i) (A) one share of its common stock or (B) one pre-funded warrant to purchase one share of common stock, and, in each case, (ii) one Series A warrant to purchase one share of common stock to a select group of institutional and accredited healthcare specialist investors led by RA Capital Management in a private placement. Each Unit will have a purchase price of $19.745 (or $19.744 in the case of Units that include a pre-funded warrant in lieu of common stock). The Company anticipates the aggregate upfront gross proceeds from the private placement will be approximately $50 million, before deducting any offering-related fees and expenses, and up to approximately $99.4 million in additional aggregate gross proceeds if the Series A warrants are fully exercised for cash. The Series A warrants have an initial exercise price of $39.24 per share, representing a 100% premium to the last sale on March 27, 2024, will be immediately exercisable, and will expire at the earlier of (i) April 1, 2027 or (ii) 180 days after the date that the Company makes a public announcement of positive top-line data from the Company’s Phase 2b RewinD-LB clinical trial evaluating neflamapimod for treatment of patients with dementia with Lewy bodies (“DLB”). The private placement is expected to close on or about April 1, 2024, subject to customary closing conditions.Appointed industry leader Joshua Boger, Ph.D., as non-executive Chair of the Board. Dr. Boger is an industry veteran who has served in multiple scientific and business leadership roles in his 40+ year career. Notably, he is the founder and former CEO of Vertex Pharmaceuticals. Full Year 2023 Financial Results Cash Position: As of December 31, 2023, CervoMed had $7.8 million in cash and cash equivalents as compared to $4.1 million as of December 31, 2022. The Company currently expects its cash position as of December 31, 2023, along with the remaining funds to be received from the NIA grant received and the upfront private placement funding of $50.0 million announced March 28, 2024, and expected to be received by the Company on April 1, 2024, will enable it to fund its operating expenses and capital expenditures through the end of 2025. Grant Revenue: Grant revenue was $7.1 million for the year ended December 31, 2023, compared to no revenue for the same period in 2022. Research and Development (R&D) Expenses: R&D expenses for the year ended December 31, 2023, were $8.4 million, compared to $1.3 million for the year ended December 31, 2022. This increase was attributed to the RewinD-LB Phase 2b clinical study in DLB which began in the first quarter of 2023. General and Administrative (G&A) Expenses: G&A expenses were $6.5 million for the year ended December 31, 2023, versus $2.1 million for the year ended December 31, 2022. This increase was attributable to the additional costs related to the reverse merger, which closed in August 2023, compensation for additional headcount, and professional service fees. Operating Loss: Operating loss was $7.8 million for the year ended December 31, 2023, compared to $3.4 million for the year ended December 31, 2022. Net Loss: Net Loss was $2.2 million for the year ended December 31, 2023, compared to a net loss of $5.8 million for year ended December 31, 2022. The lower net loss in 2023 versus 2022 was driven by a noncash gain recognized for the conversion of the convertible notes upon the closing of the reverse merger, which was based on the stock price on the date of the transaction. About the RewinD-LB Phase 2b Study in Dementia with Lewy BodiesCervoMed’s ongoing Phase 2b study, RewinD-LB, is a randomized, 16-week, double-blind, placebo-controlled clinical trial evaluating oral neflamapimod (40mg TID) in up to 160 patients with very mild or mild dementia due to DLB. Patients completing the 16-week placebo-controlled study period will be able to continue in the study while receiving open label neflamapimod treatment for an additional 32 weeks. Patients with AD-related co-pathology, assessed by a blood biomarker (plasma ptau181), will be excluded. The primary endpoint in the study is change in CDR-SB, and secondary endpoints include the Timed Up and Go test, a cognitive test battery, and the Clinician’s Global Impression of Change. The RewinD-LB study is funded by a $21 million grant from the National Institutes of Health’s National Institute on Aging, which will be disbursed over the course of the study as costs are incurred. The study includes 41 sites (30 in the United States, 8 in the United Kingdom, and 3 in the Netherlands), all of which have been initiated. More information on the RewinD-LB study, including contact information on active clinical trial sites, is available at clinicaltrials.gov. About CervoMedCervoMed Inc. is a clinical-stage company focused on developing treatments for age-related neurologic disorders. The Company is currently developing neflamapimod, an investigational, orally administered small molecule brain penetrant that inhibits p38MAP kinase alpha. Neflamapimod has the potential to treat synaptic dysfunction, the reversible aspect of the underlying neurodegenerative processes that causes disease in DLB and certain other major neurological disorders. Neflamapimod is currently being evaluated in a Phase 2b study in patients with DLB. Forward-Looking StatementsThis press release includes express and implied forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, regarding the intentions, plans, beliefs, expectations or forecasts for the future of the Company, including, but not limited to, the therapeutic potential of neflamapimod, the anticipated timing and achievement of clinical and development milestones, including the completion and achievement of primary endpoints of the Company’s Phase 2b clinical trial, the anticipated timing, size, closing and receipt of proceeds from the pending private placement transaction, expectations regarding market conditions, the satisfaction of customary closing conditions related to the private placement and the anticipated use of proceeds therefrom, and projected cash runway. Terms such as “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “approximately,” “potential” or other words that convey uncertainty of future events or outcomes may identify these forward-looking statements. Although there is believed to be reasonable basis for each forward-looking statement contained herein, forward-looking statements by their nature involve risks and uncertainties, known and unknown, many of which are beyond the Company’s control and, as a result, actual results could differ materially from those expressed or implied in any forward-looking statement. Particular risks and uncertainties include, among other things, those related to: the Company’s available cash resources and the availability of additional funds on acceptable terms; the results of the Company’s clinical trials; the likelihood and timing of any regulatory approval of neflamapimod or the nature of any feedback the Company may receive from the U.S. Food and Drug Administration; the ability to implement business plans, forecasts, and other expectations in the future; general economic, political, business, industry, and market conditions, inflationary pressures, and geopolitical conflicts; and the other factors discussed under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 filed with the U.S. Securities and Exchange Commission (SEC) on March 29, 2024, and other filings that the Company may file from time to time with the SEC. Any forward-looking statements in this press release speak only as of the date hereof (or such earlier date as may be identified). The Company does not undertake any obligation to update such forward-looking statements to reflect events or circumstances after the date of this press release, except to the extent required by law. This press release shall not constitute an offer to sell or the solicitation of an offer to buy Company securities, nor shall there be any offer, solicitation or sale of Company securities in any jurisdiction in which such offer, solicitation or sale would be unlawful. Investor Contact: PJ KelleherLifeSci AdvisorsInvestors@cervomed.com617-430-7579 CervoMed Inc. Consolidated Balance Sheets December 31, 2023 2022 Assets Current assets: Cash and cash equivalents $7,792,846 $4,093,579 Prepaid expenses 1,256,501 64,127 Grant receivable 915,404 - Total current assets 9,964,751 4,157,706 Other assets 7,770 - Total assets $9,972,521 $4,157,706 Liabilities, Convertible Preferred Stock and Stockholders’ Equity (Deficit) Current liabilities: Accounts payable $662,471 $97,302 Accrued expenses and other current liabilities 1,933,276 644,252 Convertible notes - 12,414,000 Total liabilities 2,595,747 13,155,554 Commitments and Contingencies (Note 10) Convertible preferred stock: Series A preferred stock $0.001 par value; 30,000,000 and 0 shares authorized at December 31, 2023 and 2022, respectively, 0 shares issued and outstanding at December 31, 2023 and December 31, 2022 - - Series A-1 preferred stock, $0.001 par value; 0 and 1,960,600 shares authorized at December 31, 2023 and 2022, respectively; 0 and 1,960,600 shares issued and outstanding at December 31, 2023 and December 31, 2022, respectively - 246,849 Series A-2 preferred stock, $0.001 par value; 0 and 335,711 shares authorized at December 31, 2023 and 2022, respectively; 0 and 335,711 shares issued and outstanding at December 31, 2023 and December 31, 2022, respectively - 4,173,267 Series B preferred stock, $0.001 par value; 0 and 1,034,890 shares authorized at December 31, 2023 and 2022, respectively; 0 and 1,034,890 shares issued and outstanding at December 31, 2023 and December 31, 2022, respectively - 19,867,095 Total convertible preferred stock - 24,287,211 Stockholders’ Equity (Deficit): Common stock, $0.001 par value: 1,000,000,000 and 4,163,600 shares authorized as of December 31, 2023 and 2022, respectively, 5,674,520 and 518,140 shares issued and outstanding at December 31, 2023 and December 31, 2022, respectively 5,674 518 Additional paid-in capital 61,811,889 18,983,339 Accumulated deficit (54,440,789) (52,268,916)Total stockholders' equity (deficit) $7,376,774 (33,285,059)Total liabilities, convertible preferred stock and stockholders' equity (deficit) $9,972,521 $4,157,706 CervoMed Inc. Consolidated Statements of Operations Years Ended December 31, 2023 2022 Grant revenue $7,144,872 $- Operating expenses: Research and development 8,438,499 1,336,469 General and administrative 6,519,268 2,139,065 Total operating expenses 14,957,767 3,475,534 Loss from operations (7,812,895) (3,475,534)Other income (expense): Other income (expense) 5,421,592 (2,389,152)Interest income 219,430 62,226 Interest expense - (587)Total other income (expense) 5,641,022 (2,327,513)Net loss $(2,171,873)$(5,803,047)Per share information: Net loss per share of common stock - basic and diluted $(0.82)$(11.20)Weighted average shares outstanding - basic and diluted 2,661,416 518,140

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