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Nantes, France--(News - January 3, 2024) - InFlectis BioScience, a pioneer in developing first-in-class therapies for neuromuscular diseases, announced that it has been awarded a $943,000 grant to support recruitment and to investigate biomarkers in its Phase 2 clinical trial in patients with bulbar-onset ALS (NCT05508074). The trial is investigating IFB-088 (INN: icerguastat), a multifunctional drug that modulates the PPP1R15A/PP1c phosphatase complex and inhibits NR2B-containing NMDAR. The potential of IFB-088 lies in its ability to target three major ALS cellular pathophysiological mechanisms to manage disease progression: protein aggregation, oxidative stress, and glutamate excitotoxicity. To view the full announcement, including downloadable images, bios, and more, click here. Key Takeaways: InFlectis Bioscience has been awarded a $943K grant from the ALS Association to support the recruitment of 50 patients by the end of January 2024 for its Phase 2 clinical trial evaluating IFB-088. The grant also supports a multi-omics approach, conducted by the IRCCS "Carlo Besta" Neurological Institute in Milan, Italy to investigate pharmacodynamic biomarker candidates, which will be useful in a future pivotal clinical study. The Phase 2 clinical study results will be reported by the end of 2024. Click image above to view full announcement. About InFlectis BioScience InFlectis is a France-based drug discovery company developing first-in-class therapies for neuromuscular diseases. We are in clinical development with an orally available small molecule that has the potential to extend lives or improve the quality-of-life for people suffering from ALS and CMT, a rare and progressive hereditary nerve condition. IRCCS Foundation "Carlo Besta" Neurological Institute The Foundation of the Carlo Besta Neurological Institute (FINCB) is a "Foundation of Participation" including as Members of the Board of Governors representatives of the Italian Ministry of Health, the Region of Lombardy and the Municipality of Milan. FINCB is recognized by the Italian Ministry of Health as a Scientific Institute for Research, Hospitalization and Health Care (IRCCS) of public body. It is a national and international centre of excellence for the treatment of neurological diseases, and also undertakes research aimed to improve the diagnosis and treatment of these diseases. Its major lines of activities in patients' care and research encompass child neurology, epilepsy, neuro-oncology, neuropathic pain, and genetic, cerebrovascular, neuroimmunological, neuromuscular and neurodegenerative disorders. The fundamental aims of FINCB are to provide high quality, ethical and efficient care and treatment to its patients, making use of the results of its own research and that of others, always putting the interests of patient first. About ALS Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. Over the course of the disease, people lose the ability to move, to speak, and eventually, to breathe. On average, it takes about a year before a final ALS diagnosis is made. The disease is always fatal, usually within five years of diagnosis. There is no cure. Contacts: Philippe Gu'edat philippeguedat@inflectisbioscience.com Source: Inflectis To view the source version of this press release, please visit

Company Today Also Provided Updates on R&D Portfolio and Strategy at Investor Event Crinecerfont New Drug Application Submission Planned in 2024 NBI-'770, an Oral NMDA NR2B Negative Allosteric Modulator, Entering Phase 2 for the Treatment of Major Depressive Disorder Advancing Largest Portfolio of Muscarinic Compounds in Clinical Development Top-Line Phase 2 Data Readouts for Five Programs Anticipated in 2024 Including NBI-'568, an M4 Agonist, for the Treatment of Schizophrenia SAN DIEGO, Dec. 5, 2023 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced it received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for crinecerfont in congenital adrenal hyperplasia. The Company also provided updates on its R&D portfolio and strategy at its Analyst Day, held in New York. "We are very pleased that the FDA granted Breakthrough Therapy designation for crinecerfont, thus recognizing both the seriousness of congenital adrenal hyperplasia and the significant unmet need currently faced by patients and families living with this condition," said Eiry W. Roberts, Chief Medical Officer, Neurocrine Biosciences. "The outstanding safety and efficacy results from the Phase 3 CAHtalyst™ studies in pediatric and adult patients suggest that crinecerfont has the potential to represent a substantial improvement over current standard of care in CAH by controlling androgen levels and allowing for reduced steroid doses. We remain on track to submit the new drug application in 2024." 2023 Analyst Day R&D Portfolio and Strategy Updates At today's Analyst Day, Neurocrine Biosciences provided an update on its R&D portfolio and strategy, including the Company's focus on building breadth and depth across therapeutic areas and modalities with key focus areas in VMAT2 inhibition, CRF antagonism, muscarinic agonism to antagonism, and gene therapy. With a diversified early-stage portfolio spanning a range of modalities including small molecules and biologics, and a growing pre-clinical and development candidate portfolio, Neurocrine is uniquely positioned to advance a steady flow of innovative clinical candidates to patients across its neuroscience-focused therapeutic areas of interest. The Company remains on-track to advance two gene therapies into the clinic in 2025, and anticipates at least 20 development candidates by 2027. Other key portfolio and pipeline updates include: Valbenazine Ongoing Phase 3 study of valbenazine in adjunctive treatment of schizophrenia (ATS) will inform potential advancement of NBI-'890, a next-generation, long-acting VMAT2 inhibitor, which we anticipate will enter the clinic in 2024 No additional Phase 3 studies of valbenazine in ATS planned NBI-'770 (Oral, NMDA NR2B Negative Allosteric Modulator) Represents a potential first-in-class opportunity on a clinically validated target Program entering Phase 2 for treatment of major depressive disorder Muscarinic Compounds Validated and differentiated mechanisms of action Five programs represent largest number of muscarinic assets in human studies, including lead molecule, NBI-'568, in Phase 2 study for the treatment of schizophrenia 2024 portfolio will include four Phase 1 programs: NBI-'570 (dual M1/M4 agonist) NBI-'567 (M1-preferring agonist) NBI-'569 (M4-preferring agonist) NBI-'986 (M4 antagonist, internally discovered at Neurocrine Biosciences) Phase 2 Top-Line Clinical Data Expected in 2024 NBI-'568 (M4 agonist) for the treatment of schizophrenia Luvadaxistat (DAAO inhibitor) for cognitive impairment associated with schizophrenia NBI-'845 (AMPA potentiator) for inadequate response to treatment in major depressive disorder Efmody (long-acting glucocorticoid) for adrenal insufficiency Efmody for congenital adrenal hyperplasia About Breakthrough Therapy Designation Breakthrough Therapy designation is a process developed by the FDA to expedite development and review of drugs that are intended to treat a serious condition and where clinical evidence indicates that the potential drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). To learn more about Breakthrough Therapy designation, click here. About Classic Congenital Adrenal Hyperplasia Congenital adrenal hyperplasia (CAH) refers to a group of genetic conditions that result in an enzyme deficiency that alters the production of adrenal hormones which are essential for life. Approximately 95% of CAH cases are caused by a mutation that leads to deficiency of the enzyme 21-hydroxylase (21-OHD). In classic CAH, severe deficiency of this enzyme leads to an inability of the adrenal glands to produce cortisol and, in approximately 75% of cases, aldosterone. If left untreated, classic CAH can result in salt wasting, dehydration, and even death. There are currently no non-glucocorticoid treatments approved by the U.S. Food and Drug Administration (FDA) for classic CAH. Glucocorticoids (GCs), the current standard of care, are used not only to correct the endogenous cortisol deficiency but typically used at greater than physiologic (supraphysiologic) doses to try to suppress the high levels of corticotropin-releasing factor (CRF) and adrenocorticotropic hormone (ACTH) that result in androgen excess. However, glucocorticoid treatment at supraphysiologic doses has been associated with serious and significant complications of steroid excess, including metabolic issues such as weight gain and diabetes, cardiovascular disease, and osteoporosis. Additionally, long-term treatment with supraphysiologic GC doses may have psychological and cognitive impact such as changes in mood and memory. Androgen excess has been associated with abnormal bone growth and development in pediatric patients, female health problems such as acne, excess hair growth and menstrual irregularities, testicular rest tumors in males, and fertility issues in both sexes. To learn more about CAH, click here. About Corticotropin Releasing Factor (CRF) Corticotropin releasing factor (CRF) regulates the secretion of adrenocorticotropic hormone (ACTH) by the pituitary gland. The neuropeptide was discovered, isolated and identified by the late Wylie W. Vale, PhD., a founder of Neurocrine Biosciences who led the Clayton Foundation Laboratories for Peptide Biology and was the Helen McLoraine Chair in Molecular Neurobiology at the Salk Institute. Dr. Vale mapped the key role CRF played in the regulation of the stress response. In the 95% of classic CAH cases caused by a mutation in the 21-hydroxylase enzyme, the severe enzyme deficiency results in little or no production of the stress hormone cortisol, which can lead to adrenal crisis. The lack of cortisol production also causes a break in the feedback loop that would normally regulate the body's secretion of CRF, and as a result ACTH, which maintains adrenal androgen production within normal ranges. About Crinecerfont Crinecerfont is an investigational, oral, selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist being developed to reduce and control excess adrenal androgens through a steroid-independent mechanism for the treatment of congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21-OHD). The compound was identified by Dimitri Grigoriadis, Ph.D., Chief Research Officer at Neurocrine Biosciences who conducted post-graduate research with Dr. Wylie Vale at the Salk Institute. Antagonism of CRF1 receptors in the pituitary has been shown to decrease adrenocorticotropic hormone (ACTH) levels, which in turn decreases the production of adrenal androgens and potentially the symptoms associated with classic CAH. Our data demonstrates that lowering androgen levels enables lower, more physiologic dosing of glucocorticoids and thus could potentially reduce the complications associated with exposure to greater than normal glucocorticoid doses in patients with classic CAH. Prior to receiving Breakthrough Therapy designation from the FDA, crinecerfont also received Fast Track and Rare Pediatric Disease designations in congenital adrenal hyperplasia. To learn more about crinecerfont, click here. About the CAHtalyst Phase 3 Studies The CAHtalyst Phase 3 Pediatric and Adult global registrational studies were designed to evaluate the safety, efficacy, and tolerability of crinecerfont in children and adolescents (2–17 years of age) and adults (18 years of age and older) with classic congenital adrenal hyperplasia (CAH) due to 21-OHD, respectively. The CAHtalyst Phase 3 Pediatric and Adult clinical studies met their primary and secondary endpoints. The CAHtalyst Pediatric study demonstrated a statistically significant decrease in serum androstenedione from baseline with crinecerfont at Week 4 over baseline, while both studies showed crinecerfont treatment led to statistically significant reductions in daily glucocorticoid from baseline while maintaining androgen control at Week 28 and Week 24, respectively. Crinecerfont was generally well tolerated in the CAHtalyst Phase 3 studies. The most common adverse events in the CAHtalyst Pediatric study were headache, fever, vomiting, upper respiratory tract infection, and nasopharyngitis, while the most common adverse events in the CAHtalyst Adult study were fatigue, headache, and coronavirus infection. Few serious adverse events were seen in either study, with none assessed as related to crinecerfont. Both studies achieved a >95% completion rate. Details about the Phase 3 data from the CAHtalyst Pediatric Study were reported in October 2023 and can be found here. For more information about the CAHtalyst Pediatric Phase 3 study, please visit ClinicalTrialsPediatric.gov. Details about the Phase 3 data from the CAHtalyst Adult Study were reported in September 2023 and can be found here. For more information about the CAHtalyst Phase 3 study in adults, please visit ClinicalTrialsAdult.gov. About Neurocrine Biosciences, Inc. Neurocrine Biosciences is a leading neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs, but few options. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, neuroendocrine and neuropsychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, Parkinson's disease, endometriosis* and uterine fibroids*, as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders, because you deserve brave science. For more information, visit neurocrine.com, and follow the company on LinkedIn, Twitter, and Facebook. (*in collaboration with AbbVie) NEUROCRINE BIOSCIENCES and NEUROCRINE are registered trademark of Neurocrine Biosciences, Inc. CAHtalyst and the Neurocrine logo are trademarks of Neurocrine Biosciences, Inc. Forward-Looking Statement In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements regarding the potential benefits to be derived from certain of our product candidates and our future development plans. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements include: our future financial and operating performance; the risk that our products and/or product candidates will not be found to be safe and/or effective or may not prove to be beneficial to patients; that development activities for our products and/or product candidates may not be completed on time or at all; risks that clinical development activities may be delayed for regulatory or other reasons, may not be successful or replicate previous and/or interim clinical trial results, or may not be predictive of real-world results or of results in subsequent clinical trials; risks that regulatory submissions for our products and/or product candidates may not occur or be submitted in a timely manner; risks that our products and/or product candidates may not obtain regulatory approvals; or that the U.S. Food and Drug Administration or regulatory authorities outside the U.S. may make adverse decisions regarding our products and/or product candidates; risks and uncertainties relating to competitive products and technological changes that may limit demand for our products; risks associated with our dependence on third parties for development and manufacturing activities related to our products and our product candidates, and our ability to manage these third parties; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended September 30, 2023. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof. SOURCE Neurocrine Biosciences, Inc.

Algernon Pharmaceuticals Inc. (the “Company” or “Algernon”) (CSE: AGN) (FRANKFURT: AGW0) (OTCQB: AGNPF), a clinical stage pharmaceutical development company, is pleased to announce that it has engaged Maxim Group LLC (“Maxim”) to provide financial advisory and investment banking services, and to assist in identifying and evaluating potential M&A and strategic opportunities, including the potential spin-off of the Company's NP-120 (“Ifenprodil”) chronic cough research program. Ifenprodil is an N-methyl-D-aspartate (NMDA) receptor antagonist specifically targeting the NMDA-type subunit 2B (GluN2B), which prevents glutamate signaling. Ifenprodil represents a novel first-in-class potential treatment for chronic cough and is thought to interfere with central signaling in the brain, suppressing the urge to cough. “We are very pleased to be working with Maxim, and we are especially interested in the potential to achieve significant value for our Ifenprodil asset and for our shareholders, through a spin-off transaction,” said Christopher J. Moreau, Algernon’s Chief Executive Officer. “Ifenprodil represents a novel first-in-class potential treatment for chronic cough, and we are only one Phase 2b study away from the stage of development where Bellus’ camlipixant was acquired by GSK and Afferent’s gefapixant was acquired by Merck.” Algernon’s Phase 2a Study Data The decision to advance to a Phase 2b cough study was based on positive data previously reported from the Company’s proof of concept Phase 2a study of idiopathic pulmonary fibrosis (“IPF”) and chronic cough on July 28, 2022, where Ifenprodil showed a significant reduction in cough count. Patients with IPF are usually excluded from trials in refractory chronic cough (“RCC”), and cough in this population is regarded as extremely difficult to treat. Further analysis revealed: The geometric mean 24-hour cough counts were reduced by 32.0% at 4 weeks (p = 0.023) and 39.5% at 12 weeks (p = 0.001) compared to baseline The geometric mean awake cough counts were reduced by 30.2% at 4 weeks (p = 0.038) and 37.4% at 12 weeks (p = 0.002) compared to baseline Algernon previously announced on January 14, 2022, that it had received positive feedback from the U.S. Food and Drug Administration at its pre-Investigational New Drug (pre-IND) meeting for its investigation of Ifenprodil solely for the treatment of chronic cough. Chronic Cough Market According to Data Bridge Market Research analyses, the global chronic cough market was valued at USD $6.15B in 2021 and is projected to grow up to USD $11.38B by 2029. Merck & Co. obtained the rights to gefapixant, a P2X3 receptor antagonist, as the lead asset in the acquisition of Afferent Pharmaceuticals in 2016. At the time, gefapixant had interim data from a Phase 2b dose-escalation study in RCC. The deal was worth up to USD $1.25 billion. Bellus Health, which is advancing camlipixant, its own novel P2X3 receptor antagonist, announced on April 18, 2023, that GSK plc signed a deal where they agreed to buy all the outstanding shares in Bellus for USD $14.75 per share in cash with a total deal value estimated at USD $2 billion, confirming the need for better therapies for chronic cough. Algernon’s Chronic Cough Advisors Dr. Jacky Smith is a Professor of Respiratory Medicine at the University of Manchester and an Honorary Consultant at Manchester University NHS Foundation Trust. She runs a multi-disciplinary research team whose focus is on understanding mechanisms underlying pathological cough and a regional clinical service seeing patients with refractory chronic cough. Dr. Smith is also the lead medical and scientific advisor to both Merck and Bellus Health for their RCC research programs. Dr. Peter Dicpinigaitis is board-certified in Internal Medicine, Pulmonary Diseases and Critical Care Medicine. He is a faculty member of the Division of Critical Care Medicine at Montefiore Medical Center and is the founder and director of the Montefiore Cough Center, one of the few specialty centers in the world exclusively committed to the evaluation and management of patients with chronic cough. About Chronic Cough Chronic cough is defined as a cough lasting for more than eight weeks in duration and in the United States cough continues to be one of the most common reasons that adults consult medical doctors. Some cases of chronic cough are so debilitating that quality of life is severely impacted leading to depression, anxiety, urinary incontinence, dysphonia, sleep interruption, vomiting, and even rib fractures further adding to the decay in socio-familial dynamics. Chronic cough is believed to be the result of a hypersensitivity of the cough reflex within the neuronal circuitry that governs the urge to cough, wherein one or more aspects that regulate cough are over-active to stimulus, triggering a cough at abnormal levels. Trials of cough suppressants (Antitussives) have shown differences in response that may reflect differing pathological processes driving cough in different patients. Experimental Antitussives often only engage a single receptor, while the overall cough response is governed by multiple receptors triggered by a large variety of stimuli. A compound acting centrally, like ifenprodil, where all peripheral messages are sent and coordinated, may achieve a better outcome than what has been achieved in clinical trials. About NP-120 (Ifenprodil) Ifenprodil selectively inhibits N-methyl-D-aspartate (“NMDA”) receptors containing the NR2B subunit. They are highly implicated in events such as neuronal plasticity (strengthening of neural pathways) and excitotoxicity (neurotoxic cascade resulting in neuron death). By inhibiting NMDA receptors, Ifenprodil can diminish excitability of neurons and prevent the relaying of information along neuronal circuitry, including the cough reflex. Ifenprodil may also inhibit the neuroplastic enhancement of central and peripheral cough response neurons. About Maxim Group LLC Founded in 2002, Maxim Group is a leading full-service investment bank, securities and wealth management firm headquartered in mid-town Manhattan providing a comprehensive array of financial services including investment banking, global institutional sales, equity research, fixed income and derivative sales & trading, merchant capital, private wealth management, and prime brokerage services to a diverse range of corporate clients, institutional investors and high-net-worth individuals. Maxim Group is a registered broker-dealer with the U.S. Securities and Exchange Commission and the Municipal Securities Rulemaking Board (MSRB). About Algernon Pharmaceuticals Algernon Pharmaceuticals is a Canadian clinical stage drug development and repurposing company investigating multiple drugs for unmet global medical needs. Algernon Pharmaceuticals has active research programs for IPF with chronic cough, and chronic kidney disease, and is the parent company of a newly created private subsidiary called Algernon NeuroScience, that is advancing a psychedelic program investigating a proprietary form of psychedelic DMT for stroke and traumatic brain injury (“TBI”). 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