Pharma Tech
Globenewswire | August 02, 2023
Tonix Pharmaceuticals Holding Corp. a biopharmaceutical company, announced that it has completed enrollment of its potentially final, confirmatory Phase 3 RESILIENT trial of TNX-102 SL (cyclobenzaprine HCL sublingual tablets) 5.6 mg in fibromyalgia and expects topline data next quarter. A total of 457 participants were randomized. TNX-102 SL is in development as a non-opioid, centrally acting analgesic, to be taken daily at bedtime for the management of fibromyalgia. If successful, we believe this will be the final, well-controlled efficacy trial required for submission of a New Drug Application (NDA) for approval by the U.S. Food and Drug Administration (FDA).
“The completion of enrollment in our Phase 3 RESILIENT trial is a significant milestone for both Tonix and the fibromyalgia community,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “Currently-approved treatments have not fully met the needs of fibromyalgia patients and there has not been a new FDA-approved therapy for the condition since 2009. TNX-102 SL has the potential to be a new non-addictive, non-opioid bedtime medication with broad spectrum symptom coverage and which can be used on a chronic basis for the management of fibromyalgia. With all other clinical, nonclinical and CMC requirements for an NDA submission achieved, we are looking forward to the upcoming data readout and an expeditious filing of an NDA.”
In December 2020, Tonix reported positive results from the first Phase 3 study (RELIEF) of TNX-102 SL 5.6 mg for the management of fibromyalgia. TNX-102 SL met its pre-specified primary endpoint in the Phase 3 RELIEF trial, significantly reducing daily pain compared to placebo (p=0.01) in participants with fibromyalgia. Also, when the primary endpoint was analyzed as a ≥30% pain responder analysis, there was a higher rate of responders to TNX-102 SL (47%) than to placebo (35%; p=0.006). TNX-102 SL at 5.6 mg also showed activity in key secondary endpoints demonstrating improvements in sleep quality, mitigation of fatigue, and fibromyalgia-specific global symptomatic and functional recovery. TNX-102 SL was generally safe and well tolerated in patients with fibromyalgia, with overall adverse event profile comparable to prior fibromyalgia studies. The most common treatment-emergent adverse events were oral hypoesthesia, oral paresthesia, and product taste abnormal.
About the Phase 3 RESILIENT Study
The RESILIENT study is a double-blind, randomized, placebo-controlled trial designed to evaluate the efficacy and safety of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) in the management of fibromyalgia. The two-arm trial randomized 457 participants in the U.S. The first two weeks of treatment consist of a run-in period in which participants start on TNX-102 SL 2.8 mg (1 tablet) or placebo. Thereafter, all participants increase their dose to TNX-102 SL 5.6 mg (2 x 2.8 mg tablets) or two placebo tablets for the remaining 12 weeks. The primary endpoint is the daily diary pain severity score change (TNX-102 SL 5.6 mg vs. placebo) from baseline to Week 14 (using the weekly averages of the daily numerical rating scale scores), analyzed by mixed model repeated measures with multiple imputation.
About Fibromyalgia
Fibromyalgia is a chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system. Fibromyalgia afflicts an estimated 6-12 million adults in the U.S., approximately 90% of whom are women. Symptoms of fibromyalgia include chronic widespread pain, nonrestorative sleep, fatigue, and morning stiffness. Other associated symptoms include cognitive dysfunction and mood disturbances, including anxiety and depression. Individuals suffering from fibromyalgia struggle with their daily activities, have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed products.
About TNX-102 SL
TNX-102 SL is a patented sublingual tablet formulation of cyclobenzaprine hydrochloride which provides rapid transmucosal absorption and reduced production of a long half-life active metabolite, norcyclobenzaprine, due to bypass of first-pass hepatic metabolism. As a multifunctional agent with potent binding and antagonist activities at the 5-HT2A-serotonergic, α1-adrenergic, H1-histaminergic, and M1-muscarinic receptors, TNX-102 SL is in development as a daily bedtime treatment for fibromyalgia, Long COVID (formally known as post-acute sequelae of COVID-19 [PASC]), alcohol use disorder and agitation in Alzheimer’s disease. The United States Patent and Trademark Office (USPTO) issued United States Patent No. 9636408 in May 2017, Patent No. 9956188 in May 2018, Patent No. 10117936 in November 2018, Patent No. 10,357,465 in July 2019, and Patent No. 10736859 in August 2020. The Protectic™ protective eutectic and Angstro-Technology™ formulation claimed in the patent are important elements of Tonix’s proprietary TNX-102 SL composition. These patents are expected to provide TNX-102 SL, upon NDA approval, with U.S. market exclusivity until 2034/2035.
Tonix Pharmaceuticals Holding Corp.
Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering. Tonix markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg. Zembrace SymTouch and Tosymra are each indicated for the treatment of acute migraine with or without aura in adults. Tonix’s development portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS development portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia, having completed enrollment in a potentially registration-enabling study, with topline data expected in the fourth quarter of 2023. TNX-102 SL is also being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Enrollment in a Phase 2 study has been completed, and topline results are expected in the third quarter of 2023.
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Pharmacy Market
businesswire | August 25, 2023
Novaliq GmbH, a biopharmaceutical company focusing on first- and best-in-class ocular therapeutics, announced that the European Medicines Agency (EMA) has accepted the marketing authorization application (MAA) for CyclASol (ciclosporin ophthalmic solution) for regulatory review. CyclASol is an investigational new drug treatment for dry eye disease in patients not responding to tear substitutes. The Committee for Medicinal Products for Human Use (CHMP) of the EMA will start its review of the MAA under the centralized procedure for all 27 member states of the European Union (EU).
Dry eye disease (DED) affects millions of people in Europe and is one of the most common ocular surface disorders. A leading cause of DED is inflammation of the ocular surface. The chronic inflammatory nature causes progressive corneal surface damage that can lead to direct or indirect visual impairment. In the EU only one drug therapy limited to a subset of DED patients with severe keratitis is currently approved.
CyclASol is ciclosporin, solubilized in a novel, water-free excipient and was designed to address unmet needs, providing patients with a fast acting and well tolerable dry eye drug therapy. Dispensed in a unique 10 microliter small drop the solution does not contain water or anti-microbial preservatives, oils or surfactants. As a water-free product, there is no associated pH and no osmolarity.
“Our in Heidelberg, Germany, invented water-free technology was designed to unfold the full potential of pharmaceuticals on the ocular surface,” said Christian Roesky, Ph.D., Chief Executive Officer of Novaliq. “The acceptance of the EU Marketing Authorization Application is a key milestone in our global efforts to address high unmet needs to better serve patients suffering from dry eye disease. We look forward to collaborating with the CHMP throughout the review process and hope to make CyclASol available for patients in Europe.”
The clinical development program for the MAA is based on six clinical studies, which evaluated the safety and efficacy of CyclASol in a total of 1,575 patients with dry eye disease. Results from the two registrational studies, ESSENCE-1 and ESSENCE-2 consistently demonstrated that the product is effective for the treatment of DED. Continued improvement under therapy in both signs and symptoms of DED has been clinically demonstrated over a period of up to 56 weeks in an extension study of ESSENCE‑2. With its early onset of effect and its good tolerability profile CyclASol addresses unmet medical needs in DED.
CyclASol was approved by the U.S. Food and Drug Administration (FDA) as VEVYE™ (cyclosporine ophthalmic solution) 0.1% for the treatment of the signs and symptoms of dry eye disease on May 30, 2023. VEVYE is the first and only FDA approved cyclosporine solution indicated for the treatment of signs and symptoms of dry eye disease with efficacy demonstrated after 4 weeks of treatment and is commercialized in Northern America by Harrow Health Inc, a leading U.S. eyecare pharmaceutical company.
About Novaliq
Novaliq is a private biopharmaceutical company focusing on the development and commercialization of first- and best-in-class ocular therapeutics based on EyeSol® the worldwide first water-free ophthalmic technology.
Novaliq offers an industry-leading portfolio addressing today's unmet medical needs of millions of patients with eye diseases based on its proprietary water-free eyedrop technology. On May 18th, 2023, U.S. Food and Drug Administration (FDA) approved MIEBO™ (perfluorohexyloctane ophthalmic solution; formerly known as NOV03) followed by the approval of VEVYE™ (cyclosporine ophthalmic solution, development name CyclASol®) 0.1% on May 30th, 2023.
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Pharmacy Market
PRNewswire | July 18, 2023
Karyopharm Therapeutics Inc. a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced that the United States Food and Drug Administration (FDA) has granted Fast Track Designation to the development program of selinexor for the treatment of patients with myelofibrosis, including primary myelofibrosis, post-essential thrombocythemia myelofibrosis, and post-polycythemia vera myelofibrosis.
"Fast Track Designation for selinexor highlights its potential to address the unmet medical need in myelofibrosis, an important acknowledgement as we continue our pivotal Phase 3 study," said Reshma Rangwala, MD, PhD, Chief Medical Officer of Karyopharm. "Selinexor's unique mechanism of action, XPO1 inhibition, is a novel and potentially fundamental mechanism in myelofibrosis. We have been highly encouraged by the efficacy and safety data observed to date [in our Phase 1 study] with selinexor in combination with ruxolitinib in patients with treatment-naïve myelofibrosis and believe selinexor has the potential to shift the treatment paradigm. We look forward to continued interaction with the FDA as we advance the development of this promising treatment for patients in need."
In June 2023, Karyopharm initiated a pivotal Phase 3 clinical trial to assess the efficacy and safety of once-weekly selinexor 60 mg in combination with ruxolitinib in JAKi-naïve patients with myelofibrosis. Updated data from the Phase 1 study were presented at the American Association for Cancer Research Annual Meeting 2023, American Society of Clinical Oncology 2023 and European Hematology Association 2023, which showed rapid, deep and sustained spleen responses and robust symptom improvement in patients treated with selinexor 60 mg in combination with ruxolitinib as of the April 10, 2023 cut-off date. Top-line data from the Phase 3 study is expected in 2025. The Company plans to expand its clinical development program in myelofibrosis by investigating selinexor in other JAKi-naïve settings, such as novel combinations, to benefit the greatest number of patients.
Fast Track Designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously. Features of Fast Track Designation include frequent interactions with the FDA review team, and if relevant criteria are met, eligibility for Priority Review and Rolling Review.
About Karyopharm Therapeutics
Karyopharm Therapeutics Inc. is a commercial-stage pharmaceutical company pioneering novel cancer therapies. Since its founding, Karyopharm has been an industry leader in oral Selective Inhibitor of Nuclear Export (SINE) compound technology, which was developed to address a fundamental mechanism of oncogenesis: nuclear export dysregulation. Karyopharm's lead SINE compound and first-in-class, oral exportin 1 (XPO1) inhibitor, XPOVIO® (selinexor), is approved in the U.S. and marketed by the Company in three oncology indications and has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including Europe and the United Kingdom (as NEXPOVIO®) and China. Karyopharm has a focused pipeline targeting multiple high unmet need cancer indications, including in multiple myeloma, endometrial cancer, myelodysplastic neoplasms and myelofibrosis.
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