12/15-LOX

Receives New Designation for Treatment of Platelet-Activating Anti-Platelet Factor 4 (PF4) Disorders Complements FDA Orphan Drug Designation and Fast Track Designation for VLX-1005 Aug. 14, 2024 -- Veralox Therapeutics, a clinical-stage biotechnology company developing first-in-class therapies targeting the 12-lipoxygenase (12-LOX) pathway, announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to VLX-1005, a small molecule 12-LOX inhibitor for the treatment of platelet-activating anti-platelet factor 4 (PF4) disorders. VLX-1005 previously secured ODD from the U.S. Food and Drug Administration for prophylaxis of thrombosis in patients with heparin-induced thrombocytopenia (HIT) as well as FDA Fast Track Designation. This latest decision in Europe represents another regulatory achievement and builds on the successful development of VLX-1005 that has progressed through completed Phase 1 clinical studies, and into the ongoing Phase 2 trial ALATHEA (“A study of VLX-1005 to evaluate thrombocyte change in HEpArin-induced thrombocytopenia”). “This EMA designation is an important step forward in the development of VLX-1005 in addressing serious diseases caused by platelet-activating anti-PF4 disorders such as HIT,” said Michael Hanna, M.D., Chief Medical Officer of Veralox Therapeutics. “This is a significant regulatory milestone for Veralox and is an important acknowledgement of the potential of VLX-1005 as treatment for HIT.” HIT is a serious, potentially life-threatening platelet disorder that can arise after taking the blood thinner heparin, which by American Heart Association and European Society of Cardiology guidelines is the standard-of-care anticoagulant for many cardiovascular procedures. Heparin is commonly used to prevent blood clots following heart surgeries, cardiopulmonary bypass and many other procedures such as kidney dialysis. In rare cases, heparin triggers a reaction that activates platelets, consuming them in a process that lowers platelet counts (thrombocytopenia) to unsafe levels while promoting clot formation, which then increases the risk for potentially fatal or life-threatening thromboembolic events. Between one quarter and one third of those who develop HIT die from the condition and a significant proportion of patients develop new blood clots, which could result in deep vein thrombosis, pulmonary embolism, heart attack or stroke. VLX-1005 has successfully completed Phase 1 clinical trials and has demonstrated a favorable safety and tolerability profile and preferred pharmacokinetics in healthy participants and is currently enrolling patients in the Phase 2 clinical study. ODD is a designation that provides incentives to advance the development of therapeutics for rare diseases, including reduced fees for multiple steps in the development process such as protocol assistance, marketing authorization applications, and 10 years of market exclusivity. In addition to the above-mentioned benefits within the European Union, member states may also offer specific stimuli for the development of orphan drugs. HIT is a rare but serious complication of heparin exposure during heart, orthopedic, vascular and other surgeries and during kidney dialysis, where heparin is commonly used to prevent blood clotting. Due to the high utilization of heparin, there are more than 300,000 suspected cases of HIT each year in the United States. Approximately 50,000 cases are confirmed each year and these patients have a high risk of developing severe consequences including death (25-30 percent); amputation (10 percent); new thrombosis including deep vein thrombosis (DVT), stroke, myocardial (MI) infarction (20 percent) and major bleeding (20 percent). The diagnosis and management of HIT is complex and a large unmet need remains for new therapies due to the limited available therapeutic options. VLX-1005 is a novel, small molecule inhibitor of 12-lipoxygenase (12-LOX) intended for the treatment or prevention of thrombosis in adults with heparin-induced thrombocytopenia (HIT). A potent and selective inhibitor of 12-LOX, VLX-1005 inhibits platelet 12-HETE production. In vivo inhibition of 12-HETE synthesis and efficacy of VLX-1005 have been shown in animal models of thrombosis and HIT and the efficacy in preventing or treating thrombosis was not accompanied by increases in bleeding. Two Phase 1 studies completed in 96 healthy participants showed favorable safety and tolerability with no deaths, no serious adverse events and no trend in AE reporting with increased dosing of VLX-1005. A Phase 2 study (VLX-1005-003) to evaluate VLX-1005 in patients with suspected HIT is currently enrolling patients across 12 clinical sites. Additional information is available at clinicaltrials.gov, identifier NCT05785819. Veralox Therapeutics Inc. is developing first-in-class therapeutics targeting 12-lipoxygenase, pioneering a new class of therapies that treat the underlying pathologies of serious immune-inflammatory diseases. The company’s lead candidate, VLX-1005, is in development for the treatment of patients with heparin-induced thrombocytopenia. VLX-1005 has previously been awarded Orphan Drug and Fast Track Designations by the U.S. Food and Drug Administration. The company has second-generation orally bioavailable 12-LOX antagonists under development for type 1 diabetes and other immune-mediated and inflammatory diseases. The content above comes from the network. if any infringement, please contact us to modify.

-- New Financing of $24 Million Fully Funds the Phase 2 Clinical Program for Lead Candidate VLX-1005 -- Company Prepares to Evaluate VLX-1005 in Heparin-Induced Thrombocytopenia (HIT) FREDERICK, Md., June 20, 2023 (GLOBE NEWSWIRE) -- Veralox Therapeutics, a clinical-stage biotechnology company developing a new class of therapies targeting the 12-lipoxygenase (12-LOX) pathway to address some of medicine’s most persistent and serious immune-mediated diseases, today announced the appointment of Jonathan Mow as the company’s new chief executive officer. Mr. Mow’s appointment comes as Veralox secured $24 million in funding to advance VLX-1005 through a Phase 2a proof-of-concept study evaluating its impact on heparin-induced thromobcytopenia (HIT), a life-threatening rare disease caused by an aberrant immune response to heparin exposure. The investment round included new investors Pappas Capital and NYBC Ventures and existing investors Hatteras Venture Partners, Sanofi Ventures, JDRF T1D Fund and Genesys Capital, amongst others. In conjunction with the financing, the company welcomes Peter Young of Pappas Capital as a director and Meg Wood of NYBC Ventures as an observer. “VLX-1005 has great promise to revolutionize the treatment of HIT and other immune-mediated diseases,” Mr. Young said. “I am thrilled to join the board at such an exciting time, and to be working with a leader of Jonathan’s caliber to move into later stages of clinical development.” “This is an exciting time for Veralox as we head into our proof-of-concept Phase 2 study with VLX-1005 for HIT, a serious complication subsequent to heparin exposure that is accompanied by significant morbidity and mortality,” Mr. Mow said. “Our novel approach with 12-LOX inhibitors has great potential in this and other diseases and I would like to thank our investors for their financial support of our important mission and giving me the opportunity to lead this world-class effort and team.” Mr. Mow brings more than 25 years of accomplishments in biotechnology management to Veralox, most recently serving as CEO of PhaseBio Pharmaceuticals. At PhaseBio, he led the company’s scientific and business transformations, guiding the company from early-stage research to Phase 3 development, and through a successful initial public offering in 2018. Earlier in his career, Mr. Mow served as vice president, business development for Amylin Pharmaceuticals until its sale to Bristol-Myers Squibb in 2012; was co-founder and vice president, commercial and business development of Corus Pharma, Inc. until its acquisition by Gilead Sciences in 2006; and headed business development for PathoGenesis Corporation until its acquisition by Chiron Corporation in 2000. Mr. Mow has also held positions in marketing, marketing research and sales at Bristol-Myers Squibb, Wyeth/Lederle International and Syntex Laboratories. He holds a B.S. in mechanical engineering from University of California at Berkeley and M.B.A. from Carnegie Mellon University’s Tepper School of Business. About Veralox TherapeuticsVeralox Therapeutics Inc. is the clinical leader in developing first-in-class therapeutics targeting 12-lipoxygenase, pioneering a new class of therapies that treat the underlying pathologies of serious immune-inflammatory diseases with unmet medical needs. The company’s lead candidate, VLX-1005, is in development for the treatment of patients with heparin-induced thrombocytopenia (HIT). VLX-1005 has orphan drug designation in the United States and has been awarded Fast Track Designation by the U.S. Food and Drug Administration. Second generation therapeutic products are under development for type 1 diabetes and other immune-mediated and inflammatory diseases. For more information, visit our website: https://veralox.com/. Media Contact: Lisa GuitermanScient PR202-330-3431Lisa.guiterman@gmail.com

SOUTH PLAINFIELD, NJ, USA I May 23, 2023 I PTC Therapeutics, Inc. (NASDAQ: PTCT ) today reported topline results from the MOVE-FA trial of vatiquinone in patients with Friedreich ataxia. The study did not meet its primary endpoint of statistically significant change in mFARS score at 72 weeks in the primary analysis population. However, vatiquinone treatment did demonstrate significant benefit on key disease subscales and secondary endpoints. In addition, in the population of subjects that completed the study protocol, significance was reached in the mFARS endpoint and several secondary endpoints. "While we are disappointed that the study did not achieve its primary endpoint, we are encouraged by the findings of meaningful impact on several different aspects of FA disease progression and morbidity over 72 weeks," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "Given the signals of clinical benefit, vatiquinone's well-established safety profile in children, and the unmet medical need for pediatric patients with FA, we look forward to discussing a potential path to registration with regulatory authorities." The MOVE-FA trial enrolled 146 pediatric and adult patients, the majority of which were under 18 years of age. The mean placebo corrected change in the mFARS score in the primary analysis population was 1.6 (p=0.14). Notably, there was significant benefit recorded in the bulbar and upright stability subscales (nominal p values of 0.044 and 0.021, respectively) which are regarded as reflective of key aspects of disease morbidity and predictive of loss of time to loss of ambulation. In addition, a statistically significant difference was recorded on the Modified Fatigue Scale, which captures one of the most impactful sources of disease morbidity (nominal p value of 0.025). On a prespecified sensitivity analysis of subjects who completed 72 weeks on assigned therapy, the placebo corrected difference was 2.31, which represents a 75% slowing in disease progression over 72 weeks. Overall, vatiquinone was demonstrated to be well tolerated, adding to the large volume of safety data collected in other pediatric clinical studies. Conference Call and Webcast Details: PTC will hold a conference call at 5:00 pm EDT today to discuss this news. To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays, we recommend participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at https://ir.ptcbio.com/events-presentations . A replay of the call will be available approximately two hours after completion of the call and will be archived on the company's website for 30 days following the call. About the MOVE-FA Clinical Trial The Phase 3 registration-directed trial in Friedreich Ataxia patients, called MOVE-FA, is a randomized, placebo-controlled 72-week trial with the primary endpoint being change in the modified Friedreich Ataxia Rating Scale (mFARS) score. The mFARS is a clinical assessment that measures disease progression, namely swallowing and speech, upper and lower limb coordination, and upright stability. The key secondary endpoint is the change from baseline in activities of daily living as assessed by the FA-Activities of Daily Living (ADL) scale. Patients who completed the placebo portion of the trial will be trial are eligible to enroll in an open label 24-week extension. About Vatiquinone PTC is developing vatiquinone, a potential treatment for Friedreich ataxia based on our Bio-e platform. Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich ataxia. Inhibition of 15-LO helps to alleviate the consequences of mitochondrial dysfunction and oxidative stress, ultimately preventing ferroptosis and aiding neuronal survival. 1,2,3 Vatiquinone has been evaluated in a number of clinical studies and has demonstrated an impact on mortality risk and a number of neurological and neuromuscular disease symptoms. About Friedreich Ataxia Friedreich ataxia (FA) is a rare, physically debilitating, life-shortening, neuromuscular disorder that mainly affects the central nervous system and the heart. 4 It is the most common hereditary ataxia (abnormal, uncoordinated movements) and is usually caused by a single genetic defect in the frataxin (FXN) gene that leads to reduced production of frataxin, a mitochondrial protein that is important for cellular metabolism and energy production .4,5 Decreased frataxin levels are associated with mitochondrial iron accumulation and increased oxidative stress, which can lead to cell death through ferroptosis. 6,7,8 Symptoms include progressive loss of coordination and muscle strength leading to poor balance and coordination, difficulty speaking, swallowing, and breathing, curvature of the spine, serious heart conditions, diabetes, and hearing and vision impairment. 9,10 The severity of symptoms and speed of progression varies between people and some symptoms may not be evident in all. Friedreich ataxia is usually diagnosed in childhood or adolescence. 5,11 Approximately 25,000 people have Friedreich ataxia globally. About PTC Therapeutics, Inc. PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, Instagram, LinkedIn and Twitter at @PTCBio. References: SOURCE: PTC Therapeutics