Porphyrias

Agreement includes a single digit million US$ upfront payment and additional funding that has the potential to cover the cost of studies through Phase 1b Deal is a major step forward for Fibrocor's plans to develop FIB918, its monoclonal antibody for Alport Syndrome, a rare disease with very limited treatment options Fibrocor and MSRD intend to start Phase 1 clinical trials of its monoclonal antibody in 2025 TORONTO, March 5, 2024 /PRNewswire/ -- Fibrocor Therapeutics Inc. ('Fibrocor' or 'the Company'), a privately held developer of novel therapeutics to treat fibrosis, today announces that it has entered into a research and development collaboration with the McQuade Center for Strategic Research and Development, LLC (MSRD) to advance its Alport Syndrome program. The agreement includes a single digit million US$ upfront payment and additional funding that has the potential to cover the cost of studies through Phase 1b. MSRD, a member of the Otsuka family of pharmaceutical companies, identifies and supports early-stage opportunities that can change the landscape of treatments for mental illness and renal disorders. FIB918 is now in the IND-enabling phase and Phase 1 clinical trials of the monoclonal antibody are targeted to start in late 2025. Fibrocor and MSRD will be working with patients, advocacy groups and regulators to execute safe, effective and inclusive clinical trials that take patient input and lifestyle into consideration, aiming to positively shift the clinical and treatment paradigm for patients. William P. Newsome III, CEO, President of Fibrocor said: "This collaboration with the experienced team at MSRD, coupled with research funding, represents continued validation of Fibrocor's translational, best-in-class, target discovery platform. It represents hope and potential for individuals grappling with Alport Syndrome worldwide. FIB918 has the potential to deliver a significant advancement in the treatment of patients afflicted by this debilitating disease, where current options are limited. The completion of our Investigational New Drug (IND) application and the anticipated commencement of clinical trials in 2025, mark crucial milestones in our journey to make a meaningful impact on the lives of those affected by Alport Syndrome." Piet Wigerinck, Chief Scientific Advisor of Fibrocor added, "This collaboration serves as further affirmation of our platform, aligning committed teams that are backed by world class experts and substantial resources. It propels our ongoing commitment to advancing our small molecule program, with FIB992 poised for entry into the clinic later this year for idiopathic pulmonary fibrosis (IPF), another debilitating, deadly and underserved fibrotic disease. This will be followed by the initiation of clinical trials for FIB991 for chronic allograft nephropathy in 2025. We have an unwavering dedication to making a meaningful impact on patients grappling with fibrotic diseases. We eagerly anticipate a successful collaboration with our colleagues at MSRD." Alport Syndrome is a rare form of kidney inflammation caused by specific gene mutations, resulting in structural defects and the development of renal fibrosis and kidney failure. About one in 50,000 people is thought to suffer from it, although estimates vary widely. Most experience kidney failure as young adults or by middle age. Idiopathic pulmonary fibrosis (IPF) is a progressive and generally fatal disease characterized by scarring of the lungs that thickens the lining of the lungs, causing an irreversible loss of the tissue's ability to transport oxygen. IPF ultimately robs a patient of the ability to breathe. The median survival of IPF patients is reported to range from two to five years. IPF Foundation | Ending IPF Through Research About Fibrocor Therapeutics: Fibrocor Therapeutics is redefining the landscape of fibrotic diseases through our unique portfolio of novel compounds against biologically and clinically validated targets. Leveraging our extensive expertise and one of the largest patient-derived clinically annotated biorepositories of fibrotic tissue, we strive to improve the lives of patients by delivering transformative treatments that tackle the root causes of fibrosis, leading to better outcomes and enhanced quality of life. As we advance from the preclinical stage towards clinical trials and beyond, we remain dedicated to collaborating with patients, healthcare professionals, and industry partners to continue to refine and validate our therapeutic candidates, ensuring they meet the highest standards of efficacy and safety. Our vision is to disrupt the status quo and provide hope to those affected by fibrotic diseases aiming to reshape the future of fibrosis treatment, ultimately making a meaningful difference in the lives of millions. For more information visit SOURCE Fibrocor Therapeutics

MALIBU, Calif., March 04, 2024 (GLOBE NEWSWIRE) -- Leading biotech company CLINUVEL last week joined an intimate event bringing together California’s most prolific investors and philanthropists at the Malibu home of Lady Gaga and Michael Polansky. The event, hosted by Lady Gaga and Mr. Polansky, and Sean Parker and Alexandra Parker, shone a light on the groundbreaking work of CLINUVEL, a global biopharmaceutical firm whose cutting-edge melanocortin therapies transform the lives of patients at the highest risk of photodamage and skin cancer. Mr. Parker and Mr. Polansky have supported CLINUVEL for more than 15 years, finding alignment between the Company’s pioneering approach to medicine and the boundary-pushing work of the Parker Institute for Cancer Immunotherapy which works to revolutionise healthcare. Over a family-style dinner by chef Evan Funke of Los Angeles hotspot Felix Trattoria, and a performance by Grammy-winning Attacca Quartet, the hosts shared how CLINUVEL’s treatments herald a new era for the treatment of light-related conditions. The Group’s CEO Dr. Philippe Wolgen also unveiled what is next for the most exciting biopharmaceutical firm in the field of photomedicine today. The future of photomedicine For 25 years, CLINUVEL introduced a proof of concept by treating groups of patients whose needs had been overlooked by the pharmaceutical industry. These include the genetic disorders erythropoietic protoporphyria (EPP), xeroderma pigmentosum (XP) and the skin disorder vitiligo, when people lose their pigmentation. In 2014 CLINUVEL developed the only approved treatment in the world for EPP, accelerating CLINUVEL’s evolution into a profitable, self-sustaining and dynamic company. Now the Company is exploring the vast potential of its technology: how can CLINUVEL’s unique knowledge of melanocortins be scaled up to treat widespread health conditions that affect the skin, brain and nervous system? Beyond the world of pharmaceuticals, CLINUVEL’s technology has the power to disrupt the skincare market. After 43 years of photomedicine research, the Company is launching PhotoCosmetics, a range of advanced skincare products based on a group of peptides known as melanocortins. Revolutionising healthcare for those in needPhilanthropy is central to the work of Lady Gaga and Michael Polansky, Sean Parker and Alexandra Parker. CLINUVEL shares their passion for creating long-lasting change and radically improving patients’ lives. At the event in Malibu, CLINUVEL announced the Photomedicine Foundation, which will count Mr Polansky as a trustee. The initiative will help those most affected by debilitating conditions related to the sun. Through the Foundation, CLINUVEL is taking its world-leading expertise directly to communities who cannot afford care or treatment. The foundation’s mission is to support vulnerable patients at extreme risk of photodamage and skin cancer in Africa, Middle East and Latin America. Michael Polansky said: “It’s critical that investors strive to find companies whose missions extend beyond profitability, by supporting the development of technologies that can drive meaningful and sustained improvement to human life. CLINUVEL is an example of a company that aims to improve human health and I am delighted to become a trustee of the Photomedicine Foundation, which will play a vital role in improving the lives of individuals with light-related conditions who cannot access life-saving medical care.” Sean Parker said: “CLINUVEL is one of the most innovative Life Sciences companies that exists today with a management that has the patience to see through long development. Both its groundbreaking melanocortin technology and long-term business plan will disrupt the world of biopharmaceuticals and luxury skincare. The Photomedicine Foundation is a welcome addition to a company on a path to growth unlike any other.” CLINUVEL’s CEO, Dr. Philippe Wolgen said: “Sean Parker and Michael Polansky are unafraid to rip up the rulebook in their mission to advance science and improve global health. CLINUVEL is grateful to have their support as we translate our pharmaceutical technology into PhotoCosmetics and give back to underserved communities. A bright future looms for CLINUVEL.” About The CLINUVEL Group: CLINUVEL is a public biopharmaceutical company whose pioneering melanocortin technology has revolutionised the field of photomedicine. Over the last 25 years, CLINUVEL’s work has furthered scientific knowledge, delivered breakthroughs in medical technology and transformed patients’ lives. The Company has developed and commercialised SCENESSE® (afamelanotide), the only approved treatment in the world for the prevention of phototoxicity in adult patients with EPP. The treatment was approved for use by the US FDA in 2019 and is also approved for commercial distribution in Europe, Israel and Australia. CLINUVEL is conducting clinical trials to assess the efficacy and safety of its melanocortin technology in treating severe disorders including xeroderma pigmentosum (XP), vitiligo and arterial ischemic stroke. In 2023 it announced its expansion into skincare with the introduction of its first PhotoCosmetic product CYACÊLLE, a polychromatic solar screen. For more information visit www.clinuvel.comInstagram:@clinuvel_pharmaceuticals @clinuvelDNATwitter:@ClinuvelNewsFacebook:@Clinuvel

The company focuses on discovering and developing small-molecule, targeted therapeutics for the treatment of fibrotic disease and cancer. RXC008 is a wholly owned gastro-intestinal (GI) targeted Rho-associated coiled-coil containing protein kinase (ROCK) inhibitor, being developed as a potential first-in-class treatment for patients with fibrostenotic Crohn’s disease. The primary objective of this first-in-human study is to evaluate the safety and pharmacokinetic (PK) profile of the drug and it is expected that results from the healthy volunteer cohorts will be available by the end of 2024. Lisa Anson, CEO, of Redx Pharma said: “We are delighted to confirm that the first participant has been dosed in the RXC008 Phase 1 clinical study. RXC008 is a potential first-in-class treatment for patients with fibrostenotic Crohn’s disease, a debilitating condition where successive surgeries are the only treatment option available today. “This milestone represents the sixth asset from Redx to enter clinical development, continuing our strong track record in small molecule drug discovery as a result of our world-class medicinal chemistry and translational science expertise.” Significant unmet clinical need Fibrostenotic Crohn’s disease is a chronic condition that causes inflammation and fibrotic stricture formation in the GI tract. More than 50% of patients diagnosed with Crohn’s disease will develop fibrostenosis within 10 years of diagnosis. There are currently no drugs specifically approved for the underlying fibrosis, which can progress despite intervention with anti-inflammatory therapies.  The only current treatment options are invasive surgical procedures to remove the affected part of the GI tract with the majority of patients requiring many successive surgical interventions. googletag.cmd.push(function () { googletag.display('text-ad1'); }); Dr Helen Timmis, interim chief medical officer, of Redx Pharma added: “Fibrostenotic Crohn’s disease patients face a significant unmet clinical need, and I am pleased that we have successfully progressed RXC008 into the clinic. “The strength of our preclinical package makes us hopeful that RXC008 can be a potential first-in-class therapeutic treatment option for fibrostenotic Crohn’s patients, and we look forward to reporting the phase 1 healthy volunteer data later this year.” Single and multi-ascending dose The phase 1 clinical study consists of two parts. The first in healthy volunteers includes both single ascending dose (SAD) and multiple ascending dose (MAD) cohorts, the latter being dosed for 14 days. The primary endpoint for the healthy volunteer cohorts will be safety, with secondary endpoints being related to RXC008’s PK profile. The second part of the study will investigate patients with fibrostenotic Crohn’s disease who will be dosed for a one-month duration with a placebo control, to show safety along with PK profile, target engagement, and changes in circulating biomarkers. Data from the healthy volunteer cohorts are expected to be available by the end of 2024. RXC008 is a potent, oral, small molecule non-systemic ROCK 1/2 inhibitor that avoids the significant cardiovascular side effects of pan-ROCK inhibitors, including tachycardia and hypotension, by being restricted to the GI-tract via high efflux and low permeability. This results in virtually no systemic breakthrough, with the molecule being rapidly metabolised by paraoxonase enzymes in the plasma, should any breakthrough occur under particular circumstances. RXC008 has a strong preclinical package across multiple therapeutic models, data from which was presented at the 2022 Inflammatory Bowel Disease (IBD) Nordic Conference, including results from a therapeutic 12-week DSS model with a closely related GI-targeted ROCK inhibitor, REDX08087.  In this model, Redx was able to show a complete reversal of preformed GI fibrosis as measured by trichome collagen staining, fully reversing fibrosis back to baseline levels. This level of anti-fibrotic effect is the strongest seen in any of Redx’s fibrosis models and modes of action to date. RXC008 is being developed to be used in conjunction with anti-inflammatories and other symptomatic treatments for Crohn’s to address the underlying fibrosis of the disease.