CREST Syndrome

A quiet biotech is reporting some positive data in a rare fibrotic disease. Certa Therapeutics — based in Melbourne, Australia — reported Monday that a Phase II trial showed “significant improvements across multiple efficacy measures” in patients with scleroderma, such as a systemic sclerosis index and skin scores. The main goal of the study, per Certa, was to examine the pharmacokinetics, pharmacodynamic effects and safety of FT011 in patients with scleroderma, a rare autoimmune disease that can cause inflammation in the skin and other parts of the body, like the lungs. Also known as crest syndrome, it has been associated with chronic hardening and tightness of the skin. But Certa also tracked a number of efficacy measures to see if the drug works. The trial recruited 30 adults that were randomly assigned to either a placebo group or one of two dose groups: 200mg or 400mg orally daily for 12 weeks. After those 12 weeks, Certa saw “clinically meaningful improvement in 60 percent of patients” treated with the 400mg dose of the therapy in a Phase II study, adding that it saw a p-value of p=0.019 versus placebo. Investigators also looked at a metric called a CRISS score, which predicts a patient’s chances from 0 to 1 of improvement based on five variables, including skin thickness and lung function. Three patients who were on FT011 achieved the maximum CRISS score of 1, Certa added. The biotech is running an open-label extension study for an additional nine months after the initial 12-week study. CEO Darren Kelly said in a statement that the readout “paves the way for a confirmatory global Phase III study.” A query to the Australian biotech was not immediately returned to Endpoints News . FT011 was originally developed by a company called Fibrotech, which had taken the drug through a Phase Ia study in diabetic neuropathy. Shire then acquired Fibrotech in 2014 when it bought the company for $75 million plus up to $600 million milestones — before Takeda bought out Shire in 2019. Certa, led by several former Fibrotech execs, including ex-CEO Kelly, then picked up the drug and was able to start a Phase II trial in 2021. Certa is not the only biotech that has been working on the rare disease. Cell and gene therapy biotech Castle Creek Biosciences has been working on FCX-013, a gene therapy for moderate and severe forms of scleroderma. Boehringer Ingelheim’s Ofev is also approved for interstitial lung disease associated with systemic sclerosis or scleroderma.

SAN DIEGO, CA, Jan. 11, 2023 (GLOBE NEWSWIRE) -- Emerald Health Pharmaceuticals Inc. (“EHP” or the “Company”), a clinical-stage biopharmaceutical company developing a new class of medicines to treat neurodegenerative, autoimmune and other diseases, has received a positive safety review following a pre-specified interim analysis by the Safety Review Committee (“SRC”) of its Phase 2a study of EHP-101 for systemic sclerosis (“SSc”). The SRC has recommended that the trial continue without modification. EHP expects to report interim results from the study in early 2023. The SRC is comprised of two physicians, one independent and the other the principal investigator in the trial. The SRC has the ability to recommend that the trial continues at the same dose or at a lower dose, that it escalates to a higher dose, or that the study be terminated altogether due to safety concerns. The SRC evaluated the blinded safety data from all patients enrolled in cohorts 1 and 2 at least up to one month post first dose, at 25 mg daily and 25 mg twice daily (50mg), respectively. They determined that EHP-101 was well-tolerated, with no drug-related serious adverse events and only mild related adverse events reported. The pharmacokinetic analysis of plasma samples over the course of the 3-month treatment did not show apparent drug accumulation in patients’ blood. “We are pleased that the safety analysis of the first two cohorts of this Phase 2a study of EHP-101 in systemic sclerosis has confirmed a positive safety pro the drug to date, consistent with the safety pro observed in our large Phase 1 study,” said Alain Rolland, Pharm.D., Ph.D., COO of EHP. “EHP-101’s mechanism of action offers the prospect of important therapeutic effects for patients. We look forward to reporting the full interim analysis of this study in early 2023. We are working to secure the funds necessary to continue the next phase of this Phase 2a study.” The Company’s lead product candidate, EHP-101, is an oral formulation of a patented new molecule. This SSc Phase 2a trial is a double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of EHP-101 in patients with SSc. SSc is a disease characterized by rapid fibrosis of the skin and internal organs. EHP has completed the first two cohorts, each with nine SSc patients, administering two doses either once daily or twice daily. The treatment lasted for 12 weeks, with a 4-week follow-up. Further details about the study design can be found on ClinicalTrials.gov: NCT04166552. EHP has obtained clearance from the FDA to amend the study protocol to increase the size of the higher-dose cohorts 3 and 4 from a total of 18 to a total of 48 subjects and increase the treatment duration from 12 weeks to 24 weeks. The Company applied for this amendment to create a pathway to possibly advance directly into a Phase 3 clinical trial of EHP-101 in patients suffering from SSc once the Phase 2a trial is completed, rather than first conducting a Phase 2b before continuing to a Phase 3 trial. About EHP-101 EHP-101 is an oral formulation of VCE-004.8, a first-in-class activator of the subunit B55alpha of the protein phosphatase PP2A that is implicated in the hypoxia inducible factor pathway. It also provides dual peroxisome proliferator-activated receptor gamma modulation and cannabinoid receptor type 2 agonist activity. Those receptors are important therapeutic targets for SSc, a disorder with various etiologies. EHP has received Orphan Drug Designation for EHP-101 in SSc in both the US and EU and Fast Track Designation for systemic sclerosis in the US. About Systemic Sclerosis SSc, a severe form of scleroderma, is a rare and chronic autoimmune disease, causing fibrosis of the skin and internal organs, including small blood vessel damage in the skin and multiple other organs in the body such as lung, heart, kidneys, musculoskeletal system and the gastrointestinal tract. The tissues of these organs become hard and fibrous, causing them to function less efficiently. While the symptoms of SSc vary for each person, it can be life-threatening, depending on which parts of the body are affected and the extent of the disease. SSc is subclassified into diffuse cutaneous SSc (dcSSc) or limited cutaneous SSc (lcSSc) based on the extent of skin involvement. Patients with dcSSc have more areas involved, and measurements of the effects of treatment have been validated by international clinical trial experts for this subset of SSc patients. The disease is more common in adults, with approximately 80,000-100,000 people affected in the US. There are no approved treatments specific to SSc. Current therapies for this disease include mainly drugs that suppress the immune system, are limited in efficacy, and sometimes present toxicities. New treatments will be critical to help reduce the symptoms of SSc and prevent further damage to the body. About Emerald Health Pharmaceuticals Inc. EHP is developing novel product candidates for the treatment of CNS, autoimmune, and other diseases with unmet medical needs. The Company has two families of patented new chemical entities that it has created through rational drug design to affect validated receptors and pathways in the body which are pertinent to targeted diseases. Its first drug product candidate, EHP-101, is in a Phase 2a study for the treatment of SSc, a severe form of scleroderma, and has met regulatory requirements to start a Phase II study for multiple sclerosis. Its second product candidate, EHP-102, is in preclinical development and is focused on treating Parkinson’s disease and Huntington’s disease. EHP-101 has received Orphan Drug Designation in the US and EU, as well as Fast-Track status by the US FDA for systemic sclerosis. EHP-102 has received Orphan Drug Designation in the US and EU for Huntington’s disease. For more information, visit or contact info@emeraldpharma.com. Forward Looking Statements To the extent statements contained in this news release are not descriptions of historical facts regarding Emerald Health Pharmaceuticals Inc. they should be considered "forward-looking statements," as described in the private securities litigation reform act of 1995, that reflect management's current beliefs and expectations. You can identify forward-looking statements by words such as "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "hope," "hypothesis," "intend," "may," "plan," "potential," "predict," "project," "should," "strategy," "will," "would," or the negative of those terms, and similar expressions that convey uncertainty of future events or outcomes. Forward-looking statements contained in this news release include, but are not limited to, statements regarding: (i) the success and timing of our product development activities and clinical trials; (ii) our ability to develop our product candidates; (iii) our plans to research, discover, evaluate and develop additional potential product, technology and business candidates and opportunities; (iv) the anticipated timing of clinical data availability; (v) our ability to meet our milestones; and (vi) our expectations regarding our ability to obtain and maintain intellectual property protection. Forward-looking statements are subject to known and unknown factors, risks and uncertainties that may cause actual results to differ materially from those expressed or implied by such forward-looking statements. Undue reliance should not be placed on forward-looking statements. We undertake no obligation to update any forward-looking statements. Emerald Health Pharmaceuticals' investigational drug products have not been approved or cleared by the FDA