Fortress Biotech reports positive data from CUTX-101

Fortress Biotech Inc. (FBIO) reported positive topline clinical efficacy data for CUTX-101. The trial is being conducted by its partner company Cyprium Therapeutics. The data showed the drug candidate brought about statistically significant improvement in overall survival for Menkes disease. CUTX-101 has been given Orphan Drug, Fast Track and Rare Pediatric Disease Designations by the FDA.

The primary efficacy endpoint for the trial is overall survival measured from birth. The primary efficacy analysis compared overall survival in Menkes disease patients who were administered daily dose of CUTX-101 beginning within four weeks of age to a historical control cohort of patients who were not given copper therapy. Primary efficacy analysis included 31 Menkes disease patients who received ET with CUTX-101 and 18 HC Menkes disease patients. The trial met the primary endpoint of overall survival.

The drug candidate brought about nearly 80 percent reduction in the risk of death whereas median survival for the ET cohort was 14.8 years or 177.1 months compared to 1.3 years 15.9 months for the untreated HC cohort. S. Yam, CEO of Cyprium said, “These positive topline clinical efficacy data highlight the potential of CUTX-101 as an effective therapy for Menkes disease patients. With no currently approved U.S. Food and Drug Administration (“FDA”) treatments, Menkes disease is a serious condition with a significant unmet medical need.”

The ET cohort participants carried a severe pathogenic mutation of the ATP7A gene and were born within the past 20 years. The treatment with CUTX-101 was initiated within four weeks of age, adjusted for prematurity. The participants survived at least four weeks after birth and were asymptomatic for significant neurological signs and symptoms during the first four weeks.

HC cohort participants carried a severe pathogenic mutation of the ATP7A gene and were also born within the past 20 years. These participants have not received CUTX-101 therapy and have survived at least four weeks after birth. The patients were asymptomatic for significant neurological signs and symptoms during the first four weeks.

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CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate. It has been manufactured to offer improved tolerability due to physiological pH and to circumvent the oral absorption of copper, which is diminished in patients suffering from Menkes disease. The drug candidate is being clinically developed for treating Menkes disease. It aims to treat the ailment by reloading Copper Histidinate, repairing copper homeostasis, and retaining serum copper levels in the normal age appropriate range. The enrollment for a Phase 3 trial of CUTX-101 has been completed. Currently, a Cypriot-sponsored expanded access protocol for Menkes disease patients is being carried out.

Cyprium Therapeutics is mainly engaged in developing therapies for treating Menkes disease and related copper metabolism disorders. It has been founded by Fortress Biotech Inc., which is mainly invested in acquiring, developing and commercializing high-potential marketed pharmaceutical products. It also deals in development-stage pharmaceutical product candidates. Fortress Biotech has five marketed prescription pharmaceutical products in its portfolio. It has robust development pipeline with over 25 programs spanning a wide range of market segments including oncology and gene therapy.

Lipocine suffers setback with Tlando

Lipocine Inc. (LPCN) provided regulatory update for its lead drug candidate Tlando. The company has been informed by the FDA that it required additional time to complete its review of the New Drug Application. The anticipated Prescription Drug User Food Act goal date was set at August 28, 2020. The FDA has not provided any alternate timeline or a new action date. The regulator has not demanded any additional data.

TLANDO™ is an oral testosterone replacement therapy product candidate. It comprises Testosterone Undecanoate, which helps in restoring normal testosterone levels in males for conditions associated with a deficiency or absence of endogenous testosterone. Lipocine has faced several roadblocks for Tlando. The company had received its third Complete Response Letter in November 2019. It had previously received CRLs in June 2016 and May 2018 as well.

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The company had recently reported its second quarter results. Lipocine suffered a net loss of $6.4 million or $0.13 per diluted shares for the second quarter. The company had reported $3.4 million in net loss for the corresponding quarter of the previous year. Its research and development expenses for the quarter increased from $2 million to $2.3 million on year over year basis. Its General and Administrative expenses also jumped from $1.4 million to $2 million for the second quarter.

Lipocine Inc. has a robust developmental pipeline with various candidates such as TLANDO, LPCN 1144, TLANDO XR, LPCN 1148 and LPCN 1107. LPCN 1144 is a product of bioidentical testosterone. The drug candidate recently completed a proof-of-concept clinical study showing its potential effectiveness in treating non-cirrhotic NASH. TLANDO XR is a novel oral prodrug of testosterone and LPCN 1148 is an oral prodrug of bioidentical testosterone aimed to treat cirrhosis.

Enanta presents positive data for NASH and HBV

Enanta Pharmaceuticals Inc. (ENTA) reported updates for its non-alcoholic steatohepatitis (NASH) and hepatitis B virus (HBV) development programs. The company is currently working on EDP-305 and EDP-297 for treating NASH. Its EDP-514 is being developed for treating HBV.

Phase 2a ARGON-1 study of EDP-305, its lead Farnesoid Xreceptor (FXR) agonist, showed that the drug candidate met its primary endpoint with statistically significant reduction in alanine transaminase of 28 U/L in comparison to 15 U/L in the placebo cohort at week 12. EDP-305 at the 2.5mg dose also showed statistically significant reduction in liver fat content as measured by MRI-PDFF. The drug candidate also showed robust target engagement as evidenced by decrease in C4 and the increase in FGF-19.

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EDP-297 substantially decreased fibrosis progression and enhanced liver function as measured by key biomarkers in a rat model. Rats were randomized to be administered either vehicle control (0.5% methylcellulose), 0.1 mg/kg EDP-297, or 0.3 mg/kg EDP-297 by once-daily oral gavage at the first signs of fibrosis. The data showed that the drug candidate may have a potent anti-fibrotic effect in NASH patients, including those with late-stage F3/4 fibrosis.

The data from first in human, Phase 1 study of EDP-514 showed that it was rapidly absorbed. Its exposure magnified with escalating single and multiple dosing of 600 mg and 400 mg, respectively. The drug candidate was found to be generally safe and well tolerated over a broad spectrum of single and multiple doses for up to 14 days. The trial did not report any discontinuations due to adverse events. EDP-514 demonstrated pharmacokinetics suitable for once daily oral dosing. The geometric mean plasma concentration at 24 hours was found to be significantly higher after multiple dosing with and without a standard meal. The drug candidate is currently being evaluate in two Phase 1b clinical studies.

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