Ascendis, Can-Fite and BridgeBio Start the Week with Clinical Wins

Medical Development Laboratory

This week is starting off with some positive clinical news from Ascendis Pharma, Can-Fite and BridgeBio. Read on for the latest updates on their clinical trials. 

Ascendis's Hypoparathyroidism Drug Meets All Endpoints 

Ascendis Pharma announced that its drug TransCon PTH met all primary and all key secondary endpoints in the Phase III PaTHway Trial. TransCon PTH is an investigational once-daily long-acting prodrug that works by restoring parathyroid hormone (PTH) levels in adults with hypoparathyroidism. Restoring PTH levels leads to an alleviation in the short-term symptoms of the disease, such as weakness and severe muscle cramps, and long-term complications, such as impaired kidney functioning. 

Results from the double-blind, placebo-controlled trial of 82 adults showed a 78% response rate, achieving serum calcium levels in the normal range and independence from conventional therapeutics for the disease. Patients also achieved a statistically significant decrease in disease-specific symptoms and cognitive symptoms as compared to the control group. TransCon PTH also showed a significant reduction in disease impact, and was well-tolerated. 

The results are exciting for patients with hypoparathyroidism who currently do not have treatments that address the underlying disease. Patients currently undergo treatments that involve taking Vitamin D and calcium supplements which can contribute to the risk of impaired kidney functioning. 

Can-Fite’s Liver Fibrosis Treatment Qualifies for U.S. Patent 

Can-Fite BioPharma announced that its therapeutic Namodenoson received a Notice of Allowance from the U.S. Patent Office. Namodenoson is currently being developed for the treatment of liver cancer and non-alcoholic steatohepatitis (NASH), but Can-Fite says that the allowance granted by the Patent Office opens the door for the drug to be marketed toward all conditions with advanced liver fibrosis including autoimmune hepatitis, primary billary cirrhosis and others. 

Currently, Can-Fite is enrolling patients in a Phase IIb clinical trial of Namodenoson for the treatment of NASH and is expected to commence enrollment in a Phase III trial of the drug for the treatment of advanced liver cancer. Namodensoson is an orally bioavailable drug that binds selectively to the A3 adenosine receptor (A3AR). By binding to A3AR, Namodenoson can induce robust anti-inflammatory and anti-cancer effects. 

In December 2021, the company shared clinical data that a patient with advanced liver cancer was cleared of all tumor lesions following treatment with Namodenoson. Similarly, the company shared that a patient treated in Phase II studies survived five years after treatment and showed normal liver functioning and complete clearance of all cancer lesions and disappearance of ascites, or fluid buildup in the abdomen.  

BridgeBio Pharma Shares Positive Phase II Data for Muscular Dystrophy Therapeutic 

BridgeBio Pharma’s therapeutic BBP-418 demonstrated positive results in Phase II studies. BBP-418 is intended for the treatment of limb-girdle muscular dystrophy type 2i (LGMD2i), a genetic condition that impairs glycosylation of α-DG, a protein associated with stabilizing muscle cells. 

BBP-418 is intended to increase glycosylation of α-DG to drive functional improvements for patients in measures of mobility. Phase II results demonstrated that those treated with the drug showed an average 43% increase in the rate of glycosylated α-DG and 70% reduction in creatine kinase, a key marker of muscle breakdown. Patients also demonstrated a 3% increase in velocity during the ten-meter walk test and the therapeutic was well-tolerated with no treatment-related serious adverse events reported. 

BBP-418 has the potential to become the first approved therapy for the treatment of patients with LGMD2i. BridgeBio plans to meet with regulatory health bodies in 2022 to discuss paths to drug approval and intends to initiate a Phase III clinical trial. 

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