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Regeneron (REGN)/ Alnylam Report Promising Data from NASH Study

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Regeneron Pharmaceuticals (REGN - Free Report) and its partner Alnylam Pharmaceuticals (ALNY - Free Report) reported top-line data from its preliminary phase I study, which supported the advancement of ALN-HSD for treating non-alcoholic steatohepatitis (NASH).

ALN-HSD is aninvestigational RNAi therapeutic targeting HSD17B13. NASH is a progressive form of non-alcoholic fatty liver disease (NAFLD), characterized by excessive fat buildup in the liver, accompanied by inflammation and fibrosis, which may progress to cirrhosis, liver failure, cancer and death.

Regeneron entered into a collaboration agreement with Alnylam in 2018 to co-develop and commercialize RNA interference ("RNAi") therapeutics for NASH and potentially other related diseases. The development of ALN-HSD is a part of the aforementioned collaboration.

The randomized, double-blind placebo-controlled single-ascending dose (SAD) and multiple-dose (MD) phase I study. The study is evaluating the safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) effects of ALN-HSD for NASH treatment.

The primary endpoint of the study evaluating ALM-HSD is the frequency of adverse events experienced in the subjects. The secondary and exploratory endpoints include the characterization of plasma and urine PK of ALN-HSD and the evaluation of the drug PD effect.

The study evaluated ALN-HSD in two parts — as a single ascending dose of 25, 100, 200, 400 or 800 mg of ALN-HSD in healthy adult volunteers (Part A) and as multiple doses of 25, 200 or 400 mg of ALN-HSD in adult patients with NASH (Part B).

The Part A of the study is complete, after which, ALN-HSD is now being studied in adult patients with NASH (Part B).

The Part B cohort of the study evaluating 200mg and 400 mg doses of ALN-HSD has completed six months. In Part B cohorts (200 mg and 400 mg), ALN-HSD exhibited a robust target knockdown and lowered liver enzymes as well as the biopsy-derived non-alcoholic fatty liver disease (NAFLD) Activity Score (NAS) in the subjects compared to placebo. The remaining cohorts of Part B are exploring a lower dose or a later biopsy point for the candidate.

Per the company, the study was not conducted to achieve any statistical significance on the endpoints. However, ALN-HSD has exhibited an encouraging tolerability and safety profile in the subjects to date.

The most common adverse event reported in the Part A cohort of the study evaluating ALN-HSD in healthy adults was injection site reaction in five patients, which were mild in severity.

However, no severe adverse effects have been reported in either Part A (healthy volunteers) or part B (NASH patients) of the study to date.

Based on the results of the phase I study of ALN-HSD, Regeneron and Alnylam intend to initiate a phase II study in adult NASH patients by the end of 2022.

Shares of Regeneron and Alnylam have returned 11.8% and 22.9%, respectively, against the industry’s decline of 22.4% in the year-to-date period.

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As a part of the 2019 extended collaboration with Alnylam, Regeneron is also co-developing an RNAi therapeutic targeting APP, ALN-APP to treat the early onset of Alzheimer’s disease.

ALN-HSD’s target market for NASH disease holds tremendous potential and currently has no approved therapies.

Among several players working on developing a NASH candidate, the most noteworthy has to be Intercept Pharmaceuticals (ICPT - Free Report) , evaluating obeticholic acid for NASH.

The company had previously received a complete response letter (CRL) from the FDA regarding its new drug application (NDA) for obeticholic acid for treating NASH. However, Intercept has now had a pre-submission meeting with the regulatory body to resubmit its NDA by the end of 2022, based on the positive top-line data from a new interim analysis of its ongoing pivotal phase III REGENERATE study, evaluating obeticholic acid in NASH.

 

Zacks Rank and A Key Pick

Currently, Regeneron holds a Zacks Rank #4 (Sell). You can mention ALNY’s rank here as well.

A better stock in the same sector is Immunocore (IMCR - Free Report) carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here.

Immuncore’s loss per share estimates for 2022 have narrowed from $2.44 to $1.34 in the past 30 days. The same for 2023 has narrowed from $2.76 to $1.78 in the same time frame.

Earnings of Immunocore missed estimates in two of the trailing four quarters and beat the same on the remaining two occasions. The average earnings surprise for IMCR is 33.28%.
 

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