Alnylam's NDA Accepted, And Other News: The Good, Bad And Ugly Of Biopharma

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Summary

Alnylam Reports FDA Acceptance of Lumasiran Application

Alnylam Pharmaceuticals, Inc. (ALNY) announced that the FDA has accepted its New Drug Application for Lumasiran. The drug candidate has also been awarded Priority Review status, which will reduce the review time from usual 10 months to six months. The FDA has set an action date of December 3, 2020, under its Prescription Drug User Fee Act. It has also indicated that it is not currently planning to hold an advisory committee meeting as part of the NDA review.

Lumasiran is an RNAi and is designed to treat primary hyperoxaluria type 1, which is an ultra-rare disease which leads to a progressive decline in kidney functioning and may also cause end-stage renal disease. The drug candidate holds Breakthrough Therapy, Pediatric Rare Disease and Orphan Drug designations for treating PH1. The company has already filed its marketing authorization application with the European Medicines Agency. The application is under review and is expected to reach the decision stage later this year.

Alnylam supported the application with positive data from the pivotal ILLUMINATE-A study. The results from the study demonstrated that the patients treated with Lumasiran met the primary endpoint related to efficacy as measured by the percent change from baseline, relative to placebo. The study tested six secondary endpoints and all of them showed statistical significance. The most prominent secondary endpoint was the proportion of patients achieving a near-normalization or normalization of urinary oxalate.

ILLUMINATE-A was a randomized, double-blind, placebo-controlled trial and enrolled nearly 30 patients aged six and above. It was the largest interventional study conducted specifically in PH1. The participants were randomized 2:1 to lumasiran or placebo. Select patients were given the drug candidate at 3 mg/kg monthly for three months followed by quarterly maintenance doses.

The primary endpoint of the study was defined to be the percent change from baseline in 24-hour urinary oxalate excretion averaged across months 3 to 6. The drug candidate met the primary endpoint at six months. Akshay Vaishnaw, M.D., Ph.D., President of R&D at Alnylam, said, “We are very pleased to report positive topline Phase 3 results for lumasiran, our third wholly owned investigational RNAi therapeutic. Patients living with PH1 and their families are faced with the burden of recurrent and painful stone events and a progressive and unpredictable decline in kidney function that ultimately results in end-stage renal disease and the need for intensive dialysis as a bridge to dual liver/kidney transplantation.” The study did not report any severe or serious adverse events.

The company is currently assessing the drug candidate in two Phase III studies for PH1 patients less than six years of age with preserved renal function and advanced PH1 in patients of all ages with advanced renal disease. The former is the subject matter for ILLUMINATE-B while latter is being studied in ILLUMINATE-C. Alnylam is also assessing various other drug candidates such as vutrisiran, inclisiran and fitusiran. Its HELIOS-A phase III study aims to assess vutrisiran for treating hATTR amyloidosis with polyneuropathy while ATLAS phase III program aims to develop fitusiran in collaboration with Sanofi (SNY).

Alnylam currently has its flagship product inclisiran under review in the United States as well as in Europe. The drug candidate aims to treat heterozygous familial hypercholesterolemia and atherosclerotic cardiovascular disease. The company has collaborated with Novartis for developing this treatment.

Argenx Reports Positive Data from Phase 3 Efgartigimod Trial

argenx (Argenx) (ARGX) announced that its Phase 3 trial of Efgartigimod met its primary endpoint. The trial aimed to evaluate Efgartigimod for treating patients suffering from Generalized Myasthenia Gravis. The company now plans to file a Biologics License Application with the FDA by the end of this year.

The Phase 3 ADAPT trial was a randomized, double-blind, placebo-controlled, multi-center, global trial. It enrolled 167 adult patients across its North America, Europe and Japan sites. All the patients had a confirmed gMG diagnosis and an MG-ADL total score of five or greater. Wim Parys, M.D., Chief Medical Officer of Argenx, said, “The data showed that efgartigimod drove fast and deep responses, including in a proportion of patients who achieved minimal or no symptoms after treatment. In addition, we saw responses that lasted beyond eight or 12 weeks, supporting our plans to offer individualized dosing schedules that are purpose-fit to the variability in disease course that gMG patients experience.”

The ADAPT data showed that 67.7% of AChR-Ab+ patients treated with efgartigimod achieved the primary endpoint compared with 29.7% on placebo. On the Quantitative Myasthenia Gravis (QMG) score, 63.1% of AChR-Ab+ patients showed response to efgartigimod compared with 14.1% on placebo. The responders were defined as having a three-point improvement on the QMG score for at least four consecutive weeks. Further, the drug candidate also showed 40 percent of the patients achieving minimal symptom expression in comparison to 11.1 percent of the placebo-treated patients hitting the mark. The goal was defined as MG-ADL scores of 0 or 1, where 0 defined symptom-free.

The secondary endpoints of the trial achieved statistically significant difference in the efgartigimod arm for AChR-Ab+ patients over placebo arm. However, the secondary endpoint of time to qualify for retreatment did not show statistical significance.

Co-Diagnostics Identifies Coronavirus in Cancer Tissue

Co-Diagnostics (CODX) reported that its COVID-19 test kit has been used for identifying the SARS-CoV-2 virus in cancer tissue of an asymptomatic coronavirus patient. The details of the test have been given in a paper published in the Journal of Clinical Pathology. The data shows that the Logix Smart COVID-19 Test Kit may be used for detecting the virus even before it starts to show symptoms.

The study was conducted at European Institute of Oncology in Milan, Italy, where researchers used the test to decide whether SARS-CoV-2 was present in the removed tumor of a man who later started showing symptoms and was diagnosed with COVID-19. The tumor was removed from the patient’s tongue and was embedded in formalin-fixed paraffin. In total, 10 FFPE samples and six bronchoalveolar samples from other non COVID-19 patients were also tested.

Logix Smart COVID-19 test is both FDA-approved and CE-marked. Co-Diagnostics uses its proprietary technology for designing specific tests for industries. Dwight Egan, Co-Diagnostics CEO, said, "Being able to detect the presence of the virus before symptoms even occur illustrates the sensitivity and value of our test, especially in new sample types. The world needs every advantage we can get in our battle against this deadly disease, and we believe our test has the attributes needed to benefit millions across the globe."

The company is currently selling its test kits in 50 countries as well as in 20 US states.

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