On April 27, 2021 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") reported that it booked an impairment loss in the fourth quarter of the fiscal year ended March 31, 2021 (April 1, 2020 to March 31, 2021) and that there are differences between the financial forecasts (Full basis), which were reported on October 30, 2020, and the actual results for the fiscal year 2020 (Press release, Astellas, APR 27, 2021, View Source [SID1234578522]).
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(1) Booking and details of impairment loss
Astellas booked an impairment loss of ¥58.8 billion as other expenses in the fourth quarter of fiscal year 2020 not included in the financial forecasts (Full basis) announced on October 30, 2020.
In December 2020, Astellas was notified that the U.S. Food and Drug Administration (FDA) lifted the clinical hold for the ASPIRO clinical trial evaluating investigational gene therapy AT132 in patients with X-linked myotubular myopathy (XLMTM). Astellas then reassessed the development plan and recognized a delay in approval timing in the U.S. and Europe. Astellas also revised the likely approved population from our initial assessment. As a result of these updates, Astellas booked an impairment loss.
Astellas is deeply committed to the continued safe development of AT132 for the families and patients living with XLMTM, a disease with no existing treatments. There is no change to our plan to continue development. We will conduct future discussions with regulators on the path forward toward registration filings for AT132.
(2) The Differences Between Financial Forecasts (announced on October 30, 2020) and Actual results for the Year Ended March 31, 2021 (IFRS basis)
Operating profit and other line items (Full basis) were lower than the forecasts due to the such matters as impairment loss of intangible assets as discussed above.
About Astellas Gene therapies Astellas integrated its wholly owned subsidiary, Audentes Therapeutics, as of April 1, 2021 and establish "Astellas Gene Therapies" within the organization as Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. Based on an innovative scientific approach and industry leading internal manufacturing capability and expertise, we are currently exploring three gene therapy modalities: gene replacement, exon skipping gene therapy, and vectorized RNA knockdown and will also advance additional Astellas gene therapy programs toward clinical investigation. We are based in San Francisco, with manufacturing and laboratory facilities in South San Francisco and Sanford, North Carolina.
About X-linked Myotubular Myopathy
XLMTM is a serious, life-threatening, rare neuromuscular disease that is characterized by extreme muscle weakness, respiratory failure and early death. Mortality rates are estimated to be 50 percent in the first 18 months of life. For those patients who survive past infancy, there is an estimated additional 25 percent mortality by the age of 10. XLMTM is caused by mutations in the MTM1 gene that lead to a lack or dysfunction of myotubularin, a protein that is needed for normal development, maturation and function of skeletal muscle cells. The disease affects approximately 1 in 40,000 to 50,000 newborn males.
XLMTM places a substantial burden of care on patients, families and the healthcare system, including high rates of healthcare utilization, hospitalization and surgical intervention. More than 80 percent of XLMTM patients require ventilator support, and the majority of patients require a gastrostomy tube for nutritional support. In most patients, normal developmental motor milestones are delayed or never 3 achieved. Currently, only supportive treatment options, such as ventilator use or a feeding tube, are available. About AT132 for the treatment of X-linked Myotubular Myopathy AT132 is an AAV8 vector containing a functional copy of the MTM1 gene, for the treatment of XLMTM. AT132 may provide patients with significantly improved outcomes based on the ability of AAV8 to target skeletal muscle and increase myotubularin expression in targeted tissues following a single intravenous administration. AT132 has been granted Regenerative Medicine and Advanced Therapy (RMAT), Rare Pediatric Disease, Fast Track, and Orphan Drug designations by the U.S. Food and Drug Administration (FDA), and Priority Medicines (PRIME) and Orphan Drug designations by the European Medicines Agency (EMA).
About the ASPIRO Study
ASPIRO Study (NCT03199469) is multinational, randomized, open-label ascending dose trial to evaluate the safety and preliminary efficacy of AT132 in XLMTM patients less than five years of age. Primary endpoints include safety (adverse events and certain laboratory measures) and efficacy (assessments of neuromuscular and respiratory function). Secondary endpoints include the burden of disease and healthrelated quality-of-life, and muscle tissue histology and biomarkers.