Biogen Reports Positive Data from Landmark NURTURE Study

Biogen Inc. (BIIB) reported new data from NURTURE which included nearly a year of additional follow-up for NURTURE study participants. It is the longest study of pre-symptomatic patients with spinal muscular atrophy. New data shows that in infants genetically diagnosed with SMA, early and sustained treatment with SPINRAZA for up to 4.8 years enabled unprecedented survival.

As of February 2020, all 25 patients treated were alive and remained free of permanent ventilation. All children who achieved the motor milestone of being able to walk independently, kept up that ability until the last visit. Kathryn Swoboda, M.D., the Katherine B. Sims, M.D., Endowed Chair in Neurogenetics and Director of the Neurogenetics Program, Massachusetts General Hospital said, The impact of early and sustained SPINRAZA treatment on these infants and their families is remarkable. Ive had the privilege to watch them grow into active young children, many of whom have experienced progress in motor function consistent with children their age who do not have SMA.

The patients enrolled in the study received their first dose of SPINRAZA before they were 6 weeks old. The new data showed that the participants who were previously able to walk independently and walk with assistance sustained their ability over the course of 11 months since the last data cut. Over the 11 months of follow-up, one child gained the ability to walk with assistance.

Patients with two copies of SMN2 were able to score and advance on the Hammersmith Functional Motor Scale Expanded scale. This development is atypical to the natural history of the disease. The drug candidate was found to be well-tolerated and did not show any new safety concerns over the extended follow-up period. further, no patient had to drop out of the study on account of adverse events concerned with the treatment.

SPINRAZA is the first approved therapy for treating infants, children and adults with spinal muscular atrophy, which is a rare, genetic, neuromuscular disease characterized by a loss of motor neurons in the spinal cord and lower brain stem. The drug candidate is an antisense oligonucleotide and is administered by intrathecal injection into the fluid surrounding the spinal cord. It has been developed using Ionis Pharmaceuticals (NASDAQ:IONS) proprietary technology, which aims to increase the amount of survival motor neuron protein.

Biogen licensed the global rights for developing, manufacturing and commercializing SPINRAZA from Ionis Pharmaceuticals, Inc. Both the companies collaborated for developing the therapy. Some of the most common adverse events associated with SPINRAZA are respiratory infection, fever, constipation and headache. Some patients also complained of renal toxicity.

SPINRAZAs main competitor in the market is Zolgensma, which has been developed by Novartis (NYSE:NVS). The therapy comes with a price tag of $750,000 for the first year and $375,000 per annum subsequently.

Biogen is mainly focused on developing therapies for neurological and neurodegenerative diseases. It has robust portfolio of medicines for a wide range of ailments such as multiple sclerosis and spinal muscular atrophy.

Magenta Therapeutics (MGTA) made a strategic decision to discontinue enrollment for its Phase 2 clinical trial for MGTA-456. The company stated that various factors including the impact of COVID 19 and the FDA feedback regarding the endpoints and design of a registrational study were behind the decision. Magenta stated that it plans to put its resources into promoting stem cell mobilization and conditioning programs.

Magenta made this announcement while providing portfolio update. The company also provided information about its other programs such as MGTA-145, which is a first line stem cell mobilization therapy. The data showed that the drug candidate, when used in conjunction with plerixafor, offers safe, same-day dosing, mobilization and collection of sufficient functional hematopoietic stem cells for transplant.

For MGTA-456, the company said that it has become aware of several challenges facing allogeneic stem cell transplant in patients with non-malignant diseases, leading to the decision to withdraw from the study. However, it expects the enrolment in the Phase 2 investigator-initiated trial in patients with blood cancers to be completed in the near future. Jason Gardner, CEO of the company said, As part of this strategic decision, we will discontinue enrollment in the Phase 2 study of MGTA-456 cell therapy in patients with inherited metabolic disorders. We thank the brave patients and their families who participated in the trial, as well as the dedicated investigators and team members at the clinical trial sites.

Magenta is a biotech firm which is mainly focused on developing novel medicines for various blood cancers, genetic diseases and autoimmune disorders. The company stock is currently trading nearly 30 percent low on Year to Date basis and is facing uncertainty on account of pandemic outbreak and several internal reasons.

Clovis Oncology (CLVS) announced that it has completed enrolment for its Phase 3 ATHENA trial. The company stated that it expects the topline data from the Rubraca monotherapy cohort to be available by the second half 2021, whereas the data from combination cohort will be out by the second half of 2022 or later.

ATHENA trial had the target of enrolling 1,000 ovarian cancer patients and these were enrolled in trial sites spread across 24 countries in North America, Europe and Asia. Patrick J. Mahaffy, CEO of Clovis Oncology, said, The completion of target patient enrollment in the Phase 3 ATHENA trial is an important milestone for Clovis and a critical step toward developing additional therapeutic options for women with advanced ovarian cancer.

Phase 3 ATHENA trial seeks to assess the combination of Rubraca and Opdivo as the first line maintenance treatment for newly diagnosed ovarian cancer patients after their initial treatment with platinum-based chemotherapy. Phase 3 trial is a multinational, randomized and double-blind study. This four-arm trial has investigator-assessed progression-free survival as its primary endpoint while its secondary endpoints include overall survival, response rate and duration of response. The company is using Bristol-Myers Squibb's (NYSE:BMY) PD-1 inhibitor OPDIVO for the combination arm.

Rubraca is an oral, small molecule inhibitor of PARP1, PARP2 and PARP3. It is currently being tested for a wide range of solid tumors and ovarian cancer. In the United States, the drug candidate is already approved as the maintenance treatment of adult patients with recurrent epithelial ovarian, primary peritoneal cancer or fallopian tube who are in a complete or partial response to platinum-based chemotherapy. It is also approved in Europe for certain indications.

Clovis Oncology is mainly focused on developing therapies for various cancers. The company has several collaborations for its development processes.

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