In an interview with PharmaShots, Wildon R. Farwell, M.D., MPH, Global Medical Head of Neuromuscular Diseases at Biogen shared his views on the P-IV RESPOND study that assesses the benefits of Spinraza in patients treated with Zolgensma.Shots:RESPOND is a two-year, open-label study to evaluate the efficacy and safety of Spinraza (nusinersen) in infants and children with spinal muscular…
In an interview with PharmaShots, Allen Burton, M.D., Medical Director at Abbott Neuromodulation shared his views on the NeuroSphere myPath App which is designed to track and report pain relief in chronic pain patients.Shot:The app will record pain relief during the device trial period with SCS or DRG therapy, simplifying reporting outcomes & connect patients to…
In an interview with PharmaShots, Daniel A. de Boer of ProQR shared his views on P-II/III pivotal trial of Sepofarsen to treat the root cause of a rare eye disease, Leber congenital amaurosis 10 (LCA10)Shots:The Illuminate P-II/III trial of sepofarsen is a double-masked, randomized, controlled, multiple-dose study to evaluate if sepofarsen is effective at improving vision and is…
In an interview with PharmaShots, Mark Gurney, CEO at Tetra Therapeutics shared his views on the results of the P-II exploratory study of BPN14770 in adult patients with Fragile X Syndrome.Shots:The P-II study is a two-way crossover study assessing BPN14770 (25mg, bid) vs PBO in 30 adult male patients aged 18-45yrs. with FXS due to >200 CGG…
In an interview with PharmaShots, Ying Huang, Ph.D., CEO and CFO of Legend Biotech shed light on the clinical data following the rolling submission of BLA for Cilta-cel. He also shared insight on Legends' collaboration with Janssen.Shots:The submission of BLA was based on results from the P-Ib/II CARTITUDE-1 study of cilta-cel, which were recently presented at the 62nd ASH MeetingThe…
In an interview with PharmaShots, Steve Bradford, Vice President, Medicines Commercialization Leader at GSK shed light on the US FDA's approval of Benlysta as the first and only approved treatment for both lupus and adult patients with active LN who are receiving standard therapy.Shots:The US FDA has approved GSK's Benlysta (belimumab) as the first and…
In an interview with PharmaShot, Dr. Sophia Randolph, Chief Medical Officer of ALX Oncology shared her views on the ALX Oncology's collaboration with Zymework for evaluating the combination with ALX148 and zanidatamab for patients with advanced HER2-expressing breast cancer and other HER2-expressing solid tumors.Shots:Zymeworks will conduct a P-Ib study to assess the safety and efficacy of the combination…
In an interview with PharmaShots, Matthew L. Sherman, M.D., Executive Vice President and Chief Medical Officer of Deciphera shed light on encouraging preliminary results from the ongoing P-I/II study of DCC-3014 in TGCT patients and also presented the data of QINLOCK at CTOS 2020Shots:Deciphera presented encouraging preliminary results from the ongoing Phase 1/2 study of DCC-3014 in tenosynovial giant cell tumor…
In an interview with PharmaShots, Bradley Galer, M.D., Chief Medical Officer at Zogenix shed light on data supporting the regulatory application and what the EU approval of Fintepla will mean in terms of reducing the high physical, emotional, and financial burden associated with the disease.Shots:Fintepla was approved by the EC in Dec'2020 as a new add-on therapy to…
In an interview with PharmaShots, Nathalie Landry, Executive Vice President, Scientific and Medical Affairs at Medicago shared her views on positive results of P-I clinical trial results for its plant-derived COVID-19 vaccine. Shots:The results of the trial demonstrated that 100% of participants developed a promising antibody response after two doses of Medicago's COVID-19 adjuvanted vaccine candidateThe vaccine…

