NextCure (NASDAQ:NXTC) initiated with Buy rating and $72 (41% upside) price target at Needham.
X4 Pharmaceuticals (NASDAQ:XFOR) initiated with Buy rating and $25 (146% upside) price target at B. Riley FBR.
Thursday, December 5, 2019
Dynavax up 4% premarket on Albertsons’ deal in hepatitis B
Dynavax Technologies (NASDAQ:DVAX) and Albertsons Companies have partnered to provide HEPLISAV-B at latter’s more than 1,700 pharmacies nationwide, with a special focus on people living with diabetes.
Through this partnership, patients will have access to HEPLISAV-B, the only FDA-approved two-dose hepatitis B vaccine for adults that is completed in one month.
HEPLISAV-B is indicated for prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older.
Shares are up 4% premarket.
EC OKs Astellas’ XOSPATA for Relapsed or Refractory Acute Myeloid Leukemia
For twenty-five years, Invivoscribe has improved the quality of healthcare worldwide by providing high quality, standardized reagents, tests, and bioinformatics tools to advance the field of precision medicine. The LeukoStrat® CDx FLT3 Mutation Assay may now be used as an aid in the assessment of AML patients for treatment with XOSPATA® (gilteritinib) in Europe. FLT3 mutation must be confirmed with a validated test, such as the LeukoStrat CDx FLT3 Mutation Assay, which served as the companion diagnostic in the Phase 3 ADMIRAL trial resulting in approval of XOSPATA®.
Companion diagnostics play a key role in the development and approval of targeted drug therapies. The ability to screen for biomarkers in a patient population creates patient subsets which further enables drug developers in the design of novel therapeutics and management of clinical trials. Accordingly, the successful approval of a targeted therapy is highly dependent on the performance of the companion diagnostic.
EC approval of gilteritinib is based on results from the Phase 3 ADMIRAL trial, which investigated gilteritinib versus salvage chemotherapy in patients with relapsed or refractory FLT3mut+ AML. The ADMIRAL study demonstrated that gilteritinib resulted in a statistically significant improvement in median overall survival (9.3 months) compared to salvage chemotherapy (5.6 months) when patients were selected with the LeukoStrat CDx FLT3 Mutation Assay (Hazard FLT3 Ratio = 0.637 (95%CI 0.488, 0.830, P=0.0004). This approval highlights yet another solution in patient care.
ViiV submits New Drug Application for inhibitor for the treatment of HIV
ViiV Healthcare, the global specialist HIV company majority owned by GSK , with Pfizer, Inc. and Shionogi, Inc as shareholders, today completed submission of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) seeking approval of fostemsavir, an investigational, first-in-class attachment inhibitor for the treatment of HIV-1 infection. Fostemsavir is being developed for use in combination with other antiretroviral agents in heavily treatment-experienced adults with multidrug-resistant HIV-1 infection who are unable to form a suppressive regimen due to resistance, intolerance or safety considerations.
Antiretroviral medicines that can effectively suppress HIV have been instrumental in decreasing disease progression, HIV transmission, and AIDS-related deaths, but because of HIV’s ability to constantly change, some individuals can develop viral resistance to antiretroviral medicines, causing their treatment regimens to fail. Challenges with tolerability, safety, and drug-to-drug interactions may further decrease the number of acceptable antiretroviral therapies available to design effective treatment regimens. There remains an unmet need for these individuals who are considered heavily treatment-experienced and who are unable to successfully suppress their HIV.
Deborah Waterhouse, CEO of ViiV Healthcare, said: “Fostemsavir may provide an important treatment option for the group of people living with HIV who, for a variety of reasons, are not able to suppress their virus with other medicines and could be left with few or no treatments available to them. In keeping with our mission of leaving no person with HIV behind, we have overcome many barriers to bring this important new medicine to people living with HIV, including investing in what is a very complex manufacturing process. We look forward to working with the FDA to make fostemsavir available to the people in the US who need it.”
This submission is supported by the data from the pivotal phase III BRIGHTE study in heavily treatment-experienced people living with multidrug-resistant HIV. The 96-week results from the BRIGHTE study were most recently presented in July at the 10th International AIDS Society Conference on HIV Science (IAS 2019) in Mexico City.
Regeneron: Positive Topline Phase 2 Data in Rare Blood Disorder Antibody Study
Results from initial 6-patient cohort show pozelimab reduced lactate dehydrogenase (LDH) to normal levels at week 8 in patients with paroxysmal nocturnal hemoglobinuria (PNH), utilizing a weekly subcutaneous dosing regimen
Second part of Phase 2 trial initiated; plans for Phase 3 program underway
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced topline data from the pozelimab (REGN3918) Phase 2 clinical program in paroxysmal nocturnal hemoglobinuria (PNH), validating the weekly 800 mg subcutaneous (SC) dosing regimen, following an initial intravenous (IV) loading dose. Pozelimab reduced the abnormal destruction of red blood cells, otherwise known as “hemolysis,” with patients in the initial cohort achieving normal levels of a blood biomarker of elevated hemolysis called lactate dehydrogenase (LDH).
PNH is an ultra-rare, chronic, life-threatening disease where genetic mutations cause hemolysis, resulting in a range of symptoms including fatigue, shortness of breath and blood clots. Even with existing therapies, which require regular intravenous infusions administered at infusion centers or during a home visit by a health professional, approximately 50% of newly-treated patients do not achieve normal LDH levels.
“In our view, any new medicine for PNH must deliver real change for patients, such as more patients achieving normal LDH levels, or a reduced treatment burden that potentially allows for at-home self-administration,” said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer at Regeneron. “We are encouraged by these early pozelimab results, with patients achieving normal levels of LDH by week 8 using the subcutaneous dosing regimen. We look forward to speaking with regulators about the next phase of our program for these patients.”
Cytokinetics New Results Presented at International Symposium on ALS
Cytokinetics, Inc. (Nasdaq: CYTK) today announced that new results were presented at the 30th International Symposium on ALS/MND in Perth, Australia, including additional analyses from FORTITUDE-ALS (Functional Outcomes in a Randomized Trial of Investigational Treatment with CK-2127107 to Understand Decline in Endpoints – in ALS), the Phase 2 clinical trial of reldesemtiv in patients with amyotrophic lateral sclerosis (ALS). Among the post-hoc analyses presented were results of subgroup analyses describing the effect of reldesemtiv with and without use of Radicava® (edaravone) and/or Rilutek® (riluzole) and the interaction between quality of life and depression in the placebo group. In collaboration with Astellas, Cytokinetics is developing reldesemtiv, a fast skeletal muscle troponin activator (FSTA), as a potential treatment for people with SMA and certain other debilitating diseases and conditions associated with skeletal muscle weakness and/or fatigue.
Effect of Reldesemtiv: Similar Whether or Not Patients Received Edaravone and/or Riluzole
The results of FORTITUDE-ALS, presented earlier this year at the American Academy of Neurology Annual Meeting, showed that the trial did not achieve statistical significance for a pre-specified dose-response relationship in the primary endpoint of change from baseline in slow vital capacity (SVC) after 12 weeks of dosing (p=0.11). However, patients on all dose groups of reldesemtiv declined numerically less than patients on placebo for SVC and ALS Functional Rating Scale-Revised (ALSFRS-R), with larger differences emerging over time. In this post-hoc subgroup analysis of all dose groups combined and compared to placebo, reldesemtiv demonstrated a similar effect on SVC, ALSFRS-R and muscle strength by hand held dynamometry (HHD) at 12 weeks whether or not patients were being treated with edaravone and/or riluzole. The majority of patients received riluzole alone (56.5%), 4.2% were receiving edaravone alone, and 20.6% were receiving both.
For use and non-use of edaravone, the treatment difference for reldesemtiv relative to placebo for ALSFRS-R was 1.25 points (p=0.06) and 0.77 points (p=0.06), respectively. Decline in SVC was 3.07 percentage points less on reldesemtiv versus placebo in patients using edaravone (p=0.14), and 1.21 percentage points less on reldesemtiv versus placebo in patients not using edaravone (p=0.32). HHD declined 6.94 percentage points less on reldesemtiv versus placebo for patients taking edaravone (p=0.14), and 1.31 percentage points on reldesemtiv versus placebo for patients not taking it (p=0.57).
For use and non-use of riluzole, the treatment difference for reldesemtiv compared to placebo for ALSFRS-R was 0.86 (p=0.03) and 0.84 (p=0.28) points, respectively. Decline in SVC was 1.64 percentage points less on reldesemtiv versus placebo (p=0.16) and 1.81 percentage points less on reldesemtiv versus placebo (p=0.46), respectively. Decline in HHD was 2.22 (p=0.36) and 4.36 (p=0.27) less on reldesemtiv versus placebo, respectively.
“The results from these subgroup analyses add to the growing body of evidence regarding the effects of reldesemtiv in patients with ALS,” said Jeremy Shefner, M.D., Ph.D., Lead Investigator of FORTITUDE-ALS, Professor and Chair of Neurology at Barrow Neurological Institute, and Professor and Executive Chair of Neurology at the University of Arizona, Phoenix. “As the treatment landscape evolves in ALS, these data demonstrate how we may be able to further slow the decline of disease progression when adding new mechanism therapies like reldesemtiv on top of existing treatment regimens.”
Bayer, CHOP to research novel small molecule oral treatment of hemophilia
Bayer announced today that it has entered into a three-year collaboration agreement with Children’s Hospital of Philadelphia (CHOP) for the discovery and development of small molecules (SMOLs) to develop a first-in-class oral non-replacement therapy (NRT) for the treatment of hemophilia A and B. The partnership will combine CHOP’s expertise in hemophilia and coagulation and Bayer’s research capabilities.
Hemophilia is a genetic bleeding disorder in which one of the clotting proteins needed to form blood clots in the body is missing or defective. Worldwide, it is estimated that more than 400,000 people live with hemophilia and approximately seventy five percent of them receive inadequate treatment. The main treatment for hemophilia is called replacement therapy and is often administered multiple times a week to help replace the clotting factor that’s missing or low. An orally available SMOL for the treatment of hemophilia would be a completely new modality in the market, and has the potential to remove the burden of frequent injections from patients.
“Bayer is committed to investing and researching the next-generation of groundbreaking therapies. Small molecule therapies could help thousands of people with hemophilia A and B and we are looking forward to combining our strength in hemophilia research with Children’s Hospital of Philadelphia, which is a leading institution in basic and clinical research in the field of hemophilia” said Dr. Joerg Moeller, Member of the Executive Committee of Bayer AG’s Pharmaceuticals Division and Head of Research and Development. “This innovative approach is unprecedented in pharmaceutical history and would leverage significant opportunities for continued innovation in hemophilia”.
Under the terms of the agreement, Bayer is investing USD 5 million in the joint research over three years, with the option of continuing the collaboration with the agreement of both parties. Bayer will have an option to exclusively license the collaboration results.
Bayer has a strong background in hemophilia products with Jivi at the forefront of the hemophilia replacement therapies market. The new research alliance with CHOP, a world-renowned pediatric research center, has the potential to change the treatment paradigm for patients. In addition, Bayer is currently developing a gene therapy treatment for hemophilia A patients, which could radically reduce the frequency of treatment these patients have to undergo.
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