The biotech sector continues to be in focus with regulatory and pipeline updates from quite a few companies. New drug approvals are also in the spotlight.
: Recap of the Week’s Most Important Stories : Regeneron Gets FDA Approval of Cholesterol Drug Regeneron Pharmaceuticals, Inc. ( REGN Quick Quote REGN - Free Report) announced that the FDA has approved Evkeeza (evinacumab-dgnb) as an adjunct to other low-density lipoprotein cholesterol (LDL-C) lowering therapies to treat adult and pediatric patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH). The FDA approval was based on results from the phase III ELIPSE HoFH trial. The trial met its primary endpoint, with Evkeeza-treated patients witnessing reduced LDL-C from baseline by 49%, on average, compared to placebo at week 24.
Earlier, Regeneron announced that the United States Court of Appeals for the Federal Circuit upheld the decision by the U.S. District Court for the District of Delaware that
Amgen's ( AMGN Quick Quote AMGN - Free Report) asserted patent claims directed toward PCSK9 (proprotein convertase subtilisin/kexin type 9) antibodies are invalid based on the lack of enablement. The ruling means that Regeneron and Sanofi have successfully invalidated all five of Amgen's asserted claims relevant to Praluent (alirocumab). : Amgen announced that the FDA has granted Priority Review to sotorasib for the treatment of patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), following at least one prior systemic therapy. The Prescription Drug User Fee Action (PDUFA) date for sotorasib is Aug 16, 2021. The NDA is being reviewed by the FDA under its Real-Time Oncology Review. Amgen has also submitted a Marketing Authorization Application (MAA) in the EU in December 2020. Amgen’s Lung Cancer Drug Gets Priority Review
Amgen currently carries a Zacks Rank #3 (Hold). You can see
. the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here : Shares of bluebird bio Plunges on Trial Suspension bluebird bio ( BLUE Quick Quote BLUE - Free Report) plunged 37.8% after it announced a temporary suspension of its phase I/II (HGB-206) and phase III (HGB-210) studies of LentiGlobin gene therapy for sickle cell disease (SCD) (bb1111). The suspension is primarily due to a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia (AML).
The company received a report last week that a patient who was treated more than five years ago in Group A of HGB-206 was diagnosed with AML. Consequently, the company is investigating the cause of this patient’s AML in order to determine if there is any relationship with the use of BB305 lentiviral vector in the manufacture of LentiGlobin gene therapy for SCD. In addition, a second SUSAR of myelodysplastic syndrome (MDS) in a patient from Group C of HGB-206 was reported last week. This case is also being investigated by the company.
Separately, bluebird has also decided to temporarily suspend marketing of Zynteglo (betibeglogene autotemcel) while the AML case is assessed even though no case of hematologic malignancy has been reported in any patient who has received treatment with Zynteglo for transfusion-dependent β-thalassemia. This is because it is also manufactured using the same BB305 lentiviral vector used in LentiGlobin gene therapy for SCD.
: Shares of clinical-stage biopharmaceutical company, Cortexyme Slides on CRL Cortexyme, Inc. ( CRTX Quick Quote CRTX - Free Report) , plunged after it received a CRL from the FDA. The CRL informs the company of a partial clinical hold placed on pipeline candidate, atuzaginstat (COR388). The CRL will impact the open-label extension (OLE) phase of the ongoing GAIN Trial. The study is a randomized, double-blind, placebo-controlled phase II/III study evaluating the efficacy, safety and tolerability of atuzaginstat, an investigational gingipain inhibitor, in patients with mild to moderate Alzheimer’s disease. The partial clinical hold was initiated following the review of hepatic adverse events in the atuzaginstat trial by the FDA. : Shares of Swiss-based, clinical-stage biopharmaceutical company, AC Immune Surges on Positive AD Data AC Immune SA ( ACIU Quick Quote ACIU - Free Report) , surged after it announced positive interim results from its ongoing phase Ib/IIa clinical trial evaluating its first-in-class anti-phospho-Tau (pTau) vaccine candidate, ACI-35.030, for the treatment of Alzheimer’s disease (AD). Vaccination with ACI-35.030 generated a potent antigen-specific antibody response against pTau in 100% of older patients with early AD, achieving antibody levels several orders of magnitude higher than pre-vaccination levels. Importantly, no clinically relevant adverse events were observed. Hence, AC Immune and partner Janssen Pharmaceuticals, Inc., expect that these interim findings from the first two dosing groups support plans to advance the further development of ACI-35.030 for the treatment of AD. Performance
The Nasdaq Biotechnology Index lost 2.74% in the last five trading sessions. Among the biotech giants, Gilead lost 3.71% during the period. Over the past six months, shares of Alexion have surged 52.5%. (See the last biotech stock roundup here:
). Biotech Stock Roundup: Q4 Results of BMY, REGN, BIIB & Other Regulatory Updates What's Next in Biotech?
Stay tuned for more pipeline and regulatory updates.
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