This week, the focus was on data presented at the annual conference of the American Society of Hematology (ASH) from Dec 5 to Dec 8. This apart, the regular pipeline and regulatory updates on the biotech sector were also in focus.
Recap of the Week’s Most Important Stories :
Key Highlights From ASH : Quite a few biotechs presented data on their approved and pipeline products at the ASH.
Clinical-stage biopharmaceutical company Fate Therapeutics, Inc . FATE presented an encouraging patient case study update from its phase I study of FT596, its universal, off-the-shelf, CD19-targeted chimeric antigen receptor (CAR) natural killer (NK) cell product candidate. Per the case study, a heavily pretreated patient with diffuse large B-cell lymphoma (DLBCL) achieved a partial response following administration of a single-dose treatment cycle of FT596 as a monotherapy in the first dose cohort of 30 million cells. In addition, the patient subsequently received a second single-dose treatment cycle of FT596, which led to a better response as evidenced by a further decrease in both tumor size and metabolic activity. Shares were up on the released data.
CRISPR Therapeutics AG CRSP and partner Vertex Pharmaceuticals VRTX announced new positive data on a total of 10 patients treated with the investigational CRISPR/Cas9-based gene-editing therapy CTX001. Data showed a consistent and sustained response to treatment in patients with transfusion-dependent beta thalassemia (TDT) as well as those with sickle cell disease (SCD). Treatment with CTX001 showed that all seven patients with TDT remained transfusion independent until the last follow-up while all three patients with SCD were free of vaso-occlusive crises through the last follow-up.
Vertex currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here .
Amgen’s Lung Cancer Drug Gets BTD : Amgen AMGN announced that the FDA granted a Breakthrough Therapy designation to its pipeline candidate sotorasib for the treatment locally advanced or metastatic non-small cell lung cancer (NSCLC). The candidate is an investigational KRAS inhibitor. The designation was granted NSCLC with KRAS G12C mutation as determined by an FDA-approved test following at least one prior systemic therapy. KRAS G12C is the most common KRAS mutation in NSCLC. The tag given by the regulatory agency will expedite the development and regulatory review of the same. Amgen also plans to submit a new drug application by this year-end.
BioCryst Clinches FDA Nod for HAE Drug : Shares of BioCryst Pharmaceuticals, Inc . BCRX surged after it secured an FDA approval for its oral, once-daily Orladeyo (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients, aged 12 years and above. Orladeyo is a plasma kallikrein inhibitor. It significantly reduced attacks at 24 weeks and this reduction was sustained through 48 weeks as observed in the phase III APeX-2 study. HAE patients who completed the 48-weeks regimen (150 mg) saw reductions in their HAE attack rates from a mean of 2.9 attacks per month at baseline to a mean of 1.0 attacks following 48 weeks of therapy. Additionally, patients completing the 48-week therapy (150 mg) had a mean attack rate of 0.8 attacks per month as observed in the long-term, open-label APeX-S study.
The approval bodes well for BioCryst as its oral, once-daily prophylactic option enables HAE patients to lower their burdening frequency of therapy. It is also under a regulatory review for approval in Japan and the European Union.
Curis Surges on Positive Pipeline Data : Shares of Curis CRIS jumped after it announced favorable preliminary data from its ongoing open-label, single arm phase I dose escalation study of CA-4948, a novel, small molecule IRAK4 kinase inhibitor, in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS). All evaluable patients receiving a 200 mg twice daily (BID) dose of CA-4948 in the AML/MDS study achieved marrow blast reductions with one-third of the cohort achieving a marrow complete response. No dose-limiting toxicities were noticed in the study.
Earlier, Curis announced an impressive updated data from its ongoing phase I, open-label, dose escalation study of CA-4948, an IRAK4 kinase inhibitor, for the treatment of patients with relapsed or refractory (R/R) non-Hodgkin’s lymphoma (NHL) including those patients with diffuse large B-cell lymphoma (DLBCL), Waldenström’s macroglobulinemia (WM) and oncogenic MYD88 mutations. The data demonstrated durable anti-cancer activity of the single-agent CA-4948 therapy. The candidate achieved a mean reduction of 27% in tumor burden in six of seven evaluable patients.
Enlivex Gains on Phase II Study Data : Shares of clinical-stage immunotherapy company Enlivex Therapeutics Ltd . ENLV appreciated after it reported solid interim results from an investigator-initiated phase II study, which is evaluating Allocetra in severe and critical COVID-19 patients. The data was related to eight COVID-19 patients who were treated with Allocetra in the phase II study. Six of them were in severe condition while two were in critical state. 100% patients treated through Nov 26, 2020 experienced complete recovery from their respective severe/critical conditions and were discharged from the hospital after roughly 4.7 days following Allocetra administration. The eighth treated patient in the phase II study (and the 13th treated patient overall) who got enrolled in a critical condition on Nov 27, 2020 experienced a clinical improvement after being treated with Allocetra.
Performance
Medical – Biomedical and Genetics Industry 5YR % Return
The Nasdaq Biotechnology Index lost 3.34% in the last five trading sessions. Among the biotech giants, Amgen lost 1.71% during this period. Over the past six months, shares of Amgen have rallied 2.79%. (See the last biotech stock roundup here: Biotech Stock Roundup: BIIB & SAGE Alliance, MRNA, BNTX Coronavirus Updates & More )
What’s Next in Biotech?
Stay tuned for more pipeline and regulatory updates.
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