Investors in the biotech sector were glued last week to the highly awaited 54th annual meeting of the American Society of Clinical Oncology (ASCO), which overshadowed the usual regulatory and other updates from the space. The meeting, held in Chicago (Jun 1-Jun 5) provides companies with a platform to showcase their data to scientists, physicians, the investment community and others. While the focus was mainly on ASCO data, companies like Biogen (BIIB - Free Report) and Vertex (VRTX - Free Report) remained in the limelight on pipeline updates.
Recap of Important Stories:
Biogen/Eisai Report Encouraging Data on Elenbecestat: Biogen along with partner Eisai announced encouraging results on its Alzheimer's candidate, elenbecestat, a BACE inhibitor, from a phase II study (Study 202). The study is a multicenter, randomized, double-blind, placebo-controlled parallel-group 18-month study among patients (n=70) with mild cognitive impairment (MCI) due to Alzheimer’s disease, or mild-to-moderate dementia due to Alzheimer’s disease with confirmed amyloid pathology by PET ((positron emission tomography) screening. All the patients were randomized to four treatment arms receiving elenbecestat (5, 15, or 50 mg) or placebo daily. More than half the patients in the elenbecestat 5 mg and 15 mg arms were switched to the 50 mg arm for three months or more. The data showed elenbecestat was generally safe and well tolerated. Results demonstrated a statistically significant difference in amyloid beta(Aβ) levels in the brain measured by amyloid-PET and suggested a delay of clinical symptom decline in exploratory endpoints. Eisai and Biogen are jointly developing elenbecestat since March 2014. Both companies are currently conducting two global phase III studies (MISSION AD1/2) on early Alzheimer’s disease.
Biogen currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Vertex/ CRISPR Sickle Cell Disease Candidate Put On Hold: Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that the FDA has placed a clinical hold on the Investigational New Drug Application (IND) for CTX001 for the treatment of sickle cell disease pending the resolution of certain questions raised by the FDA. Both the companies had submitted the IND to the FDA in April to support the planned initiation of a phase I/II trial in the United States in adult patients with sickle cell disease. CTX001 is an experimental, gene-edited autologous hematopoietic stem cell therapy which is also being evaluated for patients suffering from β-thalassemia along with sickle cell disease. Despite the FDA clinical hold, both the companies plan to initiate a phase I/II trial on CTX001 in Europe in adult patients with transfusion dependent β-thalassemia. CRISPR and Vertex will equally share all research and development costs and profits worldwide for CTX001. (Read more: Vertex/CRISPR's Sickle Disease Candidate Put on Hold by FDA)
Sarepta Gets Negative CHMP Opinion For Exondys: Sarepta Therapeutics, Inc. (SRPT - Free Report) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), has adopted a negative opinion for Duchenne muscular dystrophy (DMD) candidate, Exondys (eteplirsen). A negative opinion was expected following the oral explanation trend vote. Consequently, Sarepta will request a re-examination of the opinion, which will result in the assignment of a new rapporteur and co-rapporteur. Additionally, Sarepta will request a Scientific Advisory Group (SAG) on DMD be called. This in turn will allow neuromuscular specialists to provide expert guidance and insight into, among other things, the validity of the external controls used and the importance of certain functional endpoints. The re-examination will be completed by the end of 2018.
And finally a look at some ASCO results: Celgene (CELG - Free Report) had multiple presentations on its oncology drugs Revlimid, Pomalyst along with other experimental therapies at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, IL. Results from the TRANSCEND study on experimental CD19-directed CAR T cell therapy candidate, lisocabtagene maraleucel (liso-cel; JCAR017), in patients with relapsed/refractory (r/r) aggressive B-cell non-Hodgkin lymphoma (NHL) were encouraging. (Read more: Celgene Presents Data on CAR T Therapy, Revlimid&Pomalyst).
Regeneron (REGN - Free Report) and partner Sanofi also announced data from two trials evaluating cemiplimab in advanced cutaneous squamous cell carcinoma at the ASCO. The data from the study showed that patients treated with cemiplimab experienced robust anti-tumor effects. Cemiplimab-treated patients showed a 47.5% response rate with a median observed time to response of two months as of the data cut-off date.
Gilead’s (GILD - Free Report) Kite also announced results from an ongoing phase I study at the ASCO which showed that clinical responses were observed with investigational T cell receptor (TCR) cell therapy targeting human papillomavirus type 16 (HPV-16) E7 in solid tumor cancers caused by HPV. Meanwhile, Gilead has also collaborated with Hookipa Biotech AG and obtained exclusive rights to Hookipa's TheraT and Vaxwave arenavirus vector-based immunization technologies for two major chronic infectious disease indications, hepatitis B virus (HBV) and human immunodeficiency virus (HIV). Gilead and partner Galapagos NV also announced that the phase II study, EQUATOR, on pipeline candidate, filgotinib, achieved its primary endpoint of improvement in the signs and symptoms of psoriatic arthritis at Week 16.
Amgen (AMGN - Free Report) also announced results from the phase III trial, A.R.R.O.W. at the ASCO which showed that Kyprolis administered once-weekly at 70 mg/m2 with dexamethasone achieved superior progression-free survival (PFS) and overall response rates (ORR) compared to twice-weekly Kyprolisat 27 mg/m2 and dexamethasone (twice-weekly Kd) in patients with relapsed and refractory multiple myeloma. Separately, shares of Amgen were down as the FDA approved a biosimilar of its blockbuster drug Neulasta.
The NASDAQ Biotechnology Index gained 0.60% over the last five trading sessions. Among the major biotech stocks, Gilead gained 3.50%.Over the past six months, Celgene has lost 23.69%, while Alexion has gained 9.01% (See the last biotech stock roundup here: Biotech Stock Roundup: GILD Epclusa's China Approval, BMRN Palynziq's FDA Nod)
What's Next in Biotech?
Stay tuned for regulatory updates and pipeline development news.
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