(RTTNews) - Today's Daily Dose brings you news about FDA's briefing information related to Biogen's Alzheimer's drug candidate ahead of the panel vote, CRISPR/Vertex Pharma's early data from their two ongoing phase I/II clinical trials of CTX001 in severe hemoglobinopathies, and Cassava's additional data from its phase IIb study with Alzheimer's drug candidate Sumifilam.

Read on…

1. Biogen's Alzheimer's Drug Candidate Faces FDA Panel Tomorrow

Shares of Biogen Inc. (BIIB) jumped more than 46% on Wednesday, following briefing documents released ahead of an advisory committee meeting that will review Alzheimer's drug candidate Aducanumab on November 6.

In the documents, the FDA staff has said it agrees that the results of Study 302 are highly persuasive and that the study is capable of providing the primary contribution to a demonstration of substantial evidence of effectiveness of Aducanumab.

The Biologics License Application for Aducanumab, submitted to the FDA, included clinical data from the phase III trials - EMERGE and ENGAGE - also known as Study 302 and Study 301, respectively, as well as the phase Ib PRIME study.

Biogen licensed Aducanumab from Neurimmune in 2007. Since October 2017, Biogen and Eisai have collaborated on the development and commercialization of Aducanumab globally.

The FDA's final decision on Aducanumab is expected on March 7, 2021. If approved, Aducanumab will be the first new drug in over 15 years.

BIIB closed Wednesday's trading at $355.63, up 43.97%.

2. CRISPR/Vertex's CTX001 Shows Promise in Severe Hemoglobinopathies

CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals Inc.'s (VRTX) two ongoing phase I/II clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies have yielded promising early data.

The data reported were from 7 patients - 5 patients in the phase I/II trial of CTX001 for transfusion-dependent b-thalassemia (TDT), dubbed CLIMB THAL-111, and 2 patients in the phase I/II trial of CTX001 for sickle cell disease, and dubbed CLIMB SCD-121.

According to the companies, all patients demonstrated increases in total hemoglobin (Hb) and fetal hemoglobin (HbF) over time. The 5 patients with transfusion-dependent b-thalassemia ceased receiving packed red blood cell transfusions soon after CTX001 infusion. The first patient with transfusion-dependent b-thalassemia who received CTX001 has remained transfusion-free for over 15 months.

The two patients with sickle cell disease (SCD) have had no vaso-occlusive crisis (VOCs) since CTX001 infusion. The first SCD patient who received CTX001 has remained free of VOCs for over 1 year. The vaso-occlusive crisis is a common painful complication of sickle cell disease.

As for the safety profile of CTX001, it was generally consistent with busulfan myeloablation, a conditioning regimen for allogeneic stem cell transplantation, the company added.

CRSP closed Wednesday's trading at $95.51, up 10.85%.

3. Cassava Rallies on Addnl. Alzheimer's Trial Data

Shares of Cassava Sciences Inc. (SAVA) jumped more than 25% on Wednesday, after the company reported additional clinical data of a phase IIb study with Sumifilam, its lead drug candidate, in patients with Alzheimer's disease.

In the phase IIb clinical study, Sumifilam decreased levels of a protein called HMGB1 and improved measurements of the integrity of the blood-brain barrier (BBB).

HMGB1 is a protein that triggers neuroinflammation and loss of neurons. The BBB is a complex border of cells along blood vessels that prevent unwanted substances in blood from entering the brain and its integrity is essential to brain health. A healthy BBB system selectively allows the passage of some molecules into the brain, such as water and glucose, while blocking passage to molecules that may damage the brain.

The ability of a drug candidate to decrease HMGB1 and improve BBB integrity in patients with Alzheimer's disease has not been previously reported in the science literature, noted the company.

SAVA closed Wednesday's trading at $11.54, up 25.57%.

4. Stocks That Hit New Highs/Lows

Aptevo Therapeutics Inc. (APVO) closed Wednesday's trading at a new high of $16.98, up 51.20%.

Oncorus Inc. (ONCR) closed Wednesday's trading at a new high of $20, up 12.49%.

Rocket Pharmaceuticals Inc. (RCKT) closed Wednesday's trading at a new high of $31.50, up 7.22%.

Tabula Rasa HealthCare Inc. (TRHC) closed Wednesday's trading at a 2-year low of $31.06, down 16.26%.

Check-Cap Ltd. (CHEK) closed Wednesday's trading at a new low of $0.26, down 2.22%.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.



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