FDA Approves 1st Ebola Drug, CYCN Drops Sickle Cell Disease Drug, VRTX Plunges In After-hours Trade

Today’s Daily Dose brings you news about AnaptysBio’s generalized pustular psoriasis trial results, Cyclerion discontinuing its Sickle Cell Disease drug program, FDA approving the first drug for Ebola, analyst’s new price target of Replimune stock, and Vertex halting its phase II study of VX-814 in patients with alpha-1 antitrypsin deficiency.

Read on…

1. AnaptysBio’s Generalized Pustular Psoriasis Trial Hits Key Goal

AnaptysBio Inc.’s (ANAB) phase II clinical trial of Imsidolimab for the treatment of moderate-to-severe generalized pustular psoriasis, dubbed GALLOP, has yielded positive results.

Generalized pustular psoriasis, or GPP, is a severe and rarest form of psoriasis and there are no approved therapies to treat it. Imsidolimab was designated an orphan drug by the FDA for the treatment of GPP in July of this year.

In the GALLOP trial, 6 of 8 patients achieved the primary endpoint of improvement in the clinical global impression scale (CGI) on Day 29, with rapid reduction of skin pustules by 60% on Day 8 and 94% clearance on Day 29.

An end-of-phase II meeting, based upon data available from the 8 patients enrolled in the GALLOP trial, is expected to be held this quarter.

The company also intends to explore the potential of Imsidolimab in EGFR-mediated skin toxicity and ichthyosis and phase II trials in these indications are also expected to be initiated this quarter (Q4, 2020).

ANAB closed Wednesday’s trading at $20.37, up 14.70%.

2. Bayer’s CHRONOS-3 Study Achieves Main Goal

Bayer’s phase III study evaluating Aliqopa (copanlisib) in combination with Rituximab in patients with relapsed indolent non-Hodgkin’s Lymphoma, dubbed CHRONOS-3, has met its primary endpoint of significantly prolonging progression-free survival.

The company expects to present results from the CHRONOS-3 trial at a scientific congress. Bayer also plans to discuss the trial data with health authorities worldwide.

Indolent forms of non-Hodgkin’s lymphoma are a heterogenous group of malignancies characterized by a chronic pattern of remissions and recurrences and it includes follicular lymphoma, marginal zone lymphoma, small lymphocytic lymphoma, and lymphoplasmacytoid lymphoma/Waldenström macroglobulinemia.

Bayer’s Aliqopa was approved in the U.S. under the accelerated approval pathway for the treatment of adult patients with relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies in September 2017.

Aliqopa was granted accelerated approval based on a phase II trial, dubbed CHRONOS-1, which demonstrated an overall response rate (ORR) of 59% in adult patients with relapsed follicular lymphoma. Updated data for CHRONOS-1, published in the American Journal of Hematology 2020, also showed an ORR of 59%, noted the company.

BMWYY.OB closed Wednesday’s trading at $24.95, down 0.58%.

3. Cyclerion Throws in the Towel on Sickle Cell Disease Drug

Shares of Cyclerion Therapeutics Inc. (CYCN) plummeted on Wednesday on news of the company’s decision to drop its sickle cell disease program, following disappointing data from its STRONG-SCD study.

STRONG-SCD is a phase II study that evaluated Olinciguat for the potential treatment of sickle cell disease. According to the topline results, Olinciguat did not demonstrate the adequate activity to support further internal clinical development.

Sickle Cell Disease (SCD) is a genetic blood disorder affecting hemoglobin. In these patients, the hemoglobin protein present inside the red blood cells is abnormal, causing the RBCs to become rigid, sticky, and “C” or sickle, shaped.

Cyclerion noted that it will now focus on the development of treatments for serious diseases of the central nervous system (CNS). The company’s investigational drug IW-6463 is in clinical development for Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like episodes (MELAS), and Alzheimer’s disease with vascular pathology.

CYCN closed Wednesday’s trading at $3.35, down 52.95%.

4. Regeneron’s Ebola Drug First to Cross FDA Finish Line

The FDA has approved Regeneron Pharmaceuticals Inc.’s (REGN) Inmazeb for the treatment of Ebola infection in adult and pediatric patients, including newborns of mothers who have tested positive for the infection.

Inmazeb is a cocktail of three monoclonal antibodies of similar structure – Atoltivimab, Maftivimab, and Odesivima, and becomes the first FDA-approved treatment for Ebola. This drug is indicated for the infection caused only by Zaire ebolavirus and its efficacy is not established for other species of the Ebolavirus and Marburgvirus genera.

Gilead Sciences’ Remdesivir and Mapp Biopharmaceutical Inc.’s ZMapp are also being explored in the treatment of Ebola virus disease.

First discovered in 1976, the majority of the cases and outbreaks of Ebola have been in Africa. The 2014-2016 Ebola outbreak in West Africa is considered to be the largest Ebola outbreak ever.

There is one FDA-approved vaccine for the prevention of Ebola – Merck’s Ervebo, which was greenlighted last December.

REGN closed Wednesday’s trading at $600.82, down 1.18%.

5. Replimune Soars as Analyst Hikes Price Target

Shares of Replimune Group Inc. (REPL) surged more than 46% to touch a new high of $46.28 in intraday trading on Wednesday after an analyst at Roth Capital Tony Butler hiked his price target on the stock to $50 per share from $30 per share.

Replimune is developing a new generation of cancer treatments called oncolytic immunotherapies. The lead product candidate is RP1, which is based on a proprietary new strain of herpes simplex virus engineered to maximize tumor-killing potency.

RP1 in combination with Libtayo is under a phase II study in cutaneous squamous cell carcinoma. A phase I/II clinical trial of RP1 in combination with Opdivo in non-melanoma and melanoma skin cancers; a phase II trial of RP1 in combination with Opdivo in anti-PD-1 refractory melanoma and a phase Ib trial of RP1 as monotherapy in solid organ transplant recipients with cutaneous squamous cell carcinoma are some of the other trials that are exploring the potential of RP 1.

REPL closed Wednesday’s trading at $36.25, up 46.82%.

6. Vertex Plunges as VX-814 Study in AATD Flops

Shares of Vertex Pharmaceuticals Inc. (VRTX) were down more than 11% in after-hours Wednesday on news of the company discontinuing its phase II study of VX-814 in patients with alpha-1 antitrypsin deficiency.

Alpha-1 antitrypsin deficiency (AATD) is an inherited disease that can cause damage to the lungs and the liver.

The phase II study of approximately 50 patients was designed to evaluate the safety and pharmacokinetics of VX-814, and the ability of VX-814 to increase functional levels of alpha-1 antitrypsin over 28 days of dosing.

An analysis of the pharmacokinetic data from the study indicated that it would not be feasible to safely reach targeted exposure levels with VX-814, noted the company.

However, a phase II proof of concept study of another drug candidate VX-864 in patients with alpha-1 antitrypsin deficiency is ongoing, with data expected in the first half of 2021.

VRTX closed Wednesday’s trading at $271.46, down 1.68%. In after-hours, the stock was down 11.96% at $239.

7. Stocks That Moved On No News

Oncorus Inc. (ONCR) closed Wednesday’s trading at $18.55, up 23.58%.

Trinity Biotech plc (TRIB) closed Wednesday’s trading at $2.61, up 14.47%.

ClearPoint Neuro Inc. (CLPT) closed Wednesday’s trading at $6.30, up 13.11%.

Altimmune Inc. (ALT) closed Wednesday’s trading at $12.80, down 9.99%.

Akouos Inc. (AKUS) closed Wednesday’s trading at $17.36, down 9.30%.

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