BLUE Hit By SCD Trial Blues, CRTX Plunges On Partial Hold, SESN To Face FDA In August

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Today's Daily Dose brings you news about temporary suspension of bluebird bio's gene therapy trials in sickle cell disease, Sesen Bio's regulatory catalyst for this year, partial hold on Cortexyme's open-label extension phase of its ongoing phase II/III study of Atuzaginstat for Alzheimer's disease, and expanded FDA approval of Novartis' Entresto.

Read on…

1. Sickle Cell Disease Trial Blues For bluebird bio

Shares of bluebird bio Inc. (BLUE) slumped to a 5-year low on Tuesday as the company's LentiGlobin gene therapy trials for sickle cell disease were temporarily suspended after two cases of cancer were reported in study participants.

The sickle cell disease studies that were temporarily suspended include the phase I/II trial of LentiGlobin, dubbed HGB-206, and a phase III study of LentiGlobin, dubbed HGB-210.

The company has come to know that a patient who was treated more than five years ago in Group A of the HGB-206 trial has been diagnosed with acute myeloid leukemia. Another patient from Group C of the HGB-206 trial has developed myelodysplastic syndrome.

In view of the two reports of Suspected Unexpected Serious Adverse Reaction (SUSAR) associated with LentiGlobin, the company has decided to temporarily suspend marketing of ZYNTEGLO in the European Union and the United Kingdom. ZYNTEGLO is a one-time gene therapy for patients 12 years and older with transfusion-dependent ß-thalassemia and it won the European Medicines Agency's approval in October 2019.

ZYNTEGLO is also manufactured using the same BB305 lentiviral vector used in LentiGlobin gene therapy for sickle cell disease.

BLUE touched a 5-year low of $26.24 in intraday trading on Tuesday, before closing at $28.44, down 37.85%.

2. Clene Gets the Go-ahead in Brazil for Covid-19 Study

Clene Inc. (CLNN) has received regulatory clearance in Brazil to commence a phase II trial of CNM-ZnAg liquid solution in acutely symptomatic, non-hospitalized COVID-19 patients.

The primary endpoint will evaluate the rate of decreased hospitalizations at day 28, with secondary endpoints assessing time to symptom resolution. The trial is expected to enroll approximately 276 patients.

The company expects to complete enrollment in the trial in mid-2021, with results now anticipated in the second half of 2021.

CLNN closed Tuesday's trading at $16.30, up 30.50%.

3. FDA Slaps Partial Hold On Cortexyme's Trial

Shares of Cortexyme Inc. (CRTX) took a hit on Tuesday, following a partial hold on the open-label extension phase of the company's ongoing phase II/III study of Atuzaginstat for Alzheimer's disease, dubbed GAIN.

The partial clinical hold was initiated by the FDA following the review of hepatic adverse events in the Aatuzaginstat trial by the FDA. These events have been reversible and without any known long-term adverse effects for the participants, the company noted.

Under the hold, no new participants will be enrolled in the open-label extension and currently enrolled open-label extension participants will be discontinued, according to the company.

Participants in the fully enrolled double-blind, placebo-controlled randomized phase of the GAIN Trial will continue to receive study drug at their assigned dose, with top-line results from the GAIN trial expected in the fourth quarter of this year.

CRTX closed Tuesday's trading at $30.72, down 35.29%.

4. Diffusion Pharma Hits New High

Shares of Diffusion Pharmaceuticals Inc. (DFFN) touched a new 52-week high on Tuesday, following topline data from its phase Ib clinical trial of its novel, diffusion-enhancing therapeutic, trans sodium crocetinate ("TSC"), in hospitalized COVID-19 patients with confirmed hypoxemia, the most common cause of tissue hypoxia.

The primary objective of the trial was to evaluate the safety and tolerability of TSC administered on a more frequent dosing regimen not previously tested in a clinical trial setting. Secondary endpoints included pharmacokinetic measurement of TSC levels after dosing, relative improvements in blood oxygen levels, and certain other clinical parameters related to COVID-19.

According to the results announced Tuesday, no dose limiting toxicities or serious adverse events were observed in the trial. The secondary endpoint data is expected to be available early in the second quarter of 2021.

A study that will measure the effects of TSC on peripheral tissue oxygen delivery using a device called a transcutaneous oximeter, or TCOM, is expected to be initiated by the end of the first quarter 2021, with completion scheduled in the second quarter of 2021. Another study that will measure the effects of TSC on the ability of the lungs to transfer gas from inspired air to the bloodstream, using carbon monoxide as a surrogate for oxygen is expected to be initiated in the second quarter of 2021, with completion planned in the third quarter of 2021.

DFFN closed Tuesday's trading at $1.70, up 37.1%.

5. FDA Approves Expanded Label for Novartis' Entresto

The FDA has approved Novartis AG's (NVS) Entresto in the expanded indication to treat adults with chronic heart failure to help reduce the risk of death and hospitalization. The expanded indication enables potential treatment of more adults with left ventricular ejection fraction below normal, the group where benefits are most clearly evident, the company noted.

It is estimated that about 6 million Americans are living with chronic heart failure. About 3 million have heart failure with reduced ejection fraction (HFrEF) , and of the remaining 3 million, about 2 million have heart failure with preserved ejection fraction (HFpEF) with left ventricular ejection fraction below normal.

Entresto, a twice-a-day prescription medicine, is already approved to reduce the risk of cardiovascular death and hospitalization for heart failure in patients with chronic heart failure and reduced ejection fraction. Entresto is usually administered in conjunction with other heart failure therapies, in place of an ACE inhibitor or other ARB. The drug is also approved for the treatment of symptomatic heart failure with systemic left ventricular systolic dysfunction in pediatric patients aged one year and older.

The drug raked in total annual sales of $2.5 billion in 2020, up 45% over 2019. The company expects peak sales of the drug to hit $4 billion to $5 billion.

NVS closed Tuesday's trading at $91.46, down 0.62%.

6. Sesen Bio To Face FDA In August

Sesen Bio's (SESN) Biologics License Application (BLA) for Vicineum for the treatment of high-risk, *BCG-unresponsive non-muscle invasive bladder cancer has been accepted for priority review by the FDA, with a decision expected on August 18, 2021.

*Bacille Calmette-Guerin (BCG) is the most common intravesical therapy for treating non-muscle-invasive urothelial carcinoma.

Vicineum is comprised of a recombinant fusion protein that targets epithelial cell adhesion molecule (EpCAM) antigens on the surface of tumor cells to deliver a potent protein payload, Pseudomonas Exotoxin A.

The regulatory agency has also decided not to hold an advisory committee meeting to discuss the Biologics License Application for Vicineum.

The company expects to seek the European Medicines Agency approval for Vicineum in the next one or two months.

Last July, Sesen Bio had inked a deal with China-based Qilu Pharmaceutical for the manufacture, development and commercialization of Vicineum for the treatment of BCG-unresponsive non-muscle invasive bladder cancer and other types of cancer in China, Hong Kong, Macau and Taiwan. An update on the Investigational New Drug (IND) application for Vicineum in China is expected in the coming months.

If approved, Vicineum is expected to bring in peak sales of $1 billion to $3 billion globally, with sales of $400 million to $900 million in the U.S.

SESN closed Tuesday's trading at $3.02, up 7.86%.

7. Stocks That Gained/Lost The Most In A Day

Applied Genetic Technologies Corp. (AGTC) closed Tuesday's (Feb.16, 2021) trading at $8.07, up 30.58%.

Timber Pharmaceuticals Inc. (TMBR) closed at $3.06, up 30.21%.

Akers Biosciences Inc. (AKER) closed at $4.82, up 28.53%.

Neptune Wellness Solutions Inc. (NEPT) closed at $1.94, down 30.71%.

Inhibrx Inc. (INBX) closed at $23.52, down 29.77%.

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