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FDA Panel Snubs MNK, Decision On AKCA Drug Delayed, PTLA's Antidote Greenlighted

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Today's Daily Dose brings you news about FDA panel shooting down Mallinckrodt's pharmacologic treatment for neonatal jaundice; Sarepta's partnership with Myonexus for developing gene therapies for Limb-Girdle Muscular Dystrophies; FDA approval of Portola's antidote, and Dermira's upcoming milestones.

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The FDA decision on Akcea Therapeutics Inc.'s (AKCA) drug candidate Inotersen, with the proposed brand name TEGSEDI, has been pushed to October 6, 2018 from July 6, 2018.

TEGSEDI is an investigational drug for the treatment of patients with hereditary TTR amyloidosis (hATTR).

Meanwhile, the Company has an important catalyst to watch out for next week. The New Drug Application for Volanesorsen is scheduled to be reviewed by an FDA panel on May 10, 2018.

Volanesorsen, co-developed by Ionis and Akcea, with the proposed brand name WAYLIVRA, is an investigational therapy for the treatment of a rare lipid disorder called familial chylomicronemia syndrome (FCS).

The FDA is expected to render its final decision on Volanesorsen on August 30, 2018.
If approved, Volanesorsen would be the first therapy indicated for FCS patients.

AKCA closed Thursday's trading at $23.97, down 4.50%. In after-hours, the stock fell another 9.05% to $21.80.

Dermira Inc. (DERM) awaits a big event next month.

The FDA decision on the company's Glycopyrronium tosylate, proposed for the treatment of axillary hyperhidrosis is expected by June 30, 2018.

A Phase 2b dose-ranging study evaluating Lebrikizumab in adult patients with moderate-to-severe atopic dermatitis is underway, with data expected in first half of 2019.

DERM closed Thursday's trading at $9.15, down 2.45%.

It was a disappointment for Mallinckrodt plc (MNK) as the FDA's Gastrointestinal Drugs Advisory Committee and Pediatric Advisory Committee on Thursday voted 21 to 3 against approval of Stannsoporfin for the treatment of newborns at risk of developing severe jaundice.

Jaundice or hyperbilirubinemia is a common condition seen both in term and pre-term newborns. In the U.S., the total number of term births is estimated at 3.97 million per year, and, of those, approximately 750,000 infants are treated for jaundice, according to reports.

Stannsoporfin injection was developed by InfaCare Pharmaceutical Corp. Mallinckrodt acquired InfaCare Pharmaceutical last August.

The FDA's final decision on Stannsoporfin injection is expected on August 22, 2018.

MNK closed Thursday's trading at $12.07, down 7.15%.

The FDA has approved Portola Pharmaceuticals Inc.'s (PTLA) Andexxa, which is specifically designed to reverse the anticoagulant activity of Factor Xa inhibitors.

Factor Xa inhibitors are anticoagulants used to prevent deep vein thrombosis and pulmonary embolism in patients undergoing hip or knee replacement surgery. Some of the approved oral anticoagulant drugs are Pradaxa, Xarelto, Eliquis, and Lixiana/Savaysa. One of the major complications of treatment with these drugs is bleeding.

Andexxa becomes the first and only antidote indicated for patients treated with Eliquis and Xarelto.

Portola has licensed the development and commercial rights to Andexanet alfa in Japan to Bristol-Myers Squibb Co. (BMY) and Pfizer Inc. (PFE) while retaining full commercial rights to the antidote outside of Japan.

Analysts expect the drug to become a blockbuster.

PTLA closed Thursday's trading at $33.78, down 2.37%. In after-hours, the stock was up 3.35% to $34.91.

Sarepta Therapeutics Inc. (SRPT) has teamed up with Myonexus Therapeutics Inc. for the advancement of multiple gene therapy programs aimed at treating distinct forms of Limb-Girdle Muscular Dystrophies.

Myonexus has five LGMD gene therapy candidates - three clinical and two pre-clinical stage programs.

The most advanced of Myonexus' programs is MYO-101, being developed for the treatment of LGMD2E, also known as beta-sarcoglycanopathy, a severe and debilitating form of Limb-Girdle Muscular Dystrophy.

A Phase 1/2a study of MYO-101 is scheduled to begin in mid-2018, and the companies plan to report on 60-day biopsy data in late-2018 or early 2019.

Sarepta will make an upfront payment of $60 million and additional development-related milestone payments to purchase an exclusive option to acquire Myonexus at a pre-negotiated, fixed price with sales-related contingent payments.

If all development-related milestone payments are met, Sarepta will make payments of up to $45 million over an approximately two-year evaluation period. Sarepta has the option to purchase Myonexus at any time, including upon review of proof-of-concept data.

SRPT closed Thursday's trading at $78.17, down 0.31%.

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