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MGEN Shines, NBRV Breathes Easy, SRPT Slips On FDA Snub

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Today's Daily Dose brings you news about FDA approval of Nabriva's new pneumonia antibiotic; Sarepta's disappointment; Vanda's receipt of Complete Response Letter for the expanded indication of HETLIOZ; and Miragen Therapeutics' anticipated milestones.

Read on…

Shares of Miragen Therapeutics Inc. (MGEN) rose 49.45% on Monday after H.C. Wainwright analyst Vernon Bernardino initiated coverage on the stock with a Buy rating and set a price target of $3 per share.

miRagen Therapeutics is a clinical-stage biopharmaceutical company developing proprietary RNA-targeted therapies with a specific focus on microRNAs.

Near-term Catalysts:

-- Results from phase I clinical trials for MRG-110 that assessed safety, tolerability and proof of mechanism in humans are expected in the fourth quarter of 2019. MRG-110 is a potential treatment for chronic ischemic disorders.
-- Interim data from phase II trial of Remlarsen in the prevention or reduction of keloid scarring are anticipated before the end of this year.
-- A phase II study of Cobomarsen in cutaneous T-cell lymphoma, dubbed SOLAR, is underway, with primary endpoint data expected in the first half of 2021.

MGEN closed Monday's trading at $1.36, up 49.45%.

Moderna Inc. (MRNA) has been granted Fast Track designation for its investigational Zika vaccine currently being evaluated in a phase I study for the prevention of Zika virus infection in healthy adults.

The Company is a developer of messenger RNA therapeutics and vaccines, which are designed to instruct a patient's own cells to produce proteins that could prevent, treat, or cure a disease.

Some of the other ongoing clinical programs are:

-- Phase 1 study of mRNA-1647 vaccine for Cytomegalovirus.
-- Phase 1 study of mRNA-1653 vaccine for Human metapneumovirus (hMPV) and parainfluenza type 3 (PIV3).
-- Phase 1 study of mRNA-1172 vaccine for Respiratory syncytial virus.
-- Phase 2 study investigating Merck's KEYTRUDA in combination with Moderna's mRNA-4157, compared to Keytruda alone, for the adjuvant treatment of high-risk resectable melanoma.
-- Phase 1 study of mRNA-5671 both as a monotherapy and in combination with Keytruda in patients with KRAS mutant advanced or metastatic non-small cell lung cancer, colorectal cancer or pancreatic adenocarcinoma.
-- Phase I trial of MEDI1191 injected intratumorally alone and in combination with a checkpoint inhibitor in patients with advanced solid tumors, being led by AstraZeneca.
-- Phase 1 antibody against chikungunya virus (mRNA-1944) study.
-- Phase I/II study evaluating mRNA-3704 for the treatment of methylmalonic academia.

Moderna went public on the Nasdaq Global Select Market on December 7, 2018, offering its shares at a price of $23 each.

MRNA closed Monday's trading at $13.40, up 1.82%. In after-hours, the stock was up 8.43% to $14.53.

The FDA has approved Nabriva Therapeutics plc's (NBRV) new drug applications for the oral and intravenous (IV) formulations of Xenleta (lefamulin) for the treatment of community-acquired bacterial pneumonia (CABP) in adults.

Xenleta becomes the first IV and oral antibiotic with a novel mechanism of action to be approved by the FDA in nearly two decades. The drug works by inhibiting the synthesis of bacterial protein, which is required for bacteria to grow.

Analysts expect the drug to achieve peak sales of over $600 million.

According to Nabriva, Xenleta will be available through major U.S. specialty distributors in mid-September 2019. The drug will have a wholesale acquisition price of $205 per IV patient treatment day and $275 per oral patient treatment day.

Another regulatory event to watch out for in the coming months is the resubmission of CONTEPO New Drug Application early in the fourth quarter of 2019.

Nabriva is seeking approval of CONTEPO for the treatment of complicated urinary tract infections (cUTI), including acute pyelonephritis. CONTEPO was turned down by the FDA in April 2019 due to issues related to facility inspections and manufacturing deficiencies at one of Nabriva's contract manufacturers.

NBRV closed Monday's trading at $2.21, up 3.07%. In after-hours, the stock was up 21.71% to $2.69.

The FDA has turned down Sarepta Therapeutics Inc.'s (SRPT) New Drug Application seeking accelerated approval of Golodirsen injection for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping. Patients with a 53 mutation represent 8 percent of those with Duchenne.

In its Complete Response Letter, the regulatory agency has raised concerns regarding the risk of infections related to intravenous infusion ports and renal toxicity seen in pre-clinical models of Golodirsen.

Commenting on the FDA decision, Doug Ingram, president, and chief executive officer, Sarepta, said, "We will work with the Division to address the issues raised in the letter and, to the fullest extent possible, find an expeditious pathway forward for the approval of golodirsen. We know that the patient community is waiting".

The Company already has one approved drug for Duchenne muscular dystrophy, by the name Exondys 51, but it is used to treat those with DMD caused by a mutation amenable to skipping exon 51, which affects about 13 percent of the population with DMD.

The net product sales of Exondys 51 were $181.7 million in the first half of 2019 compared to $138 million in the year-ago period.

SRPT closed Monday's trading at $120.31, down 3.71%. In after-hours, the stock fell another 13.30% to $104.31.

Another company that was snubbed by the FDA is Vanda Pharmaceuticals Inc. (VNDA).

As previously expected, the U.S. regulatory agency has declined to approve Vanda's supplemental New Drug Application for HETLIOZ for the treatment of Jet Lag Disorder, saying the measures demonstrating improved sleep are of unclear clinical significance.

Jet lag is a temporary sleep disorder that results from a mismatch between a person's circadian (24-hour) rhythms and the time of day in the new time zone.

Hetlioz is already approved for the treatment of Non-24-Hour Sleep-Wake Disorder for the totally blind. Non-24-Hour Sleep-Wake Disorder is a serious, chronic disorder that disrupts a person's circadian rhythms.

The drug brought home annual sales of $115.8 million for Vanda in 2018 compared to $89.97 million in 2017. In the first half of 2019, Hetlioz net product sales were $66.79 million.

Vanda expects to file an sNDA for Hetlioz for the treatment of Smith-Magenis Syndrome in the third quarter of 2019. A phase II clinical study of Hetlioz in delayed sleep phase disorder (DSPD) in patients who have a mutation in the CRY1 gene, which is believed to be causative in a subset of patients with DSPD, is also expected to be initiated this quarter.

VNDA closed Monday's trading at $15.33, up 0.92%.

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