BPC December 12 update

​Sarepta SRPT receives FDA Approval of Vyondys 53 for DMD

Price and Volume Movers

Sarepta Therapeutics, Inc. (NASDAQ: SRPT) received FDA approval of Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. Shares spiked 23% to $124 after hours before being halted. The approval follows the issuance of a Complete Response Letter (CRL) in August.

Assertio Therapeutics, Inc. (NASDAQ: ASRT) shares closed up 57% to $1.23 following its announcement that privately-held Alvogen will purchase its product Gralise (gabapentin) for the treatment of pain after shingles. Assertio will receive $75m in cash upon closing, expected in early January 2020, and the balance payable in the form of a royalty on the first $70m in Gralise net sales.

Amneal Pharmaceuticals, Inc. (NYSE:AMRX) announced that it has received Abbreviated New Drug Application (ANDA) approval from the FDA for EluRyng, a generic version of the vaginal ring NuvaRing used to prevent pregnancy. Shares closed up 45% to $5.04.

Assembly Biosciences, Inc. (NASDAQ:ASMB) announced a public offering priced at $16.50 per share for gross proceeds of $125m. Shares bucked the normal trend usually accustomed with offerings, closing up 34% to $23.81.

Diffusion Pharmaceuticals Inc. (NASDAQ:DFFN) shares closed down 25% to $0.43 following news of a registered direct offering of 6,266,787 shares and attached warrants at a price of $0.5585 per share and associated warrant, for gross proceeds of approximately $3.5 million.

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Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume

ADVANCERS:

CTI BioPharma Corp. (CTIC): $1.53; +26%.

Tonix Pharmaceuticals Holding Corp. (TNXP): $1.19; +22%.

PhaseBio Pharmaceuticals, Inc. (PHAS): $3.86; +18%.

Allena Pharmaceuticals, Inc. (ALNA): $2.26; +17%.

Spring Bank Pharmaceuticals, Inc. (SBPH): $3.89; +14%.

DECLINERS:

Forward Pharma A/S (FWP): 8.12; -15%.

Neon Therapeutics, Inc. (NTGN): $1.15; -15%.

Aquestive Therapeutics, Inc. (AQST): $6.70; -13%.

Spectrum Pharmaceuticals, Inc. (SPPI): $8.70; -13%

Akero Therapeutics, Inc. (AKRO): $20.28; -12%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

LIFE – aTyr Pharma Inc.
ATYR1923
Pulmonary sarcoidosis

Phase 1/2 Phase 1/2 interim data noted treatment was generally safe and well tolerated - December 12, 2019. Final data due mid-2020.
$35.5 million

SCPH – scPharmaceuticals Inc.
Furoscix
Heart failure

NDA Filing CRL issued June 13, 2018. NDA to be refiled mid-2020.
$188.4 million

SNGX – Soligenix Inc.
RiVax
Ricin toxin vaccine

Phase 1b Phase 1c trial initiation announced December 12, 2019 with initial safety data due 2Q 2020 and longer-term safety and immunogenicity data due 4Q 2020.
$62.6 million

SRPT – Sarepta Therapeutics Inc.
Golodirsen - Exon 53
Duchenne muscular dystrophy

Approved FDA Approval announced December 12, 2019.
$9.2 billion

VERU – Veru Inc.
Zuclomiphene citrate
Hot flashes

Phase 2 Phase 3 trial to commence by early summer 2020.
$281 million

VERU – Veru Inc.
VERU-111 (bisindole)
Prostate cancer

Phase 1/2 Phase 2 trials to be initiated in 2Q 2020.
$281 million