BioPharm Update:


​Puma PBYI receives FDA Approval; Repros RPRX clinical hold remains in place; NVAX data July 24
Puma Biotechnology, Inc. (Nasdaq: PBYI) announced that the FDA has approved NERLYNX (neratinib) for the extended adjuvant treatment of adult patients with early stage HER2-overexpressed/amplified breast cancer. Approval was based on the placebo controlled ExteNET trial. After two years, invasive disease-free survival (iDFS) was 94.2% in patients treated with neratinib compared with 91.9% in those receiving placebo (HR 0.66; 95% CI: 0.49, 0.90, p=0.008). Shares are currently trading up 9% to $93.65 in the after-hours session.

Paratek Pharmaceuticals, Inc. (NasdaqRTK) announced that it met all primary and secondary endpoints in its Phase 3 trial of omadacycline for the treatment of acute bacterial skin and skin structure infections (ABSSSI). Shares are currently trading up 4.2% in the after-hours session to $26.00 after closing up 3.7% during normal trading hours.

Repros Therapeutics Inc., (Nasdaq:RPRX) noted that the FDA has kept in place the clinical hold for Proellex. The FDA also indicated that the company will be required to compile a large pre-approval safety data base to support future development. Shares slumped to close down 33% to $0.385.

Novavax, Inc. (Nasdaq:NVAX) announced it will host a conference on Monday, July 24, 2017, at 4:30 p.m. ET where it will announce top-line data from its Phase 2 safety and immunogenicity trial of the RSV F Vaccine in older adults (60 years of age and older).

Spark Therapeutics (NASDAQ:ONCE) announced that the FDA has granted priority review for the Biologics License Application (BLA) of voretigene neparvovec, for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD). The PDUFA date is January 12, 2018.

CymaBay Therapeutics, Inc. (Nasdaq:CBAY) announced interim results from its ongoing low-dose Phase 2 study of seladelpar in patients with primary biliary cholangitis (PBC). A planned interim analysis showed after 12 weeks of treatment a significant reduction in alkaline phosphatase (AP) levels of of 39% and 45% for the 5 mg and 10 mg groups, respectively. Shares closed up 6% to $5.98 after trading as high as $7.77 early in the session. After hours, the company announced that it intends to offer and sell 10m shares of its common stock.



Other Major price movers (stocks priced > $1.00, volume > 100k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume INCLUDING sub-$1 stocks.

ADVANCERS:

Soligenix, Inc. (NASDAQ:SNGX): $2.82; +36%.

AcelRx Pharmaceuticals Inc (NASDAQ:ACRX): $3.15; +31%.

ImmunoCellular Therapeutics Ltd (NYSEMKT:IMUC): $1.22; +18%.

Inotek Pharmaceuticals Corp (NASDAQ:ITEK): $1.05; +13%.

Mirati Therapeutics, Inc. (NASDAQ:MRTX): $4.35; +12%.

DECLINERS:

Cyclacel Pharmaceuticals Inc (NASDAQ:CYCC): $3.08; -12%.

Concordia International Corp (NASDAQ:CXRX): $1.59; -10%.

Radius Health Inc (NASDAQ:RDUS): $42.97; -9%.

Dimension Therapeutics Inc (NASDAQMTX): $1.30; -7%.

Sangamo Therapeutics Inc (NASDAQ:SGMO): $9.40; -7%.



Pipeline updates below:

DRUG STAGE CATALYST
TICKER: AMGN

EVENITY (Romosozumab)

Osteoporosis

CRL CRL issued July 16, 2017.
TICKER: AXSM

AXS-05 ADVANCE-1

Agitation in patients with Alzheimer’s disease (AD)

PHASE 2/3 Phase 2/3 initiation announced July 17, 2017.
TICKER: CBAY

Seladelpar

Primary biliary cholangitis (PBC)

PHASE 2 Phase 2 top-line data released late May 2016. Positive interim data from second trial data released July 17, 2017.
TICKER: NVAX

RSV F Vaccine immunogenicity trial

Respiratory Syncytial Virus (RSV) in older adults

PHASE 2 Phase 2 trial initiation announced January 19, 2017. Data to be released July 24, 2017.
TICKER: PBYI

Neratinib (NERLYNX)

Extended adjuvant HER2-positive early stage breast cancer

APPROVED Approval announced July 17, 2017. Advisory Committee Meeting May 24, 2017 voted 12-4 in favor of recommending approval.
TICKER: ONCE

Voretigene neparvovec (SPK-RPE65)

Inherited retinal dystrophies

PDUFA PRIORITY REVIEW PDUFA date January 12, 2018 under priority review.
TICKER: PRTK

Omadacycline - oral

Acute bacterial skin and skin structure infections (ABSSSI)

PHASE 3 Phase 3 data released July 17, 2017 - all endpoints met.
TICKER: RDHL

BEKINDA

IBS-D

PHASE 2 Phase 2 top-line data due September 2017. Noted July 17, 2017 that last patient has been enrolled.
TICKER: RPRX

Proellex

Uterine fibroids (oral treatment)

PHASE 2B Noted July 17, 2017 that clinical hold remains in place. Also noted large pre-approval safety data base required to support future development.