BPC February 25 update

Biotech Week in Review - February 24; Ten upcoming 1Q Biotech Catalysts; Historical Catalyst Calendar coming next week to BioPharmCatalyst

Weekly watchlist

The shortened trading week dominated by a number of clinical trial readouts and key regulatory decisions brought for the most part disappointing readouts for long investors.

The trend of poor trial and regulatory news will be reflected in the next tool released by BioPharmCatalyst early next week. The BioPharmCatalyst Historical Catalyst Calendar will provide  a snapshot of recently released catalysts, allowing users to track the history of clinical and regulatory key decisions.

In regulatory news, Amphastar Pharmaceuticals, Inc. (NASDAQ:AMPH) delivered disappointing news to start up the week, with shares dropping 23% to $14.20 following the receipt of a Complete Response Letter (CRL) for its New Drug Application (NDA) for Intranasal Naloxone, indicated for the emergency treatment of known or suspected opioid overdose.

On the clinical trial front, negative data started up Tuesday with Trevena, Inc. (NASDAQ:TRVN), which saw its shares plunge 40% to $4.27 following the release of top-line data from its Phase 3 APOLLO-1 and APOLLO-2 pivotal efficacy studies of oliceridine in moderate-to-severe acute pain following bunionectomy and abdominoplasty. Although the company noted that the primary endpoint was met across three doses, not all secondary endpoints were met, resulting in concerns of the commercial viability of the product.

Also on Tuesday, Cidara Therapeutics, Inc. (Nasdaq:CDTX) announced that it will not be furthering development of CD101 for the treatment of acute vulvovaginal candidiasis (VVC) following a lack of efficacy shown in its Phase 2 RADIANT trial. The news sent shares down 38% on Monday to $7.20.

Wednesday brought further disappointing data with shares of Argos Therapeutics Inc. (Nasdaq:ARGS) plummeting to close down 66% to $1.48 following a recommendation from the Independent Data Monitoring Committee (IDMC) that its pivotal Phase 3 ADAPT trial for the treatment of metastatic renal cell carcinoma (mRCC) be discontinued due to poor efficacy.

Ocular Therapeutix, Inc. (NASDAQ:OCUL) shares dropped 9% to $8.32 following news that the FDA accepted its New Drug Application (NDA) resubmission for DEXTENZA for intracanalicular use for the treatment of ocular pain occurring after ophthalmic surgery. A Class 2, 6-month response was designated by the FDA with a PDUFA date of July 19, 2017. Investors had hoped for a Class 1, 2-month response, hence the negative reaction to the share price.

Cyclacel Pharmaceuticals, Inc. (Nasdaq:CYCC) followed with news on Thursday that its Phase 3 SEAMLESS study in elderly patients aged 70 years or older with newly diagnosed acute myeloid leukemia (AML) did not meet its primary endpoint. The company had already indicated in December 2014 that interim analysis suggested the trial would not be successful. However, the news proved to be unexpected for some with shares closing down 25% on the day to $4.05.

The good news in terms of clinical trial readouts was seen at the close of the week when on Friday Cempra, Inc. (Nasdaq: CEMP) announced that its Phase 3 trial of oral fusidic acid for the treatment of Acute bacterial skin and skin structure infections (ABSSSI) met primary and secondary endpoints. The positive news sent shares surging to close up 29% to $4.05. The news was certainly a relief to investors who had seen its share price plunge following its Complete Response Letter (CRL) in December 2016 for Solithera.

Just five weeks remain in the first quarter of 2017. A number of late-stage catalysts still remain on the road map, ten of which are noted below:

Drug Stage Catalyst

mGluR mutation positive ADHD

Phase 2/3 Phase 2/3 data released March 20, 2017 - primary endpoint not met. Subset of data released April 20, 2017 - to be presented April 21, 2017.


PDUFA priority review Phase 3 data released February 27, 2016 - primary endpoint hit. Data published May 21, 2017 confirmed primary endpoint was met but with lack of mortality improvement. PDUFA date under priority review February 28, 2018. No Advisory Committee Meeting planned.

Telotristat etiprate (XERMELO)
Carcinoid Syndrome

Approved Approved February 28, 2017.

Selinexor - SADAL
Cancer - Diffuse Large B-Cell Lymphoma (DLBCL)

Phase 2b Phase 2b additional interim data released June 23, 2017 at EHA. Final top-line data due 2H 2018.

ND0612H (Trial 006)
Severe Parkinson's Disease

Phase 2 Phase 2 trial met key primary and secondary endpoints - March 1, 2017.

Lower back pain

Phase 3 Phase 3 data released March 20, 2017 - endpoints met.

Roche (RHHBY) - Perjeta + Herceptin (APHINITY)
Adjuvant HER2+ breast cancer

Phase 3 Note this trial is conducted by competitor Roche (RHHBY), NOT by Puma. Primary endpoint was met March 2, 2017. Data released June 5, 2017 - HR 0.81. Disease-free-survival +0.9% over Herceptin.

Cystic fibrosis

Phase 2 Phase 2 28-day data due 4Q 2017.

Chronic pain

CRL CRL issued September 26, 2016. NDA to be resubmitted in 1Q 2018 following additional trials to be completed by the end of 2017.

Lactose intolerance

Phase 2/3 Phase 2/3 top-line data released March 28, 2017. Primary endpoint met. End of Phase 2 meeting with FDA planned by the end of 2017 with Phase 3 trial to potentially commence 1H 2018.