15 Biotech Stock Catalysts for 4Q 2017; Week in Review ~ October 13, 2017
The second trading week of the fourth quarter saw another steady release of biotech news flow. Six such events are listed below. This week’s watch list takes a look at 15 biotech catalysts slated for release this quarter. A range of small/large cap companies are noted with data from Phase 1-3 trials.

AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX) shares closed down 60% to $2.15 Thursday following the issuance of a Complete Response Letter (CRL) from the FDA for its New Drug Application (NDA) for Dsuvia for treatment of post-operative pain. The FDA noted that additional data on at least 50 patients would be required to assess the safety of Dsuvia.

Antares Pharma, Inc. (NASDAQ:ATRS) shares closed Friday down 38% to $2.32 following its announcement after hours Thursday that the FDA had identified deficiencies for its NDA for XYOSTED (testosterone enanthate) injection. The set PDUFA date of October 20, 2017 still stands. However, given the circumstances it is almost without a doubt that the company will be issued a CRL.

Spark Therapeutics (NASDAQ:ONCE) achieved a major milestone Thursday when a FDA Advisory Committee voted unanimously (16-0) to recommend approval of Luxturna, a gene therapy for retinal disease. The PDUFA date under priority review for the final decision by the FDA is January, 12, 2018.

AnaptysBio, Inc. (Nasdaq:ANAB) shares soared to close the week up 95% to $69.55 following the release of positive data from its Phase 2a trial of ANB020 in adult patients with moderate-to-severe atopic dermatitis.

Aerie Pharmaceuticals, Inc. (NASDAQ:AERI) announced that a FDA Advisory Committee voted in support of approval for Rhopressa for the treatment of glaucoma. The expert panel voted 9-1 in favor that the efficacy of Rhopressa, outweigh the safety risks identified. The PDUFA date is February 28, 2018.

Ardelyx, Inc. (NASDAQ: ARDX) shares closed the week up 22% to $7.28 following promising data from its Phase 3 T3MPO-2 trial of tenapanor for the treatment of irritable bowel syndrome with constipation (IBS-C). The primary endpoint, the combined responder rate for six of 12 weeks, showed that 36.5% of patients, compared with 23.7% on placebo ( p<0.001), had at least a 30% reduction in abdominal pain and an increase of one or more complete spontaneous bowel movements (CSBM) in the same week for at least six of the 12 weeks of the treatment period. Rates of diarrhea were higher in the tenapanor arm (16.0% vs. 3.7%). Results from its first Phase 3 trial, released in May, showed primary endpoint data of 27% vs 18.7% (p=0.02) and rates of diarrhea of 14.6% vs 1.7% for placebo.



15 Biotech Catalysts slated for 4Q 2017:

DRUG STAGE CATALYST
TICKER: ABBV

Risankizumab

Psoriasis

PHASE 3 Phase 3 data due by the end of 2017.
TICKER: ALXN

Eculizumab

Refractory generalized myasthenia gravis (gMG)

PDUFA (sBLA) PDUFA date October 23, 2017.
TICKER: AXON

Intepirdine - HEADWAY-DLB

Dementia with Lewy bodies (DLB)

PHASE 2B Phase 2b data due 4Q 2017.
TICKER: BLUE

LentiGlobin - HGB-206

Sickle disease

PHASE 1/2 Update at ASH 2016. Further data due at ASH 2017. Exact date TBC.
TICKER: CALA

CB-839 + nivolumab

Renal cell carcinoma, malignant melanoma and non-small cell lung cancer

PHASE 1/2 Phase 1/2 enrollment commenced August 2016. Oral presentation November 11, 2017 at 32nd Annual Meeting of the Society for Immunotherapy of Cancer (SITC).
TICKER: CEMP

Taksta - Fusidic acid

Refractory infections in bones and joints

PHASE 2/3 Phase 2/3 data due by the end of 2017.
TICKER: CRIS

CA170

Solid Tumors and Lymphomas

PHASE 1 Phase 1 initial data at SITC Meeting - November 8-12, 2017.
TICKER: CYTK

Tirasemtiv - VITALITY

Amyotrophic lateral sclerosis (ALS)

PHASE 3 Phase 3 data are due 4Q 2017.
TICKER: DBVT

Viaskin Peanut (PEPITES)

Peanut allergy

PHASE 3 Phase 3 top-line data due October 2017.
TICKER: EARS

AM-111 HEALOS

Acute inner ear hearing loss

PHASE 3 Phase 3 HEALOS completion of enrollment announced June 5, 2017. Data due 4Q 2017 (likely late November).
TICKER: FPRX

Cabiralizumab (FPA008) and Opdivo (nivolumab)

Solid tumors

PHASE 1B Phase 1a/1b late breaker at SITC Meeting - November 11, 2017.
TICKER: LOXO

LOXO-292

RET-fusion non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC) and other tumors

PHASE 1 Phase 1 initial data to be presented October 18, 2017.
TICKER: PTI

PTI-428

Cystic fibrosis

PHASE 2 Phase 2 28-day data due 4Q 2017.
TICKER: RVNC

RT002 - SAKURA 1

Moderate to severe glabellar (frown) lines

PHASE 3 Phase 3 completion of enrollment announced March 8, 2017. Data due 4Q 2017.
TICKER: SAGE

SAGE-217

Major Depressive Disorder (MDD)

PHASE 2 Phase 2 initiation announced December 19, 2016. Part B data due 2H 2017.