BPC March 17 update

Biotech Week in Review ~ March 17, 2017; TEN Upcoming March and April Biotech Catalysts

Weekly watchlist

AstraZeneca plc (ADR) (NYSE:AZN) presented positive data on Tuesday from its Phase 3 SOLO-2 trial of LYNPARZA for the treatment of germline BRCA-mutated (gBRCA), platinum-sensitive, relapsed ovarian cancer. The trial met its primary endpoint of investigator-assessed progression-free survival (PFS) of 19.1 months compared with 5.5 months for those on placebo. Competitor, TESARO Inc (NASDAQ:TSRO) bore the brunt of AstraZeneca’s success, with shares closing the week down 13% to $149.99.

Shares of Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX) got a welcome boost on Wednesday following news that it met the primary endpoints in its Phase 2/3 trial of Firdapse as a treatment for myasthenia gravis patients with anti-MuSK antibodies (MuSK-MG). Shares closed the week up 39% to $1.54.

AveXis, Inc. (NASDAQ:AVXS) also impressed with top-line data late on Thursday from its Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1, with all patients event-free at 13.6 months of age, sending shares up to close 15% higher on Friday at $84.00.

To round out the week on the clinical data front, Amgen (NASDAQ:AMGN) released much awaited data on Friday from its FOURIER cardiovascular outcomes trial for its approved cholesterol drug Repatha. The company had already notified in early February that both primary and secondary endpoints were met so investors were eagerly awaiting the actual data from the trial. The 15% reduction in cardiovascular events (angina, coronary revascularization, heart attack, stroke or cardiovascular death) was below investor expectations which saw shares closing the session down 6.4% to $168.61. Esperion Therapeutics Inc (NASDAQ:ESPR), which saw its shares spike 29% in early February when news that both primary and secondary endpoints were met, saw its shares reverse, closing Friday’s session down 20% to $23.67.

On the offering front shares of Editas Medicine, Inc. (NASDAQ:EDIT), Zosano Pharma Corporation (NASDAQ:ZSAN) , Anthera Pharmaceuticals, Inc. (Nasdaq:ANTH) and Aurinia Pharmaceuticals Inc. (NASDAQ:AUPH) all suffered from sharply discounted offerings. However, Aurinia is still well up over 100% since the start of March.

Upcoming March and April catalysts on watch noted below:

Drug Stage Catalyst

AEZS
Zoptrex
Endometrial cancer

Phase 3 Noted January 30, 2017 completion of Phase 3 trial with data due April 2017.

CARA
IV CR845
Uremic pruritus

Phase 2/3 Phase 2/3 top-line data from Part A portion of trial released March 28, 2017 - endpoints met. Part B to be initiated later in 2017.

CRBP
Anabasum (Resunab)
Cystic Fibrosis

Phase 2 Phase 2 data released March 30, 2017 - primary endpoint met but some concerns regarding efficacy.

GNMX
NFC-1 - SAGA
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.

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

Phase 2b Phase 2b additional interim data released April 4, 2017 at AACR. Final top-line data due mid-2018.

NBIX
INGREZZA
Tardive dyskinesia

Approved Approved April 11, 2017.

NKTR
NKTR-181
Lower back pain

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

PLX
AIR DNase (PRX-110)
Cystic Fibrosis

Phase 2 Phase 2 interim data released January 3, 2017. Full data released April 12, 2017. Company noted data were positive but results showed efficacy down on January data.

PTI
PTI-428
Cystic fibrosis

Phase 1 Phase 1 preliminary 7-day data due 2Q 2017. 28-day data due 2H 2017.

RTTR
RP-G28
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