BPC April 01 update

Biotech Week in Review ~March 31, 2017; Upcoming PDUFA and biotech data catalysts for 2Q / mid-2017

Weekly watchlist

We highlight five significant biotech events as we look back at the final trading week of 1Q 2017.

On the clinical data front, Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) led the way when it announced Tuesday positive data from its Phase 3 trials of the tezacaftor / ivacaftor combination treatment in patients with cystic fibrosis patients. Shares closed the week at $109.35, up 22% from Tuesday's close. The company intends to submit a New Drug Application (NDA) in 3Q 2017.

Proteostasis Therapeutics, Inc. (NASDAQ:PTI) shares shed 28% to $7.82 on Friday following its announcement after-hours Thursday of a delay in the release of Phase 1 data from its trial of PTI-428 for the treatment of cystic fibrosis. Data originally set for release last quarter will now be released in 2Q (preliminary 7-day data) and 2H 2017 (28-day data). 

Novartis (NYSE:NVS) announced Wednesday that it has been awarded a priority review for its Biologics License Application (BLA) filing for CTL019, its CAR-T therapy for patients with B-cell acute lymphoblastic leukemia (ALL). CAR-T therapy involves extracting T cells from a patient's blood, which are altered through genetic coding before being sent back into the patient's blood stream to attack the cancer cells. Novartis stands to be the first company to be awarded approval for a CAR-T therapy.

Late Friday, Kite Pharma, Inc. (Nasdaq:KITE) announced that it has completed its rolling BLA for its CAR-T therapy, axicabtagene ciloleucel (KTE-C19), as a treatment for patients with relapsed or refractory aggressive non-Hodgkin lymphoma (NHL).

TESARO, Inc. (NASDAQ: TSRO) announced Monday that the FDA approved its PARP inhibitor ZEJULA (niraparib) for the maintenance treatment of ovarian cancer, three months ahead of its PDUFA action date. The approval also came with the broadest label compared with its competitors AstraZeneca (Lynparza) and Clovis Oncology Inc (Rubraca).

Foamix Pharmaceuticals Ltd. (NASDAQ: FOMX) led the decliners among those releasing clinical data. Shares plunged to close the week down 46% to $4.95 following news that it only met three of four primary endpoints in its two Phase 3 trials of FMX101 in patients with moderate-to-severe acne. 

A look below at a sample of upcoming 2Q/mid-2017 PDUFA binary events and scheduled releases of clinical data:

Drug Stage Catalyst

Tardive dyskinesia

Approved Approved April 11, 2017.

Cerliponase alfa
Batten Disease

PDUFA PDUFA date extended by three months to April 27 2017

Moderate-to-severe vaginal pain

PDUFA PDUFA date May 7, 2016. Noted April 10, 2017 that FDA has identified deficiencies that preclude discussion of labeling and postmarketing requirements. Further noted April 19, 2017 that it expects a decision on or before the PDUFA date - (reading between the lines this seems to infer that the NDA will be rejected).

Betrixaban (The APEX Study)
Venous thromboembolism (VTE) Prevention

PDUFA priority review PDUFA date under priority review June 24, 2017. Phase 3 data released March 2016 did not meet primary endpoint.

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.

Community-acquired bacterial pneumonia (CABP)

Phase 3 Phase 3 data released April 3, 2017 - all endpoints met.

Tenapanor (T3MPO-1)
Constipation-predominant irritable bowel syndrome (IBS-C)

Phase 3 Phase 3 data due 2Q 2017.


Phase 3 Phase 3 data due 1H 2017.

Durvalumab +/- tremelimumab (MYSTIC)
Lung cancer

Phase 3 Phase 3 PFS data mid-2017 and final OS data at the latest in 2018.

Rucaparib ARIEL3
Ovarian cancer patients with a BRCA-like mutation

Phase 3 Pivotal trial initiated late 2013. Enrollment completed 2Q 2016. Data due mid-2017.