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

Approved PDUFA date extended by three months to April 27 2017. Approval announced April 27, 2017.

Moderate-to-severe vaginal pain

CRL CRL announced May 8, 2017. Company noted that FDA cited a lack of long-term endometrial safety data. Meeting with FDA planned for November 3, 2017 with NDA to be resubmitted shortly after meeting.

Bevyxxa (betrixaban)
Venous thromboembolism (VTE) Prevention

Approved FDA Approval noted June 23, 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.

Community-acquired bacterial pneumonia (CABP)

Phase 3 Phase 3 data released April 3, 2017 - all endpoints met. Regulatory filing due 1Q 2018.

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

Phase 3 Phase 3 data released May 12, 2017. Primary endpoint met - however, competitive concerns raised.


Phase 3 Phase 3 data released September 12, 2017 - primary endpoint not met.

Durvalumab +/- tremelimumab (MYSTIC)
Lung cancer

Phase 3 Phase 3 data released July 27, 2017 - primary endpoint not met. Overall survival data due 1H 2018.

Rucaparib ARIEL3
Ovarian cancer patients with a BRCA-like mutation

Phase 3 Phase 3 data released June 20 2017 - primary endpoint met. sNDA filing due by the end of October 2017. Late breaker at ESMO September 8, 2017.