BPC March 03 update

Top ten biotech events for the week ending March 3, 2017; 10 biotech events for the upcoming month

Weekly watchlist

The plate was certainly full of data from biotech binary events released over the last week. Given the plethora of news, we’ll break it down into short and sharp summaries of our top ten events for the week.


La Jolla Pharmaceutical Company (NASDAQ: LJPC) shares surged to close the week up 74% to $34.53 after it announced that it met the primary endpoint in its Phase 3 ATHOS-3 trial of LJPC-501 in patients with catecholamine resistant hypotension (CRH). Key secondary endpoint data were not disclosed.

Kite Pharma, Inc., (Nasdaq:KITE) met the primary endpoint in its ZUMA-1 for its lead CAR-T candidate, axicabtagene ciloleucel (KTE-C19), in patients with chemorefractory aggressive B-cell non-Hodgkin lymphoma (NHL). After six months 31% of diffuse large B-cell lymphoma (DLBCL) patients showed a complete response. Shares closed the week up 38% to $72.53.

Aurinia Pharmaceuticals Inc. (NASDAQ:AUPH) shares closed the week up 29% to $4.81 after it announced on Wednesday that it met complete and partial remission endpoints in its Phase 2b AURA-LV (AURA) trial in lupus nephritis. 49% of patients obtained a complete response compared with 24% on placebo with no additional deaths.

Dynavax Technologies Corporation (NASDAQ:DVAX) shares closed up 54% for the week to $6.25 following receipt of a PDUFA date of August 10, 2017 for the resubmission of its Complete Response Letter (CRL) for HEPLISAV-B, a vaccine against hepatitis B infection.

NeuroDerm Ltd. (Nasdaq:NDRM) met key endpoints in its Phase 2 trial of ND0612H, in patients with advanced Parkinson's disease. Shares closed the week up 23% to $28.95.


Puma Biotechnology Inc (NASDAQ:PBYI) shares lost 14% to $32.80 on Thursday after news that Roche (OTCQX: RHHBY) met the primary endpoint in its Phase 3 APHINITY trial of study of Perjeta and Herceptin and chemotherapy. Puma is developing neratinib and chemotherapy against Herceptin.

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that it did not achieve the primary or secondary endpoints in its Phase 3 trial of ataluren in cystic fibrosis. Shares closed down 20% to $10.58 on Thursday.

Juno Therapeutics, Inc. (NASDAQ: JUNO) shut up shop with its CAR-T program of JCAR015. Shares closed the week at $21.61, down 17% from Wednesday’s close.

SCYNEXIS Inc (NASDAQ:SCYX) shares closed down Friday by 17% to $2.70 following news that the FDA has placed a clinical hold on intravenous (IV) SCY-078 following mild-to-moderate thrombotic events in healthy patients.

Karyopharm Therapeutics Inc. (Nasdaq:KPTI) actually finished the week in the green but lost 16% on Friday to $11.63 following news that it will not meet the primary endpoint in its Phase 2 SOPRA trial of selinexor in relapsed/refractory acute myeloid leukemia (AML).

Upcoming Catalysts below for the next month:

Drug Stage Catalyst

Improve walking in patients with multiple sclerosis

Decision on Inter Partes Review (IPR) released March 10, 2017 - patents upheld.

Lynparza - SOLO 2
Second-line ovarian cancer

Approved Approval announced August 17, 2017.

Cardiovascular disease

PDUFA Phase 3 cardiovascular outcomes study data released March 17, 2017 - endpoints met but down on expectations. PDUFA date under priority review December 2, 2017.

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. Phase 3 trial to be initated 4Q 2017.

Anabasum (Resunab)
Cystic Fibrosis

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

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.

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.

Lower back pain

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

Cystic fibrosis

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

TYMLOS (Abaloparatide)

Approved PDUFA date originally scheduled for March 30 2017 delayed by three months to June 30, 2017. Approval announced April 28, 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.