BPC November 15 update

Biotech week in Review; Biotech Phase 1/2 updated upcoming catalysts

Weekly watchlist

The final week of 3Q earnings, combined with several major clinical readouts and regulatory news releases, produced one of the most eventful price-moving weeks of the year which we highlight below. Given the number of data readouts and pipeline updates over the past few weeks, we also update key catalysts remaining for the final six weeks of the year, with a focus on Phase 2 events, with Phase 3 catalysts to be updated next week.

Amarin Corporation plc (NASDAQ: AMRN) announced Thursday that the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the FDA voted 16-0 to recommend approval of an indication and label expansion for Vascepa capsules to reduce the risk of cardiovascular events in high-risk patients. The PDUFA date for a final decision by the FDA is December 28, 2019. Shares closed the week up 37% to $24.02.

resTORbio, Inc (Nasdaq: TORC) announced that top line data from its Phase 3 trial evaluating RTB101 in preventing clinically symptomatic respiratory illness (CSRI) in adults age 65 and older, did not meet its primary endpoint. Shares slumped to close Friday down 87% to $1.09.

VistaGen Therapeutics (NASDAQ: VTGN) shares slid to close Thursday down 72% to $0.30 following news its Phase 2 trial of AV-101, as an adjunctive treatment of major depressive disorder (MDD), did not meet the primary endpoint.

NextCure, Inc. (NASDAQ: NXTC) shares closed the week down 57% to $36.05. Updated data from its pipeline candidate, NC318, released at the SITC conference noted that there were no further responses in additional patients with non-small cell lung cancer (NSCLC) compared with earlier data released in the initial abstract for the meeting.

ASLAN Pharmaceuticals (NASDAQ:ASLN) announced top-line data from its Phase 2/3 TreeTopp trial of varlitinib in patients with biliary tract cancer, did not met the primary endpoints of progression-free survival (PFS) and overall response rate (ORR). Shares closed the week down 78% to $0.45.

Lipocine Inc. (NASDAQ: LPCN) announced it received a Complete Response Letter (CRL) from the FDA for its New Drug Application (NDA) for Tlando in males with conditions associated with a deficiency of endogenous testosterone. Shares slumped to close the week down 86% to $0.39.

Solid Biosciences Inc. (Nasdaq: SLDB) announced its Phase 1/2 trial of SGT-001 for the treatment of Duchenne muscular dystrophy (DMD), has been placed on clinical hold by the FDA. Shares closed the week down 68% to $3.51.

Kadmon Holdings, Inc. (NYSE: KDMN) shares closed the week up 30% to $3.92 following data released Monday from its ROCKstar trial of KD025 in patients with chronic graft-versus-host disease (cGVHD), which met the primary endpoint of Overall Response Rate (ORR).

Agile Therapeutics, Inc. (Nasdaq: AGRX) shares closed Friday down 37% to $1.49 on news that the FDA has extended the PDUFA date for its review of the New Drug Application (NDA) of its Twirla contraceptive patch, from November 16, 2019 to February 16, 2020. The delay follows submission by the company of requested information by the FDA, which were determined to be a major amendment and will take additional time to review.

Rubius Therapeutics, Inc. (NASDAQ: RUBY) shares closed the week down 33% to $7.36 following news data from its Phase 1b trial of RTX-134 for the treatment of patients with phenylketonuria, will now be released in 1Q 2020. The company had previously guided that data would be released this year.


Key Phase 1/2 catalysts left for 2019:

Drug Stage Catalyst Market Cap

BIIB – Biogen Inc.
BIIB092 - Anti Tau
Progressive supranuclear palsy (PSP)

Phase 2 Phase 2 trial did not meet primary endpoint - December 13, 2019.
$52.4 billion

BLUE – bluebird bio Inc.
ide-cel bb2121 - KarMMa
Relapsed/refractory multiple myeloma

BLA Filing BLA filing announced March 31, 2020.
$2.3 billion

BMY – Bristol-Myers Squibb Company
liso-cel (JCAR017) - TRANSCEND NHL-001
Relapsed or refractory (R/R) large B-cell lymphoma

PDUFA priority review PDUFA date under priority review August 17, 2020.
$128.7 billion

BPMC – Blueprint Medicines Corporation
Avapritinib BLU-285 - PIONEER
Indolent and smoldering systemic mastocytosis (SM)

Phase 2 Phase 2 updated data presented March 16, 2020 - no patients discontinued treatment due to AEs. Enrolment in Part 2 of the PIONEER trial to be completed by the end of 2020.
$3.5 billion

EVFM – Evofem Biosciences Inc.
Prevention of urogenital Chlamydia trachomatis infection in women

Phase 2/3 Phase 2b data met primary endpoint - December 2, 2019.
$252.7 million

FIXX – Homology Medicines Inc.
Phenylketonuria (PKU)

Phase 1/2 Phase 1/2 update due mid-2020.
$742.7 million

KALV – KalVista Pharmaceuticals Inc.
Diabetic macular edema (DME)

Phase 2 Phase 2 data did not meet primary endpoint - December 9, 2019.
$137.6 million

MGEN – Miragen Therapeutics Inc.
MRG-201 Remlarsen

Phase 2 Phase 2 interim data released December 11, 2019 - generally safe and well tolerated. Trial to continue through to primary endpoint.
$21.8 million

NERV – Minerva Neurosciences Inc
Major Depressive Disorder

Phase 2b Phase 2b data failed to meet endpoints - December 18, 2019. Development to be discontinued in MDD.
$215.7 million

PTI – Proteostasis Therapeutics Inc.
PTI-428 + PTI-801 + PTI-808
Cystic fibrosis

Phase 2 Phase 2 negative data released December 17, 2019. Mean changes in ppFEV1 were not statistically significant in the heterozygous population. Phase 3 trial to be initiated in 2020.
$57.9 million

SGEN – Seattle Genetics Inc.
(Tucatinib ONT-380) + Herceptin and Xeloda - HER2CLIMB trial
HER2+ Metastatic Breast Cancer (MBC)

PDUFA priority review PDUFA date August 20, 2020 under priority review.
$21 billion

SPPI – Spectrum Pharmaceuticals Inc.
Poziotinib - Zenith20
Non-small cell lung cancer (NSCLC) with exon 20 insertion mutation in EGFR or HER2

Phase 2 Phase 2 primary endpoint from Cohort 1 was not met - December 26, 2019.Cohort 2 data due mid-2020 with Cohort 3 data due year-end 2020.
$261.7 million

VRTX – Vertex Pharmaceuticals Incorporated
Sickle cell disease

Phase 1/2 Phase 1/2 further data due in 2020.
$64.2 billion

WVE – Wave Life Sciences Ltd.
Huntington’s disease

Phase 1/2 Phase 1b/2a top line data released December 30, 2019 noted statistically significant reduction of 12.4% (p<0.05) in mutant huntingtin (mHTT) protein but no difference in total HTT (tHTT) protein. 32 mg data due 2H 2020.
$267.4 million

WVE – Wave Life Sciences Ltd.
Suvodirsen (WVE-210201)
DMD Exon 51

Phase 1 Phase 1 development to be discontinued following interim analysis - December 16, 2019.
$267.4 million

XLRN – Acceleron Pharma Inc.

Phase 2 Phase 2 data at ASH December 2019 noted primary endpoint met in 10%~32% of patients across four cohorts.
$4.6 billion