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.

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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 data due 4Q 2019.
$54.2 billion

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

Phase 2 Phase 2 data met primary and secondary endpoints - December 6, 2019.
$4.4 billion

BMY – Bristol-Myers Squibb Company
liso-cel (JCAR017) - TRANSCEND NHL-001
r/r B cell non-Hodgkin lymphoma - cancer

Phase 1 Phase 1 data due at ASH December 7, 8:45 a.m ET.
$140.5 billion

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

Phase 2 Phase 2 initial data due at ASH December 8, 2019 at 6pm ET.
$3.9 billion

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

Phase 2/3 Phase 2b data met primary endpoint - December 2, 2019.
$322.1 million

FIXX – Homology Medicines Inc.
HMI-102
Phenylketonuria (PKU)

Phase 1/2 Phase 1/2 data due 4Q 2019.
$800.1 million

KALV – KalVista Pharmaceuticals Inc.
KVD001
Diabetic macular edema (DME)

Phase 2 Phase 2 data due 4Q 2019.
$255.3 million

MGEN – Miragen Therapeutics Inc.
MRG-201 Remlarsen
Keloids

Phase 2 Phase 2 data due 4Q 2019.
$18.4 million

NERV – Minerva Neurosciences Inc
MIN-117
Major Depressive Disorder

Phase 2a Phase 2b data due 4Q 2019.
$243.9 million

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

Phase 2 Phase 2 data due 4Q 2019.
$181.5 million

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

Phase 2 Phase 2 trial met primary and key secondary endpoints - October 21, 2019. Data to be presented at SABCS December 11, 2019. NDA filing due 1Q 2020.
$20.1 billion

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

Phase 2 Phase 2 top-line data from Cohort 1 due December 2019. Cohort 2 data due mid-2020.
$1.1 billion

VRTX – Vertex Pharmaceuticals Incorporated
CTX001
Sickle cell disease

Phase 1/2 Phase 1/2 initial data noted patient is free of vaso-occlusive crises - November 19, 2019.
$57.4 billion

WVE – Wave Life Sciences Ltd.
WVE-120102
Huntington’s disease

Phase 1/2 Phase 1b/2a top line data due by YE 2019.
$1.1 billion

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

Phase 1 Phase 1 interim efficacy data from open-label trial due 4Q 2019.
$1.1 billion

XLRN – Acceleron Pharma Inc.
Luspatercept
Myelofibrosis

Phase 2 Phase 2 data due at ASH December 9, 7:00 a.m. EST.
$2.6 billion