BPC May 14 update

Myovant MYOV meets primary but shares fall 25%; MeiraGTx MGTX meets early stage trial endpoint +21%

Price and Volume Movers

Myovant Sciences (NYSE: MYOV) announced data from its Phase 3 Liberty 1 trial, the first of two Phase 3 trials of relugolix for the treatment of uterine fibroids. The trial met its primary efficacy endpoint and six key secondary endpoints. In the primary endpoint analysis, 73.4% of women receiving once daily oral relugolix combination therapy achieved the responder criteria compared with 18.9% of women receiving placebo (p < 0.0001). Shares, however, closed down 25% to $12.83 as investors compared data with Abbvie’s Elagolix with concerns on how, if approved, it would compete with the larger company. The placebo adjusted response rate of 55% compared with 60% and 66% from two trials conducted by Abbvie. Data from a second Phase 3 trial are due next quarter.

MeiraGTx Holdings plc (NASDAQ:MGTX) shares closed up 21% to $21.50 following news that its Phase 1/2 dose escalation trial of AAV-RPE65, for the treatment of RPE65-deficiency, a condition that causes blindness, achieved the primary endpoint of safety and tolerability.

Lipocine Inc. (NASDAQ: LPCN) shares closed up 9% to $1.93. The company announced that the FDA has accepted its New Drug Application for Tlando as testosterone replacement therapy in adult males. The PDUFA date is November 9, 2019. The company previously received a Complete Response Letter (CRL) in May 2018.

INSYS Therapeutics, Inc. (NASDAQ: INSY) shares, which slumped 74% Monday on bankruptcy concerns, pared back some of its losses to close Tuesday up 31% to $1.24, but still well below Friday’s close of $3.60.

Athersys, Inc. (Nasdaq: ATHX) shares closed up 14% to $1.80 on news its program evaluating MultiStem cell therapy for the treatment of Acute Respiratory Distress Syndrome (ARDS) has received Fast Track designation from the FDA.

Solid Biosciences Inc. (Nasdaq: SLDB) shares closed down 29% to $6.32 following a series of analyst downgrades resulting form news released yesterday of a serious adverse event in one patient of the second cohort in its Phase 1/2 IGNITE DMD trial of SGT-001.

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Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume

ADVANCERS:

Novavax, Inc. (NVAX): $7.13; +24%.

Bio-Path Holdings, Inc. (BPTH): $18.00; +22%.

Verastem, Inc. (VSTM): $1.50; +16%.

Arena Pharmaceuticals, Inc. (ARNA): $54.85; +13%.

Trevi Therapeutics, Inc. (TRVI): $8.90; +12%.

DECLINERS:

Navidea Biopharmaceuticals, Inc (NAVB): $1.65; -12%.

Armata Pharmaceuticals, Inc. (ARMP): $3.63; -10%.

Idera Pharmaceuticals, Inc. (IDRA): $2.82; -10%.

Forty Seven, Inc. (FTSV): $16.04; -10%.

Sienna Biopharmaceuticals, Inc. (SNNA): $1.32; -8%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ACER – Acer Therapeutics Inc.
ACER-001
Urea cycle disorder (UCD)

Phase 2 Pivotal bridging trial to be initiated 4Q 2019 with NDA filing mid-2020.
$35.3 million

ALT – Altimmune Inc.
HepTCell
Hepatitis B

Phase 1 Phase 2 trial planned for 2020.
$32.5 million

APTX – Aptinyx Inc.
NYX-2925
Fibromyalgia

Phase 2 Phase 2 trial to be initiated 2H 2019 with data due 1H 2021.
$135 million

ARDS – Aridis Pharmaceuticals Inc.
AR-105 (Aerucin)
Pseudomonas aeruginosa

Phase 2 Phase 2 trial did not met primary endpoint - September 3, 2019.
$67.7 million

AUPH – Aurinia Pharmaceuticals Inc
Voclosporin - AURORA
Lupus

Phase 3 Phase 3 data due 4Q 2019.
$546.6 million

AUTL – Autolus Therapeutics plc
AUTO3 - (ALEXANDER)
Relapsed or refractory diffuse large B-celllymphoma (DLBCL)

Phase 1/2 Phase 1 interim data due at ASH 2019. Phase 2 decision on initiation in DLBCL is expected in mid-2020
$572.2 million

BLRX – BioLineRx Ltd.
BL-8040 in combination with KEYTRUDA - COMBAT trial
Pancreatic cancer

Phase 2 Phase 2 top-line data due 2H 2019 with PFS data due mid-2020.
$28.8 million

BLRX – BioLineRx Ltd.
BL-8040
Consolidation treatment for AML cancer patients

Phase 2b Phase 2b interim data due 2H 2019.
$28.8 million

CATB – Catabasis Pharmaceuticals Inc.
Edasalonexent (CAT-1004)
Duchenne muscular dystrophy (DMD)

Phase 3 Phase 3 data due 2H 2020.
$69.2 million

CRIS – Curis Inc.
CA-4948
Relapsed or Refractory Non-Hodgkin Lymphoma

Phase 1 Phase 1 interim data released August 6, 2019. Further update due later in 2019.
$71.7 million

CYCC – Cyclacel Pharmaceuticals Inc.
CYC140
Leukemias

Phase 1 Phase 1 initial data due 2019.
$7.2 million

DARE – Dare Bioscience Inc.
Ovaprene
Contraception

Phase 2 Top-line data due 4Q 2019.
$14.3 million

LPCN – Lipocine Inc.
TLANDO - LPCN 1021
Men with low testosterone (Low T)

PDUFA CRL issued June 2016. Further CRL issued May 9, 2018. PDUFA date November 9, 2019.
$72.5 million

MYOV – Myovant Sciences Ltd.
Relugolix - LIBERTY 1 and LIBERTY 2
Menstrual bleeding associated with uterine fibroids

Phase 3 NDA filing due 4Q 2019.
$535.9 million