BPC October 02 update

Phase 3 catalysts to watch for 4Q; Biotech week in Review

Weekly watchlist

With the final quarter of 2020 upon us, this week’s watch list focuses on key Phase 3 readouts slated for release this quarter. Over the coming weeks we will also highlight earlier stage trials and PDUFA dates scheduled for this quarter.

First, let’s review the week that was with key price moving events.

REGULATORY

Mesoblast Limited (NASDAQ:MESO) shares closed Friday down 35% to $12.03 following news the FDA issued a Complete Response Letter for its Biologics License Application (BLA) for remestemcel-L for the treatment of pediatric steroid-refractory acute graft versus host disease (SR-aGVHD). This was despite an FDA Advisory Committee voting 9-1 in favor that the available data supported the efficacy of the treatment. The FDA has recommended that Mesoblast conduct at least one additional randomized, controlled study to provide further evidence of efficacy.

Iterum Therapeutics plc (Nasdaq: ITRM) announced that it plans to file a New Drug Application (NDA) submission for sulopenem etzadroxil/probenecid for the treatment of uncomplicated urinary tract infections (uUTI) in patients with a quinolone-resistant pathogen by the end of the fourth quarter this year. Shares closed Wednesday up 63% to $1.05.

CTI BioPharma Corp. (Nasdaq: CTIC) announced that following a recent meeting with the FDA it intends to submit a New Drug Application for pacritinib as a treatment for myelofibrosis patients with severe thrombocytopenia based on data from its completed Phase 3 PERSIST-1 and PERSIST-2 trials and the Phase 2 PAC203 dose-ranging trial. A rolling NDA submission is expected to commence within a few weeks, with completion of the NDA submission anticipated in 1Q 2021, well ahead of previous timeline guidance. Shares closed the week up 185% to $2.99.

CLINICAL DATA:

Synthetic Biologics, Inc. (NYSE:SYN) shares closed Friday down 32% to $0.32 following planned interim futility analysis of its Phase 2b trial of SYN-010 in patients with irritable bowel syndrome with constipation (IBS-C). The analysis showed it was unlikely to meet its primary objective and as a result the trial will be discontinued.

Selecta Biosciences, Inc. (NASDAQ:SELB) shares closed the week down 31% to $1.67. The company announced Wednesday that its Phase 2 trial comparing SEL-212 to Krystexxa, for the treatment of chronic refractory gout, did not meet its primary endpoint.

OTHER:

Solid Biosciences Inc. (Nasdaq: SLDB) shares closed the week up 98% to $4.18 following news the FDA has lifted the clinical hold placed on its Phase 1/2 IGNITE DMD (Duchenne muscular dystrophy) clinical trial. Dosing is expected to resume during the first quarter of 2021.

AMAG Pharmaceuticals, Inc. (NASDAQ:AMAG) announced that it will be acquired by Covis Group for $13.75 per share in cash, or approximately $498m on a fully diluted basis and approximately $647m on an enterprise basis. Shares closed Thursday up 45% to $13.65.

Inovio (NASDAQ:INO) shares closed Monday down 28% to $12.14 on news the FDA has placed a partial clinical hold on its Phase 2/3 trial for its COVID-19 vaccine. The company noted the FDA has additional questions about its delivery device to be used in the trial.

Drug Stage Catalyst Market Cap

AMGN – Amgen Inc.
AMG 510
Non-small cell lung cancer (NSCLC)

Phase 2 Phase 2 data released October 5, 2020. Objective response rate (primary end point) consistent with previously reported Phase 1 data. Detailed data at IASLC meeting January 26-29, 2021.
$126.7 billion

APRE – Aprea Therapeutics Inc.
APR-246 (eprenetapopt) and azacitidine
TP53 mutant Myelodysplastic syndromes (MDS)

Phase 2/3 Phase 3 top-line data due by the end of 2020.
$493.2 million

AUPH – Aurinia Pharmaceuticals Inc
Voclosporin ophthalmic solution (VOS) - AUDREY
Dry eye syndrome

Phase 2/3 Phase 2/3 data due 4Q 2020.
$2 billion

AZN – AstraZeneca PLC
AZD1222 (ChAdOx1 nCoV-19)
COVID-19 vaccine

Phase 3 Phase 3 trial ongoing Japan, UK, Brazil, South Africa and India. U.S. trial currently on hold set to resume soon. Initial data 4Q 2020.
$133.8 billion

BCLI – Brainstorm Cell Therapeutics Inc.
NurOwn
Amytrophic lateral sclerosis (ALS)

Phase 3 Phase 3 top-line data due by the end of November 2020.
$318.2 million

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

Phase 3 Phase 3 trial did not meet primary endpoint - October 26, 2020.
$27.3 million

CRTX – Cortexyme Inc.
COR388 - GAIN
Alzheimer’s disease

Phase 2/3 Phase 2/3 interim analysis to occur 4Q 2020. Final data due 4Q 2021.
$1.5 billion

CYTK – Cytokinetics Incorporated
Omecamtiv mecarbil GALACTIC-HF
Acute heart failure

Phase 3 Phase 3 top-line data met primary composite endpoint of reduction in cardiovascular death or heart failure but did not meet secondary endpoint of reduction in cardiovascular death - October 8, 2020. Late breaker at AHA, November 13, 2020
$1.1 billion

MRNA – Moderna Inc.
mRNA-1273
COVID-19 vaccine

Phase 2/3 Phase 2 and 3 trials ongoing. Phase 3 completion of enrollment announced October 22, 2020. Interim data due November 2020.
$25.9 billion

PFE – Pfizer Inc.
BNT162b2
COVID-19 vaccine

Phase 3 Phase 3 trial ongoing. Data Monitoring Committee has not conducted any interim efficacy analysis as of October 27, 2020. Safety milestone required for FDA EUA estimated around the third week of November, 2020.
$197 billion

PLX – Protalix BioTherapeutics Inc. (DE)
Pegunigalsidase alfa (PRX-102) - BRIGHT
Fabry disease

Phase 3 Phase 3 final data due 4Q 2020.
$118.3 million

QURE – uniQure N.V.
AMT-061 (HOPE-B)
Hemophilia B

Phase 3 Phase 3 top-line data due by the end of 2020.
$1.7 billion

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
Alstrom Syndrome and Bardet-Biedl Syndrome

Phase 3 Phase 3 top-line data due 4Q 2020 or early 1Q 2021.
$982.1 million

TGTX – TG Therapeutics Inc.
TG-1101 - ULTIMATE I
Multiple Sclerosis

Phase 3 Phase 3 data due 2H 2020.
$3.3 billion