BPC August 31 update

Biotech catalysts for September; Biotech Week in Review

Weekly watchlist

The end of summer brought a quiet trading week of newsflow, with just a few price-moving events to highlight from the week that was. We also update our catalyst list for September.

The Medicines Company (NASDAQ: MDCO) shares closed the week up 20% to $41.96 following news that its first pivotal Phase 3 clinical trial, ORION-11, evaluating inclisiran in its treatment of cholesterol, met all primary and secondary endpoints. Detailed data will be presented at a late-breaking science session at the European Society of Cardiology’s ESC Congress on Monday, September 2 at 8:30 am CET / 2:30 am EDT.

Prothena Corporation plc (NASDAQ:PRTA) shares closed the week up 22% to $8.41, following the filing of a Schedule 13D by EcoR1 Capital on Monday, where it reported a $23.6% ownership stake of the company.

Amgen (NASDAQ:AMGN) announced that it has entered into an agreement with Celgene Corporation (NASDAQ:CELG) in connection with its pending merger with Bristol-Myers Squibb Company (NYSE:BMY) to acquire worldwide rights to Otezla, a treatment for psoriasis and psoriatic arthritis for $13.4b in cash. Amgen shares closed the week up 5% to $208.62.

Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN), which had been subject to rumors last week that Amgen may be interested in a buyout of the company, lost further ground on news the U.S. Patent and Trademark Office (PTO) will review patents of its blood disorder treatment, Soliris, following a challenge from Amgen. Shares closed the week down 13% to $100.74.


Biotech stock events for September:

Drug Stage Catalyst Market Cap

ADVM – Adverum Biotechnologies Inc.
ADVM-022
Wet age-related macular degeneration (Wet-AMD)

Phase 1 Phase 1 third cohort to commence enrolment in 4Q 2019. Cohort 1 52-week data and Cohort 2 24-week data due 1H 2020.
$563.9 million

AIMT – Aimmune Therapeutics Inc.
AR101
Peanut Allergy

PDUFA PDUFA date late-January 2020. Advisory Committee meeting September 13, 2019 voted in favor of supporting approval.
$1.8 billion

AMGN – Amgen Inc.
AMG 510
Solid tumors

Phase 1 Phase 1 update at ESMO noted 54% PR rate for NSCLC patients (960mg); 1/12 for CRC patients. Next update due in 2020.
$131.5 billion

ANAB – AnaptysBio Inc.
ANB019
Generalized pustular psoriasis

Phase 2 Phase 2 interim data on first TWO patients announced September 30, 2019 with patients reaching primary endpoint. Further data due 2020.
$289.4 million

AVEO – AVEO Pharmaceuticals Inc.
TIVO-3 - tivozanib
Third line treatment of patients with renal cell cancer

NDA Filing NDA to be filed in 1Q 2020. Will withdraw if overall survival data in 2Q 2020 yields an OS HR above 1.00.
$90.1 million

AZRX – AzurRx BioPharma Inc.
MS1819-SD
Cystic fibrosis

Phase 2 Phase 2 higher dose trial to be initiated early 2020 with data due mid-2020.
$17 million

ENTA – Enanta Pharmaceuticals Inc.
EDP-305 ARGON-1
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2 data met primary endpoint - September 25, 2019. Pruritus was present in approximately 51% of the subjects in the 2.5mg arm. Phase 2b trial to be initiated 1H 2020.
$1.2 billion

GTHX – G1 Therapeutics Inc.
G1T48
ER+, HER2- breast cancer

Phase 1/2 Phase 1/2 preliminary data ESMO 2019 noted 1/19 PRs. Clinical benefit rate 3/19. Phase 3 planned for 2020.
$862.3 million

LLY – Eli Lilly and Company
LOXO-292
RET-fusion non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC) and other tumors

NDA Filing NDA filing due YE 2019.
$108.7 billion

MRNS – Marinus Pharmaceuticals Inc.
Ganaxolone
Refractory status epilepticus (RSE)

Phase 2 Phase 3 trial to be initiated mid-2020.
$60.9 million

NERV – Minerva Neurosciences Inc
MIN-202 (seltorexant) vs quetiapine
Major Depressive Disorder

Phase 2 Phase 2b data noted quantitative advantage but no statistical separation between two arms. Company noted trial was not designed to detect statistical significance.
$202.5 million

NTRP – Neurotrope Inc.
Bryostatin
Alzheimer's disease

Phase 2 Phase 2 trial did not meet primary endpoint - September 9, 2019.
$9.2 million

URGN – UroGen Pharma Ltd.
VesiGel UGN-102
Low grade non-muscle invasive bladder cancer (LG-NMIBC)

Phase 2b Phase 2b initial data noted 20/32 patients recorded a complete response. Phase 3 trial planned for 2020.
$574.9 million

XERS – Xeris Pharmaceuticals Inc.
Glucagon Rescue Pen
Severe hypoglycemia

Approved FDA Approval announced September 10, 2019.
$221.1 million