BPC September 13 update

Biotech week in Review; Biotech stock events left for September

Weekly watchlist

With two weeks remaining in the third quarter, while much attention will be on the end-of-month ESMO conference discussed last week, several other biotech stock catalysts remain on the calendar scheduled for release in September. We note these below but first let’s review the last week which saw a significant number or price-moving events.

Aimmune Therapeutics, Inc. (Nasdaq:AIMT) announced that the FDA Allergenic Products Advisory Committee (APAC) voted 7 to 2 that the efficacy data and 8 to 1 that the safety data, are adequate to support the use of AR101 in children and teens with peanut allergy. The PDUFA date for a final decision by the FDA is in late-January 2020. Shares closed the after-hours session at $28.80, a gain of 40% for the week.

Mallinckrodt Plc (NYSE: MNK) announced it will sell its contract development and manufacturing organization (CDMO), to BioVectra Inc, for approximately $250m, likely putting to rest earlier reports by Bloomberg that the company had hired a law firm to explore restructuring and potential bankruptcy. Shares closed the week up 76% to $3.30.

Lexicon Pharmaceuticals, Inc. (NASDAQ:LXRX) shares closed the week up 70% to $2.34 following the announcement of the termination of its alliance with Sanofi for the development and commercialization of Zynquista (sotagliflozin). Lexicon will regain all rights to Zynquista and will receive from Sanofi $260m, of which $208m is payable upfront and the remainder is payable within twelve months.

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) announced its Phase 3 trial evaluating pimavanserin for the treatment of dementia-related psychosis, met its primary endpoint, demonstrating a highly statistically significant longer time to relapse of psychosis with pimavanserin compared to placebo. Shares closed the week up 70% to $40.45.

Neurotrope, Inc. (NASDAQ: NTRP) shares slumped to close the week down 80% to $0.88 following news its Phase 2 trial of Bryostatin-1 in moderate to severe Alzheimer’s Disease did not meet the primary endpoint.

Fibrocell Science, Inc. (NASDAQ:FCSC) shares closed Friday up 60% to $2.92 on news that it will be acquired by Castle Creek Pharmaceutical Holdings, Inc. for $63.3m. Fibrocell stockholders will receive $3 per share.

Tocagen Inc. (Nasdaq: TOCA) shares slid to close down Thursday 78% to $0.93 on news that its Toca 5 Phase 3 trial evaluating Toca 511 & Toca FC in patients with recurrent high grade glioma (HGG) missed both primary and secondary endpoints.

Adverum Biotechnologies, Inc. (NASDAQ: ADVM) shares closed down Thursday 50% to $5.96 following the release of initial Phase 1 data from the first cohort of six patients treated with ADVM-022 with wet age-related macular degeneration (wet AMD). The best corrected visual acuity (BCVA), which measures how many more letters patients can see on an eye chart, ranged from -9.1 to +5.1 letters, or a mean change of -2 letters.

Ritter Pharmaceuticals, Inc. (Nasdaq: RTTR) announced Thursday that its Phase 3 clinical trial of RP-G28 for lactose intolerance (LI) failed to demonstrate statistical significance in its pre-specified primary endpoint. Shares closed Friday down 70% to $0.32.

Non-ESMO biotech stock events to watch left for 3Q 2019:

Drug Stage Catalyst Market Cap

ALNY – Alnylam Pharmaceuticals Inc.
Inclisiran - ORION-9

Phase 3 Phase 3 data met all endpoints - September 25, 2019. Data November 18, 2019 noted LDL-C reductions of 50%.
$12.4 billion

ANAB – AnaptysBio Inc.
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.
$383.6 million

ENTA – Enanta Pharmaceuticals Inc.
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2b trial initiation has been delayed due to COVID-19 - March 26, 2020.
$1 billion

MRNS – Marinus Pharmaceuticals Inc.
Ganaxolone - Marigold
CDKL5 Deficiency Disorder (CDD)

Phase 3 Phase 3 top-line data due 3Q 2020.
$141.4 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.
$215.7 million

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

Phase 2b Phase 3 trial planned for 2H 2020.
$420.6 million

ZYNE – Zynerba Pharmaceuticals Inc.
Developmental and Epileptic Encephalopathies (DEE)

Phase 2 Phase 2 top-line data released September 18, 2019. 10/46 patients reported a serious adverse event (SAE). Intends to meet with FDA 2Q 2020 to discuss path forward.
$81.7 million