BPC November 17 update

Upcoming key biotech stock catalysts due by year end; Biotech week in review

Weekly watchlist

As 3Q earnings season for most biotech companies comes to an end, a number of clinical readouts and license deals made headlines in a week where key biotech ETFs either set or came close to new 52-week lows.

Arena Pharmaceuticals, Inc. (Nasdaq: ARNA) shares closed Thursday up 22% to $39.55 following news that it will license out worldwide rights for ralinepag, currently in Phase 3 development for pulmonary arterial hypertension (PAH), to United Therapeutics Corporation. Arena will receive $800m upfront and potential milestone payments totaling up to $400m and low double-digit tiered royalties on annual net sales of ralinepag.

uniQure N.V. (NASDAQ: QURE) shares surged Thursday to close up 36% to $30.93 following the release of promising initial clinical data in its Phase 2b trial of AMT-061 for the treatment of patients with severe and moderately severe hemophilia B. The data showed mean levels of Factor IX (FIX) activity in three patients of 31%, exceeding threshold FIX levels generally considered sufficient to significantly reduce the risk of bleeding events. The company expects to initiate the dosing phase of its Phase 3 HOPE-B pivotal study in 1Q 2019.

Eidos Therapeutics, Inc. (Nasdaq:EIDX) shares closed Monday up 15% to $14.60 following the presentation of promising data from its Phase 2 trial evaluating AG10 in patients with symptomatic transthyretin (TTR) amyloidosis cardiomyopathy (ATTR-CM). AG10 was well tolerated and demonstrated >90% TTR average stabilization at day 28. Phase 3 pivotal trials are planned for 1H 2019. 

Mesoblast Limited (Nasdaq:MESO) shares closed Monday down 28% to $5.48 following news that its Phase 2 trial of MPC-150- IM in end-stage heart failure patients implanted with a left ventricular assist device (LVAD) did not its primary endpoint.

Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) shares slid 45% Monday to $1.31 on the back of updated Phase 1b data at the Society for Immunotherapy of Cancer (SITC) meeting of IPI-549 in combination with Opdivo. The data showed two out of 44 patients with solid tumors exhibited a partial response.

AMAG Pharmaceuticals, Inc. (AMAG) and Palatin Technologies, Inc. (PTN) announced that potential approval of Vyleesi, for the treatment of Female sexual dysfunction (FSD)is likely to be delayed by three to six months following a request from the FDA for additional data from a Phase 1 trial. Officially, the PDUFA date stands at March 23, 2019 but will be extended closer to the date. The FDA will assess the need for an Advisory Committee meeting after receipt of the requested data. 

Tesaro, Inc. (NASDAQ: TSRO) shares closed Friday up 31% to $34.96 following a report by Bloomberg that the company is exploring a possible sale of the company.

Over the past two weeks we have noted upcoming Phase 3 catalysts for micro and small-cap companies slated for this quarter. Companies have since noted in their recent earnings releases updated timelines, with a number of catalysts now pushed out to early 2019. In light of this, an updated list of mainly Phase 3 catalysts together with other key regulatory dates is noted below. 

Drug Stage Catalyst Market Cap

ACHN – Achillion Pharmaceuticals Inc.
ACH-4471
Paroxysmal nocturnal hemoglobinuria (PNH)

Phase 2 Phase 2 final data presented at EHA June 15, 2019.
$342.7 million

ALKS – Alkermes plc
ALKS 3831 - ENLIGHTEN-2
Schizophrenia

NDA Filing Phase 3 data released November 29, 2018 met co-primary endpoints. NDA filing due mid-2019.
$3.7 billion

CELG – Celgene Corporation
REVLIMID - ROBUST
First-line ABC Diffuse large B-cell lymphoma (DLBCL)

Phase 3 Phase 3 data released April 25, 2019 did not meet primary endpoint.
$64.7 billion

CFRX – ContraFect Corporation
CF-301
Serious infections caused byStaph aureus including MRSA

Phase 2 Phase 2 trial noted clinically meaningful data but not statistically significant. Phase 3 trial planned.
$38.6 million

CPRX – Catalyst Pharmaceuticals Inc.
Firdapse
Lambert-Easton Myasthenic Syndrome (LEMS)

Approved FDA approval announced November 28, 2018.
$419.6 million

EVFM – Evofem Biosciences Inc.
Amphora - AMPOWER
contraceptive vaginal gel

NDA Filing Phase 3 data released December 17, 2018. Primary endpoint met. NDA to be refiled 2H 2019.
$218.6 million

FGEN – FibroGen Inc
Roxadustat
Anaemia in Chronic Kidney Disease

NDA Filing Phase 3 MACE safety data released May 9, 2019. Non-inferior in non-dialysis, no worse risk than placebo. NDA filing due September/October 2019.
$4 billion

MRNS – Marinus Pharmaceuticals Inc.
Ganaxolone - Magnolia
Postpartum depression

Phase 2 Data due first half of 3Q 2019.
$233.9 million

OCUL – Ocular Therapeutix Inc.
DEXTENZA
Ocular inflammation and pain following cataract surgery

Approved FDA approval announced December 3, 2018.
$227 million

RDHL – Redhill Biopharma Ltd.
RHB-105
H. pylori

PDUFA priority review PDUFA date under priority review November 2, 2019.
$223.5 million

SESN – Sesen Bio Inc.
Vicinium
Non-muscle invasive bladder cancer (NMIBC)

BLA Filing Rolling BLA filing to commence 4Q 2019.
$98.5 million

SUPN – Supernus Pharmaceuticals Inc.
SPN-812
ADHD

NDA Filing NDA filing due 2H 2019.
$1.7 billion

VRTX – Vertex Pharmaceuticals Incorporated
VX-659 in combination with tezacaftor and ivacaftor
Cystic fibrosis (CF) who have one F508del mutation and one minimal function mutation (F508del/Min)

Phase 3 Phase 3 positive data released November 27, 2018. 14% improvement in ppFEV1 cf placebo.
$45.2 billion