BPC November 02 update

Small cap biotech stocks with key Phase 3 readouts due this quarter; Biotech week in Review

Weekly watchlist

The last week saw significant newsflow hit trader screens with a combination of abstracts for upcoming ASH and SITC meetings, 3Q earnings releases and several important regulatory decisions. We review key events below while also looking ahead to small-cap biotech stocks with Phase 3 catalysts slated for this quarter.

Sage Therapeutics (NASDAQ: SAGE) shares closed up 8% to $148.50 after hours Friday following news an FDA Advisory Committee voted 17-1 in support of approval of Zulresso (brexanolone) injection for the treatment of postpartum depression (PPD). The PDUFA date, under priority review, for a final decision by the FDA is December 19, 2018.

AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX) announced the approval of Dsuvia by the FDA for the management of acute pain in adults that is severe enough to require an opioid analgesic. Shares closed Friday up 16% to $4.80. 

Trevena, Inc. (NASDAQ: TRVN) shares slid to close Friday down 32% to $0.71 following its announcement the FDA issued a Complete Response Letter (CRL) regarding the New Drug Application (NDA) for oliceridine.

Coherus BioSciences, Inc. (NASDAQ: CHRS) shares closed Friday up 6% to $12.30 following news the FDA approved Udenyca, a pegfilgrastim biosimilar for patients with cancer receiving myelosuppressive chemotherapy.  

Alkermes plc (Nasdaq: ALKS) shares closed Friday down 8% to $37.74 following a negative vote (2-21) by an FDA Advisory Committee against recommending approval for the New Drug Application (NDA) for ALKS 5461 for the adjunctive treatment of major depressive disorder (MDD). 

argenx (Nasdaq: ARGX) shares closed Thursday up 20% to $96.05 following the release of updated data from its Phase 1/2 clinical trial of cusatuzumab (ARGX-110) in acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS). The data showed a 92% (11/12 patients) overall response rate (ORR) including 9 patients (82%) with a complete response. Further updated data are due at the ASH meeting in early December. 

Strongbridge Biopharma plc (Nasdaq: SBBP) shares surged to close Wednesday up 58% to $6.40 following news it will sell rights to Macrilen (macimorelin), for the diagnosis of adult growth hormone deficiency (AGHD), to Novo Nordisk for an upfront payment of $145m, compared with the $24m it paid Aeterna Zentaris Inc. (Nasdaq: AEZS) earlier in January this year. 

On the earnings front, two companies to take a battering were Puma Biotechnology, Inc. (PBYI) $20.07; -48% and Clovis Oncology, Inc. (CLVS) $11.63; -30% (Thur).

Small cap biotech stocks with key Phase 3 readouts this quarter:

Drug Stage Catalyst Market Cap

AIMT – Aimmune Therapeutics Inc.
Peanut Allergy

Phase 3 Phase 3 data released November 8, 2018 noted no serious adverse events
$1.3 billion

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

Phase 3 Noted January 31, 2019 that FDA recommended against filing an NDA with current OS data. Additional interim OS analysis due August 2019 with further OS data 4Q 2019.
$140.4 million

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
Zydis ODT formulation of rimegepant

Phase 3 Phase 3 data to be presented at AAN May 7, 2019 with abstract embargoed until May 3.
$2.7 billion

CLSD – Clearside Biomedical Inc.
Suprachoroidal CLS-TA - SAPPHIRE
Macular edema associated with retinal vein occlusion (RVO)

Phase 3 Phase 3 data released November 5, 2018 did not meet primary endpoint.
$44.7 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 2Q 2019.
$144.7 million

FOMX – Foamix Pharmaceuticals Ltd.
Papulopustular rosacea

Phase 3 Phase 3 top-line data released November 7, 2018. Primary endpoints met. Safety data reported February 27, 2019.
$183.2 million

ITCI – Intra-Cellular Therapies Inc.
ITI-007 - Monotherapy (Study 401)
Bipolar depression

Phase 3 Phase 3 top-line data due 2Q 2019.
$675.7 million

MDWD – MediWound Ltd.
Severe burns

Phase 3 BLA filing due 2H 2019.
$131.5 million

RDHL – Redhill Biopharma Ltd.
H. pylori

NDA Filing Phase 3 trial met primary endpoint. NDA filing 1H 2019.
$229.8 million

SNNA – Sienna Biopharmaceuticals Inc.
Reduction of light-pigmented hair

Phase 3 Phase 3 data met non-inferiority primary endpoint - February 8, 2019. Seeking partner for further advancement.
$60.8 million

URGN – UroGen Pharma Ltd.
MitoGel - UGN-101
Urothelial Carcinoma

Phase 3 Phase 3 data released January 8, 2019 noted 57% CR. Initiation of rolling NDA announced December 17, 2018. Presentation of data May 5, 2019 at AUA.
$748.6 million

VNDA – Vanda Pharmaceuticals Inc.
HETLIOZ (tasimelteon)
Smith-Magenis Syndrome

sNDA Filing Phase 3 data released December 10, 2018 - one of two primary endpoints met. sNDA filing due 3Q 2019.
$876.5 million