BPC January 31 update

Key biotech stock earnings dates and data presentations for the week ahead; Biotech week in review

Weekly watchlist

 The main focus for next week will be the second week of 4Q earnings. Noted below is a selection of companies set to report together with companies scheduled to present at the WORLDSymposium and EAHAD meetings. First, let’s review the week’s major news.

Evolus, Inc. (NASDAQ: EOLS) announced Friday the FDA approved Jeuveau for glabellar lines. Launch is slated for spring, with plans to price the treatment at a discount to Botox. Shares closed the after-hours session at $21.35, up 31% from Thursday’s close.

Alkermes plc (Nasdaq: ALKS) announced Friday it received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for ALKS 5461 for the adjunctive treatment of major depressive disorder (MDD).

CTI BioPharma Corp. (NASDAQ:CTIC) shares closed Friday down 16% to $1.00 following news it will withdraw its European Marketing Authorization Application (MAA) for pacritinib as a treatment for myelofibrosis.

Fortress Biotech, Inc. (NASDAQ: FBIO) shares closed the week up 133% to $2.47. The company announced that Caelum Biosciences, founded by Fortress, signed an agreement with Alexion Pharmaceuticals, Inc (NASDAQ: ALXN) to advance the development of CAEL-101 for light chain (AL) amyloidosis. Alexion will make a payment of $60m.

Eyenovia, Inc. (NASDAQ: EYEN) shares closed Wednesday up 25% to $3.29 following news it met the primary endpoint in its Phase 3 MIST-1 trial of MicroStat for the treatment of mydriasis (dilation of the pupil).

Voyager Therapeutics, Inc. (NASDAQ: VYGR) shares closed the week up 25% to $10.55 following a collaboration with Neurocrine Biosciences, Inc. (NASDAQ: NBIX) for its gene therapy programs. Voyager will receive $165m in cash including a $115m upfront payment and a $50m equity investment.

Trevena, Inc. (NASDAQ: TRVN) shares closed the week up 87% to $1.01. The company announced that the FDA will agree on labeling to a maximum daily dose of 27 mg for oliceridine for the treatment of pain relief. The meeting follows the issuance of a Complete Response Letter (CRL) last year.

MeiraGTx Holdings plc (NASDAQ:MGTX) shares closed Thursday up 30% to $12.38 on news of a collaboration with Janssen Pharmaceuticals, Inc. to develop gene therapies for the treatment of inherited retinal diseases (IRDs). MeiraGTx will receive $100m cash upfront.

AVEO Oncology (NASDAQ: AVEO) shares slumped to close Thursday down 60% to $0.70 on news the FDA has recommended the company not to submit its New Drug Application (NDA) for tivozanib with the preliminary overall survival (OS) results from the Phase 3 TIVO-3 trial. The company now plans to make a NDA filing decision following the availability of more mature OS results. Further interim OS data are due 4Q 2019.

AC Immune SA (NASDAQ:ACIU) shares slid Wednesday to close down 66% to $3.65 on news Roche (RHHBY) is discontinuing the CREAD 1 and CREAD 2 Phase 3 trials of crenezumab, for the treatment of Alzheimer’s disease (AD), following interim analysis conducted by the Independent Data Monitoring Committee (IDMC).

Aquestive Therapeutics, Inc. (NASDAQ: AQST) announced its licensee, Sunovion Pharmaceuticals Inc. received a CRL from the FDA for the New Drug Application (NDA) for apomorphine sublingual film for the treatment of OFF episodes experienced by people with Parkinson's disease (PD). Shares closed Thursday down 14% to $6.13.

Selection companies reporting 4Q earnings:

Mon: ALXN GILD

Tue: NBIX VRTX

Wed: GSK LLY REGN

Thur: SGEN

Fri: IMGN

Drug Stage Catalyst Market Cap

ABEO – Abeona Therapeutics Inc.
ABO-102
Sanfilippo syndrome type A (MPS IIIA)

Phase 1/2 Phase 1/2 safety and tolerability data to be presented at WORLDSymposium February 6, 2019.
$207.5 million

AVRO – AVROBIO Inc.
AVR-RD-01 (FAB-201)
Fabry disease

Phase 1/2 Phase 2 additional preliminary data due summer 2019.
$396.5 million

CBIO – Catalyst Biosciences Inc.
Marzeptacog alfa
Hemophilia

Phase 2 Phase 2 final data released July 7, 2019. Primary endpoint met.
$100.4 million

FOLD – Amicus Therapeutics Inc.
ATB200
Pompe Disease

Phase 1/2 Further Phase 1/2 preliminary positive data released February 5, 2019.
$3.2 billion

JNJ – Johnson & Johnson
Esketamine
Treatment resistant depression

Approved FDA approval announced March 5, 2019.
$357.7 billion

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

Phase 3 Phase 3 completion of enrolment announced June 17, 2019.
$60.7 million

QURE – uniQure N.V.
AMT-061
Hemophilia B

Phase 2b Phase 2b nine month data presented at ISTH July 5, 2019. FIX activity up to 54% of normal, with mean of 45% of normal.
$2.6 billion

SGMO – Sangamo Therapeutics Inc.
SB-913 - CHAMPIONS
MPS Type 2

Phase 1/2 Phase 1/2 updates during 2019.
$1.4 billion

SGMO – Sangamo Therapeutics Inc.
SB-318
MPS Type 1

Phase 1/2 Phase 1/2 longer-term safety and biochemical data due in 2019.
$1.4 billion

SNNA – Sienna Biopharmaceuticals Inc.
SNA-001
Reduction of light-pigmented hair

Phase 3 Phase 3 data met non-inferiority primary endpoint - February 8, 2019. Seeking partner for further advancement.
$24.3 million