BPC March 05 update

FDA commissioner to leave agency; Sarepta SRPT and CymaBay CBAY offerings; Celgene CELG myelofibrosis PDUFA September 3

Price and Volume Movers

Celgene Corporation (NASDAQ:CELG) announced the FDA has accepted its New Drug Application (NDA) for fedratinib for the treatment of patients with myelofibrosis and granted a priority review. The PDUFA date is September 3, 2019.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced that it is offering $350m in shares of its common stock in an underwritten public offering. Shares are trading down 2.8% after hours to $144.63 after closing normal hours up 2.3%.

CymaBay Therapeutics, Inc. (Nasdaq: CBAY) also announced that it has commenced an underwritten public offering of its common stock. Shares are trading down 5% to $12.60 after hours.

Exact Sciences Corporation (NASDAQ: EXAS) announced an underwritten public offering of $600m of convertible senior notes due 2027.

Pacira Pharmaceuticals, Inc. (NASDAQ: PCRX) will acquire privately-held MyoScience, Inc, a medical technology company that currently markets a non-opioid treatment that alleviates pain through a mechanism known as cryoanalgesia. Pacira will make an initial payment of $120m, with MyoScience shareholders eligible to receive up to an additional $100m in milestones payments. Pacira also intends to change its corporate name to Pacira BioSciences, Inc. but will continue to trade under the symbol “PCRX.” Shares closed down 4% to $39.08.

In non-company specific news, Dr. Scott Gottlieb, commissioner of the FDA is leaving the agency next month. He has become well known for his efforts to regulate e-cigarettes and his tenure at the FDA came at a time of record drug approvals.

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Major price movers (stocks priced > $1.00, volume > 50k):

ADVANCERS:

Intrexon Corporation (XON): $5.02; +21%.

Anavex Life Sciences Corp. (AVXL): $2.95; +14%.

Diffusion Pharmaceuticals Inc. (DFFN): $3.80; +11%.

La Jolla Pharmaceutical Company (LJPC): $7.00; +11%.

Chiasma, Inc. (CHMA): $5.25; +11%.

DECLINERS:

Adamas Pharmaceuticals, Inc. (ADMS): $8.16; -33%.

22nd Century Group, Inc. (XXII): $1.72; -27%.

Cerecor Inc. (CERC): $5.45; -20%.

Chimerix, Inc. (CMRX): $1.93; -16%.

Bio-Path Holdings, Inc. (BPTH): $4.62; -16%.


Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ARPO – Aerpio Pharmaceuticals Inc.
AKB-9778 - TIME 2B
Non-Proliferative Diabetic Retinopathy

Phase 2 Phase 2 data released March 18, 2019 did not meet primary endpoint.
$44.6 million

ARPO – Aerpio Pharmaceuticals Inc.
AKB-9778
Open angle glaucoma

Phase 1b Phase 1b trial to be initiated 2Q 2019.
$44.6 million

CELG – Celgene Corporation
Fedratinib
Myelofibrosis

PDUFA priority review PDUFA date under priority review September 3, 2019.
$61.8 billion

CMRX – Chimerix Inc.
Oral Brincidofovir
Smallpox

Pivotal mouse trial data 2019.
$102.1 million

CMRX – Chimerix Inc.
IV Brincidofovir
Adult allo-HCT recipients with Adenovirus

Phase 2 Phase 2 update due mid-2019.
$102.1 million

CMRX – Chimerix Inc.
Oral Brincidofovir - AdAPT Study
Allogeneic hematopoietic stem cell transplant (HCT) recipients with adenovirus (AdV)

Phase 2 Phase 2 enrolment has been delayed - noted March 5, 2019. Enrolment update due mid-2019.
$102.1 million

DOVA – Dova Pharmaceuticals Inc.
Avatrombopag
Thrombocytopenia

Phase 3 Phase 3 enrolment has been discontinued to focus on other development - noted March 5, 2019.
$239.9 million

FATE – Fate Therapeutics Inc.
FT500
Soild tumors

Phase 1 Phase 1 dosing commenced February 2019.
$1 billion

KURA – Kura Oncology Inc.
Tipifarnib
Head and neck squamous cell carcinomas (HNSCC)

Phase 2 Phase 2 data due 2H 2019.
$580.4 million

KURA – Kura Oncology Inc.
KO-539
Relapsed or refractory acute myeloid leukemia (AML)

Phase 1 Phase 1 trial to be initiated 2Q 2019.
$580.4 million

KURA – Kura Oncology Inc.
Tipifarnib
Relapsed or refractory peripheral T-cell lymphoma

Phase 2 Phase 2 additional data due mid-2019.
$580.4 million

KURA – Kura Oncology Inc.
KO-947
Solid tumors

Phase 1 Phase 1 data due 2019.
$580.4 million

KURA – Kura Oncology Inc.
Tipifarnib
Chronic myelomonocytic leukemia (CMML) - cancer

Phase 2 Phase 2 data due 2019.
$580.4 million

LLY – Eli Lilly and Company
Galcanezumab
Cluster headache

PDUFA priority review PDUFA date under priority review for sBLA announced March 5, 2019. No PDUFA date given - estimate July 2019.
$134.3 billion

OBSV – ObsEva SA
Nolasiban (OBE001) - IMPLANT 4
Improving IVF outcomes

Phase 3 Phase 3 data due 4Q 2019.
$550.9 million

OBSV – ObsEva SA
OBE2109
Endometriosis

Phase 2b Phase 2b 48 and 52-week data due 1H 2019.
$550.9 million

OBSV – ObsEva SA
OBE2109 - PRIMROSE
Uterine fibroids

Phase 3 Phase 3 data due early 2020.
$550.9 million

OBSV – ObsEva SA
OBE2109 - PRIMROSE 2
Uterine fibroids

Phase 3 Phase 3 data due 4Q 2019.
$550.9 million

OBSV – ObsEva SA
OBE022 - PROLONG
Pre-term labor

Phase 2a Phase 2a Part B interim analysis due 1H 2019.
$550.9 million

OBSV – ObsEva SA
OBE001 - IMPLANT2
IVF

Phase 3 Phase 3 data released February 26,2018 - primary endpoint met. Improvement shown in ET D5 subgroup but not in ET D3. Further data due 2Q 2019.
$550.9 million

RDUS – Radius Health Inc.
AB122
Solid tumors

Phase 1 Phase 1 dose-expansion cohort to be initiated mid-2019.
$840.4 million

RTTR – Ritter Pharmaceuticals Inc.
RP-G28
Lactose intolerance

Phase 3 Phase 3 data due early 4Q 2019.
$5.5 million

ZIOP – ZIOPHARM Oncology Inc
Ad-RTS-hIL-12 plus veledimex in combination with OPDIVO (nivolumab)
Refractory glioblastoma multiforme (GBM)

Phase 1 Phase 1 initial data due 2019.
$630.5 million

ZIOP – ZIOPHARM Oncology Inc
Ad-RTS-hIL-12 plus veledimex
recurrent glioblastoma (rGBM)

Phase 1 Phase 1 initial data due 2019.
$630.5 million

ZIOP – ZIOPHARM Oncology Inc
Sleeping beauty - TCR-T cell therapy
Solid tumors

Phase 1 Phase 1 trial to begin dosing mid-2019.
$630.5 million

ZIOP – ZIOPHARM Oncology Inc
Ad-RTS-hIL-12 plus veledimex plus Libtayo
Refractory glioblastoma multiforme (GBM)

Phase 2 Phase 2 trial to commence 2Q 2019.
$630.5 million