BPC July 10 update

Phase 3 small/mid-cap readouts to watch slated for the third quarter; Biotech week in Review

Weekly watchlist

Last week we previewed events to watch due either in July or mid-2020. This week, our focus turns towards the quarter as a whole, as we look ahead at key Phase 3 clinical trial readouts for small and mid-cap companies slated for release this quarter. Let us first review the week that was with notable biotech stock news.


Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) announced the initiation of trials evaluating REGN-COV2, its antibody cocktail for the treatment and prevention of COVID-19. A Phase 3 trial will evaluate the ability to prevent infection among uninfected people who have had close exposure to a COVID-19 patient. Additionally, Phase 2/3 trials in hospitalized and non-hospitalized patients have also been initiated with initial data due this summer.

Novavax, Inc. (Nasdaq: NVAX) shares rallied to close the week up 16% to $94.34 following news it has been selected to participate in Operation Warp Speed (OWS), a U.S. government program to deliver vaccines for COVID-19. Novavax has been awarded $1.6 billion to complete development and deliver 100m doses of its vaccine candidate.


Immunomedics, Inc. (NASDAQ: IMMU) shares closed the week up 10% to $41.32 following the release of Phase 3 data of Trodelvy (sacituzumab govitecan) in patients with Triple-Negative Breast Cancer. The trial met its primary endpoint demonstrating a statistically significant improvement in progression free survival (PFS) compared to chemotherapy, with a hazard ratio of 0.41 and median PFS of 5.6 months compared to 1.7 months for chemo.

Otonomy, Inc. (Nasdaq: OTIC) announced top-line data from its Phase 1/2 clinical trial of OTO-313 in patients with persistent tinnitus exhibited responder rates of 43% at both Day 29 and Day 57 compared to 13% of placebo patients. Shares closed the week up 33% to $4.20.

Bellus Health Inc. (Nasdaq: BLU) shares slumped to close Monday down 72% to $3.40 on news top-line data from its Phase 2 trial of BLU-5937 in patients with refractory chronic cough failed to meet the primary endpoint.

ObsEva SA (NASDAQ: OBSV) announced its Phase 3 PRIMROSE 1 trial of linzagolix in women with heavy menstrual bleeding due to uterine fibroids, met the primary endpoint, showing a statistically significant reduction in menstrual blood loss compared to placebo. However, shares fell to close the week down 57% to $2.66 on data which noted lower placebo adjusted responder rates than in previous trials by competitors Abbvie and Myovant.


Biogen (Nasdaq: BIIB) announced that it has completed its Biologics License Application (BLA) filing for aducanumab for the treatment of Alzheimer’s disease. Biogen has requested Priority Review for the application. Shares closed Wednesday up 4% to $280.19.

Osmotica Pharmaceuticals (NASDAQ:OSMT) shares closed the week up 36% to $8.77. It announced Thursday that the FDA approved Upneeq, for the treatment of acquired blepharoptosis.


Intersect ENT, Inc. (NASDAQ:XENT) shares closed Wednesday up 29% to $17.98 reports by Bloomberg that medical device company Medtronic (NYSE:MDT) has made an offer to acquire Intersect.

Unum Therapeutics Inc. (NASDAQ:UMRX) announced the acquisition of Kiq LLC, a biotech focused on precision kinase inhibitors. Unum also entered into a private placement for proceeds of $104.4m. Shares soared to close the week up 655% to $3.32.

Phase 3 small/mid-cap readouts to watch slated for the third quarter:

Drug Price Stage Catalyst Market Cap

ACAD – ACADIA Pharmaceuticals Inc.
NUPLAZID (pimavanserin) CLARITY-2 and CLARITY-3
Adjunctive Treatment in Patients With Major Depressive Disorder

-0.13  -1%
Phase 3 Phase 3 trial did not meet primary endpoint - July 20, 2020.
$4 billion

ALBO – Albireo Pharma Inc.
BYLVAY (odevixibat)
Progressive familial intrahepatic cholestasis (PFIC)

-0.61  -2%
PDUFA priority review PDUFA date under priority review July 20, 2021.
$666.8 million

BTAI – BioXcel Therapeutics Inc.
Dexmedetomidine (BXCL501)
Agitation associated with schizophrenia and bipolar disorders

-1.39  -4%
PDUFA PDUFA date January 5, 2022.
$907.7 million

CPRX – Catalyst Pharmaceuticals Inc.
FIRDAPSE (amifampridine)
MuSK-antibody positive myasthenia gravis (MuSK-MG)

-0.03  -1%
Phase 3 Phase 3 trial did not meet primary endpoint - August 10, 2020.
$596.4 million

CRBP – Corbus Pharmaceuticals Holdings Inc.
Resunab - RESOLVE-1
Systemic Sclerosis

-0.04  -2%
Phase 3 Phase 3 top-line data did not meet endpoints - September 8, 2020.
$242.6 million

ITCI – Intra-Cellular Therapies Inc.
CAPLYTA (Lumateperone)
Bipolar depression

+1.05  +2%
PDUFA PDUFA date December 17, 2021.
$3.6 billion

MCRB – Seres Therapeutics Inc.
Recurrent C. Difficile infection

+0.87  +4%
Phase 3 Phase 3 data met primary endpoint - August 10, 2020. Open-label trial enrollment to be completed 3Q 2021.
$2.1 billion

MESO – Mesoblast Limited
Rexlemestrocel (MPC-06-ID)
Chronic low back pain

+0.12  +1%
Phase 3 Phase 3 data released February 10, 2021.
$1.1 billion

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

-0.47  -2%
NDA Filing Phase 3 trial met primary endpoint - September 14, 2020. NDA filing due mid-2021.
$674.5 million

ODT – Odonate Therapeutics Inc.
Tesetaxel - CONTESSA
Metastatic breast cancer (MBC)

+0.03  +1%
Phase 3 Phase 3 trial met primary endpoint - August 24, 2020. Median PFS was 9.8 months for tesetaxel plus capecitabine vs 6.9 months capecitabine alone. Announced March 22, 2021 that development will be discontinued.
$130.2 million

OTLK – Outlook Therapeutics Inc.
NORSE 1 (ONS-5010-001)
Wet age-related macular degeneration (wet AMD)

-0.11  -4%
Phase 3 Phase 3 data released August 26, 2020. No statistical differences between Lucentis (ranibizumab) and ONS-5010.
$406.2 million

SBBP – Strongbridge Biopharma plc
COR-003 (levoketoconazole) - LOGICS
Endogenous Cushing’s syndrome

-0.02  -1%
Phase 3 Phase 3 top-line data met primary endpoint.
$189.1 million

SPRO – Spero Therapeutics Inc.
Tebipenem (SPR994)
Complicated urinary tract infections (cUTI)

-0.33  -2%
NDA Filing NDA filing due 2H 2021.
$422.1 million