BPC August 07 update

Biotech stock catalysts to watch in August; Biotech week in Review

Weekly watchlist

Over the last three weeks we have highlighted upcoming Phase 1/2, Phase 3, and PDUFA catalysts to watch this quarter. This week we narrow our focus down to key events likely due by the end of this month.

First, let’s review the week that was with notable news.

COVID-19 VACCINE:

Novavax, Inc. (NASDAQ: NVAX) shares closed the week up 19% to $170.29. The company released initial Phase 1 data from its trial of its COVID‑19 vaccine noting that it “was generally well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera.”

CLINICAL DATA:

CymaBay Therapeutics, Inc. (NASDAQ: CBAY) shares closed the week up 76% to $6.26 on news its ENHANCE trial evaluating the safety and efficacy of seladelpar for the treatment of primary biliary cholangitis (PBC) achieved the primary endpoint.

REGULATORY:

GW Pharmaceuticals plc (Nasdaq: GWPH) announced the FDA approved Epidiolex (cannabidiol) oral solution to treat seizures associated with tuberous sclerosis complex (TSC) in patients one year of age and older. Shares closed up 4% on Monday to $132.87 on the news but fell sharply on Friday following its earnings release, closing down at $109.37.

Biogen (Nasdaq: BIIB) and Eisai, Co., Ltd. announced the FDA has accepted their Biologics License Application (BLA) for aducanumab for Alzheimer’s disease with a PDUFA date under Priority Review of March 7, 2021. Shares closed Friday up 10% to $305.71.

PTC Therapeutics, Inc. (NASDAQ:PTCT) and Roche (OTCQX:RHHBY) announced that the FDA has approved Evrysdi (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older.

DBV Technologies (NASDAQ: DBVT) announced the FDA issued a Complete Response Letter regarding its Biologics License Application (BLA) for Viaskin Peanut to treat peanut allergies in children ages 4 to 11 years. The FDA requested the company run a new human factor trial and supply additional Chemistry, Manufacturing and Controls data. Shares slid to close the week down 45% to $2.26.

Trevena, Inc. (NASDAQ: TRVN) received approval from the FDA late-Friday for Olinvyk (oliceridine), an opioid agonist for the management of moderate to severe acute pain in adults

COLLABORATIONS:

Denali Therapeutics Inc. (NASDAQ: DNLI) announced that it has selected DNL151 to progress into late stage studies in Parkinson’s disease patients and through a collaboration with Biogen (NASDAQ:BIIB) it will receive $560m upfront and a $465m equity investment. Patient enrollment is expected to commence in 2021. Shares closed Thursday up 36% to $31.66.

EARNINGS:

Mallinckrodt plc (NYSE:MNK) shares closed the week down 35% to $1.44 following the release of second quarter earnings. The company reported a net loss of $933.1m, or $11.04 a share. The company noted that due to pressures from the Acthar Gel Medicaid matter, the ongoing opioid litigation and its current poor debt situation, it is exploring a range of options, including the possibility of a filing for reorganization in bankruptcy under Chapter 11.

Horizon Therapeutics plc (NASDAQ:HZNP) shares closed Wednesday up 24% to $76.38 on the back of its second quarter earnings report. The company saw net sales rising 44% over the year to $463m, well above analyst expectations.

Likely August biotech stock events to watch (note ODT and SPRO are officially down for release this quarter so data might come out in September).

Drug Stage Catalyst Market Cap

ALBO – Albireo Pharma Inc.
Odevixibat (A4250) - PEDFIC- 1
Progressive familial intrahepatic cholestasis (PFIC)

Phase 3 Phase 3 top-line data met primary endpoints - September 8, 2020.
$738.7 million

BMRN – BioMarin Pharmaceutical Inc.
Valoctocogene roxaparvovec (BMN 270)
Hemophilia A

CRL CRL announced August 19, 2020.
$14.4 billion

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

Phase 3 Phase 3 top-line data did not meet endpoints - September 8, 2020.
$155.3 million

FIXX – Homology Medicines Inc.
HMI-102
Phenylketonuria (PKU)

Phase 1/2 Phase 1/2 update due 3Q/4Q 2020.
$521.1 million

ITCI – Intra-Cellular Therapies Inc.
Lumateperone ITI-007-402
Bipolar depression

Phase 3 Phase 3 top-line data released September 9, 2020. High dose met primary endpoint but low dose missed.
$2.5 billion

LPCN – Lipocine Inc.
TLANDO - LPCN 1021
Men with low testosterone (Low T)

PDUFA PDUFA date originally set for August 28, 2020. Company noted on August 28 that the FDA requires additional time for their review. New PDUFA date not issued, however the review is expected to be completed in the coming weeks.
$105.8 million

MCRB – Seres Therapeutics Inc.
SER-109 - ECOSPOR III
Recurrent C. Difficile infection

Phase 3 Phase 3 data met primary endpoint - August 10, 2020.
$2.3 billion

MESO – Mesoblast Limited
Rexlemestrocel
Heart failure

Phase 3 Phase 3 data due 4Q 2020.
$2.1 billion

MESO – Mesoblast Limited
Remestemcel-L (MSC-100-IV)
acute graft versus host disease (aGVHD) in children

PDUFA priority review PDUFA date under priority review September 30, 2020. FDA Oncologic Drugs Advisory Committee meeting August 13, 2020 voted 8-2 supporting approval.
$2.1 billion

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

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. High levels of neutropenia noted (71.2%). Overall survival data due in 2022.
$483.2 million

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

Phase 3 Phase 3 data released August 26, 2020. No statistical differences between Lucentis (ranibizumab) and ONS-5010.
$88.5 million

SPRO – Spero Therapeutics Inc.
SPR994 (ADAPT-PO)
Complicated urinary tract infections (cUTI)

Phase 3 Phase 3 data released September 8, 2020 showed non-inferiority versus intravenous ertapenem.
$262.2 million

TCDA – Tricida Inc.
TRC 101
Chronic kidney disease

CRL CRL announced August 24, 2020.
$568.6 million