BPC September 18 update

Biotech stock catalysts remaining for September; Biotech week in Review

Weekly watchlist

Just two weeks remain in the third quarter of 2020, yet several key catalysts remain on the calendar. A selection is displayed below, however, it should be noted that due to the nature of clinical trials, it is not uncommon for companies to miss their timeline guidance by a few days/weeks.

First let’s review the week that was with notable price-moving events in the healthcare sector.

CLINICAL DATA

Cardiff Oncology, Inc. (Nasdaq: CRDF) announced data at the European Society of Medical Oncology (ESMO) Virtual Congress 2020 from its Phase 1/2 trial of onvansertib in KRAS-mutated metastatic colorectal cancer (mCRC) patients that showed 5 out of 11 (45%) patients exhibited a partial response with 5 other patients showing stable disease, with only one patient progressing while on treatment. Shares closed the week up 46% to $8.71.

Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS) shares closed the week up 73% to $3.46 on news its Phase 3 Marigold trial evaluating oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD), a rare, genetic epilepsy with refractory seizures, met the primary endpoint. Patients given ganaxolone showed a significant 32% reduction in 28-day major motor seizure frequency, compared to a 4% reduction for those receiving the placebo (p=0.002).

REGULATORY

Mallinckrodt plc (NYSE: MNK) announced that the FDA issued a Complete Response Letter (CRL) regarding its New Drug Application (NDA) for terlipressin to treat adults with hepatorenal syndrome type 1 (HRS-1). The CRL stated that, based on the available data, the agency cannot approve the NDA in its current form and requires more information to support a positive risk-benefit profile for terlipressin for patients with HRS-1.

BUYOUTS

Gilead Sciences, Inc. (NASDAQ:GILD) announced that it will acquire Immunomedics (NASDAQ:IMMU) for $88 per share in cash or approximately $21b. Immunomedics shares closed Monday up 98% to $83.65.

Novus Therapeutics, Inc. (NASDAQ:NVUS) announced it has completed the acquisition of Anelixis Therapeutics, Inc, a privately held biotechnology company developing treatments for organ and cellular transplantation, autoimmune diseases, and neurodegenerative diseases. Novus also announced a private placement for gross proceeds of $108m. Shares closed the week up 134% to $0.89.

OTHER

Seattle Genetics, Inc. (NASDAQ:SGEN) shares closed the week up 21% to 180.94 on the back of several positive news releases. Early in the week it announced a collaboration with Merck (NYSE:MRK) to develop and commercialize ladiratuzumab vedotin, currently in Phase 2 clinical trials for breast cancer and other solid tumors. Seattle Genetics will receive a $600m upfront payment and Merck will make a $1b equity investment of 5m million shares at a price of $200 per share. Seattle Genetics also granted Merck a license to commercialize tucatinib, for the treatment of HER2-positive cancers outside of the U.S., Canada and Europe and will receive $125m upfront. On Friday the company announced its Phase 3 EV-301 trial of PADCEV (enfortumab vedotin) met its primary endpoint of overall survival compared to chemotherapy in patients with urothelial cancer.

Drug Stage Catalyst Market Cap

ASND – Ascendis Pharma A/S
TransCon
Hypoparathyroidism

Phase 2 Phase 3 trial to commence 4Q 2020.
$8.6 billion

ATRA – Atara Biotherapeutics Inc.
ATA 129 (tab-cel) - ALLELE
Epstein-Barr virus (EBV-PTLD) after solid organ transplant (SOT)

BLA Filing BLA filing due 4Q 2020.
$1 billion

BCRX – BioCryst Pharmaceuticals Inc.
Galidesivir
COVID-19

Phase 1 Information from Part 1 of the trial are due 4Q 2020.
$640.9 million

CYCN – Cyclerion Therapeutics Inc.
Olinciguat (IW-1701) - STRONG-SCD
Sickle Cell disease

Phase 2 Phase 2 data released October 14, 2020 - insufficient efficacy shown to support further development.
$76.3 million

DVAX – Dynavax Technologies Corporation
SCB-2019 + adjuvant system (Clover)
COVID-19 vaccine

Phase 1 Phase 2/3 trial is planned before the end of 2020. Detailed Phase 1 data will be made available in a peer-reviewed publication in the near future.
$410.7 million

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

Phase 1/2 Phase 1/2 update due 4Q 2020.
$479.8 million

GLYC – GlycoMimetics Inc.
GMI-1070 (rivipansel)
Vaso-occlusive crisis of sickle cell disease

Phase 3 Phase 3 data failed to meet endpoints - August 2, 2019.
$142.2 million

JNJ – Johnson & Johnson
JNJ-78436735 (Ad26.COV2-S) -ENSEMBLE
COVID-19 vaccine

Phase 3 Phase 3 trial initiation announced September 23, 2020. Emergency use authorization expected early 2021. Trial currently paused due to unexplained illness in one patient.
$364.2 billion

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

CRL CRL announced October 1, 2020.
$1.2 billion

MRNA – Moderna Inc.
mRNA-1273
COVID-19 vaccine

Phase 2/3 Phase 2 and 3 trials ongoing. Phase 3 completion of enrollment announced October 22, 2020. Interim data due November 2020.
$25.9 billion

MYOV – Myovant Sciences Ltd.
Relugolix
Advanced prostate cancer

PDUFA priority review PDUFA date under priority review December 20, 2020. Phase 3 data meet primary endpoint - November 19, 2019. Castration Resistance Free Survival data released September 29, 2020 did not achieve statistical superiority (p = 0.84).
$1.3 billion

OVID – Ovid Therapeutics Inc.
TAK-935/OV935 - ARCADE
CDKL5 Deficiency

Phase 2 Phase 2 data released September 30, 2020. Median motor seizure frequency reduction was 24%.
$326.8 million

REGN – Regeneron Pharmaceuticals Inc.
REGN-COV2
COVID-19 (Anti-viral antibody)

Phase 2/3 Phase 2/3 initial data September 29, 2020 showed virus levels decreased more quickly in non-hospitalized patients on the high dose. EUA filed October 7, 2020. Data from outpatient setting hit primary and secondary endpoints, significantly reducing viral load and patient medical visits.
$60.3 billion

SELB – Selecta Biosciences Inc.
SEL-212 (COMPARE) - Head to head trial vs Krystexxa
Tophaceous gout

Phase 2 Phase 2 top-line data released September 30, 2020. Numerically higher response rate on the primary endpoint during months 3 and 6 combined, but did not meet the primary endpoint of statistical superiority.
$360 million