BPC December 18 update

Final biotech week and review and weekly watch list for 2020

Weekly watchlist

This week’s edition is the final BioPharmCatalyst weekly review for 2020. We thank you for your readership and wish all readers a relaxing holiday season ahead.

A number of clinical catalysts still remain on the calendar for 2020 as listed below. Expect a number to be released during the upcoming week. However, it is not uncommon for companies to miss their timeline guidance so expect some events to slip into 2021.

First, as usual, let’s review the week the week that was with a selection of newsmakers.

REGULATORY:

Karyopharm Therapeutics Inc. (NASDAQ:KPTI) announced Friday that the FDA approved Xpovio (selinexor) in combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy. Shares closed up Friday up 8% to $16.47.

Myovant Sciences (NYSE:MYOV) also announced Friday that the FDA approved Orgovyx (relugolix) for the treatment of adult patients with advanced prostate cancer.

MacroGenics, Inc. (Nasdaq: MGNX) shares closed the week up 14% to $25.27 on news the FDA approved Margenza in combination with chemotherapy, for the treatment of adult patients with metastatic HER2-positive breast cancer who have received two or more prior anti-HER2 regimens.

FibroGen, Inc. (Nasdaq: FGEN) announced late Friday that the FDA has extended the review period of the New Drug Application (NDA) for roxadustat for the treatment of anemia of chronic kidney disease (CKD) by three months. The new PDUFA date is March 20, 2021. The delay is due to the submission of additional analyses of existing data from the company which require an extension of the original PDUFA date.

Avenue Therapeutics, Inc. (NASDAQ:ATXI) shares closed the week up 75% to $6.29 on news that it intends to resubmit its New Drug Application (NDA) for IV tramadol in February 2021. The FDA issued a Complete Response Letter (CRL) in October.

CLINICAL DATA:

Arvinas, Inc. (NASDAQ:ARVN) shares closed the week up 130% to $68.95 following the release of early data from its Phase 1 trial in patients with prostate cancer. PSA levels dropped by more than 50% in 2/5 (40%) patients with certain androgen receptor mutations.

vTv Therapeutics Inc. (NASDAQ:VTVT) announced that its Phase 2 Elevage study of azeliragon in people with mild Alzheimer's disease and type 2 diabetes did not meet its primary endpoint. Shares closed Wednesday down 23% to $2.30.

COVID-19 VACCINE:

The FDA Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted 20-0 (1 abstention) in favor that the benefits of the Moderna, Inc (NASDAQ:MRNA) COVID-19 vaccine, outweigh the risks in individuals 18 years and over.

OTHER

Prevail Therapeutics Inc. (NASDAQ:PRVL) shares closed Tuesday up 82% to $22.75 on news it will be acquired by Eli Lilly and Company (NYSE:LLY) for $22.50 per share in cash plus one non-tradable contingent value right ("CVR") worth up to $4.00 per share.

Alexion Pharmaceuticals, Inc (NASDAQ:ALXN) shares closed Monday up 29% to $156.31 on news that AstraZeneca (NYSE:AZN) intends to acquire the company for $175 per share.

Mereo BioPharma (Nasdaq: MREO) shares closed Friday up 63% to $3.60 following a license deal with Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE) for setrusumab, a monoclonal antibody in clinical development for osteogenesis imperfecta (OI). Ultragenyx will have an exclusive license to develop and commercialize setrusumab in the US and rest of the world, excluding Europe where Mereo retains commercial rights. Ultragenyx will make an upfront payment of $50m to Mereo.

Drug Stage Catalyst Market Cap

APRE – Aprea Therapeutics Inc.
APR-246 (eprenetapopt) and azacitidine
TP53 mutant Myelodysplastic syndromes (MDS)

Phase 3 Phase 3 top-line data did not meet primary endpoint - December 28, 2020.
$124.4 million

BLPH – Bellerophon Therapeutics Inc.
INOpulse
Pulmonary Hypertension-Sarcoidosis (PH-Sarc)

Phase 2 Phase 2 top-line data due 1Q 2021.
$73.8 million

CALA – Calithera Biosciences Inc.
CB-839 + Cabozantinib (CANTATA)
Renal cell carcinoma

Phase 2 Phase 2 top-line data did not meet primary endpoint - January 4, 2020.
$206.7 million

CCXI – ChemoCentryx Inc.
Avacopan - CCX168 (ACCOLADE)
C3 glomerulopathy (C3G)

Phase 2 Phase 2 trial did not meet primary endpoint - December 21, 2020.
$4.4 billion

CERC – Cerecor Inc.
CERC-002
COVID-19 induced Acute Respiratory Distress Syndrome (ARDS)

Phase 2 Phase 2 data released January 5, 2021.
$273.4 million

CPRX – Catalyst Pharmaceuticals Inc.
Firdapse
Spinal Muscular Atrophy (SMA) Type 3

Phase 2 Phase 2 trial met primary endpoint. Secondary endpoints not met. Will not pursue development further.
$380.4 million

MRUS – Merus N.V.
MCLA-158
Colorectal cancer

Phase 1 Phase 1 trial ongoing.
$791.8 million

RYTM – Rhythm Pharmaceuticals Inc.
Setmelanotide
Alstrom Syndrome and Bardet-Biedl Syndrome

Phase 3 Phase 3 top-line data met primary endpoint - December 22, 2020. All primary endpoint responders were patients with BBS. Alstrom syndrome patients did not meet primary endpoint.
$1.6 billion

SNGX – Soligenix Inc.
SGX942 (dusquetide)
Oral Mucositis in Patients with Head & Neck Cancer

Phase 3 Phase 3 trial did not meet primary endpoint - December 22, 2020.
$50.4 million