BPC March 25 update

ProQR PRQR shares rally on delayed reaction to data +62%; ChemoCentryx CCXI Advisory Committee meeting May 6

Price and Volume Movers

The FDA announced that the Arthritis Advisory Committee will meet on May 6, 2021 to discuss the new drug application (NDA) filed by ChemoCentryx, Inc. (NASDAQ: CCXI) for avacopan oral capsules for the treatment of anti-neutrophil cytoplasmic antibody-associated vasculitis. The PDUFA date is July 7.

ProQR Therapeutics N.V. (Nasdaq: PRQR) shares rallied to close up 62% to $9.22, after initially only adding 5% Wednesday following the release of data from its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP). Across all treated patients (n=14), a mean benefit of 6 letters was observed at week 48 in the treated eyes compared to the untreated eyes after a single injection.

Bristol Myers Squibb (NYSE:BMY) announced that its Phase 2/3 RELATIVITY-047 (CA224-047) trial evaluating a combination of relatlimab, an anti-LAG-3 antibody, and Opdivo (nivolumab) versus Opdivo alone in patients with previously untreated metastatic or unresectable melanoma, met its primary endpoint of progression-free survival (PFS). Other companies running Lag3-targeting programs include Immutep Limited (NASDAQ: IMMP) and its drug Eftilagimod alpha. Shares of Immutep rose 40% to $3.68 on the news.

Pfizer Inc. (NYSE:PFE) and Eli Lilly and Company (NYSE:LLY) announced that the FDA Arthritis Drug Safety and Risk Management Advisory Committees voted 19 to 1 against whether the proposed risk evaluation and mitigation strategy (REMS) for tanezumab, in patients with osteoarthritis pain, will ensure its benefits outweigh risks.

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Major price movers (stocks priced > $1.00, volume > 50k listed on NASDAQ/NYSE):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume:

ADVANCERS:

Rubius Therapeutics, Inc. (RUBY): $31.78; +27%.

Intercept Pharmaceuticals, Inc. (ICPT): $24.88; +22%.

F-star Therapeutics, Inc. (FSTX): $13.84; +20%.

NanoViricides, Inc. (NNVC): $5.50; +20%.

Petros Pharmaceuticals, Inc. (PTPI): $3.89; +19%.

DECLINERS:

Evofem Biosciences, Inc. (EVFM): $1.71; -27%.

Evolus, Inc. (EOLS): $12.69; -23%.

Cyclo Therapeutics, Inc. (CYTH): $9.00; -18%.

Oncolytics Biotech Inc. (ONCY): $3.49; -15%.

Gritstone Oncology, Inc. (GRTS): $10.13; -14%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ALBO – Albireo Pharma Inc.
Odevixibat (ASSERT)
Alagille syndrome

Phase 3 Phase 3 data due 2022.
$628.5 million

ATHA – Athira Pharma Inc.
ATH-1017 (ACT-AD)
Mild-to-moderate Alzheimer's disease

Phase 2 Phase 2 top-line data due early-2022.
$708.3 million

ATHX – Athersys Inc.
MultiStem
Ischemic stroke

Phase 2/3 Phase 2/3 top-line data due 2021.
$383.1 million

BMY – Bristol-Myers Squibb Company
Relatlimab and Nivolumab (CA224-047)
Melanoma

Phase 2/3 Phase 2/3 trial met its primary endpoint of progression-free survival.
$139.9 billion

CANF – Can-Fite Biopharma Ltd Sponsored ADR (Israel)
Namodenoson (CF102)
Cancer - advanced liver cancer second line

Phase 3 Phase 3 trial to commence 4Q 2021.
$40.9 million

CCXI – ChemoCentryx Inc.
Avacopan - CCX168 (ADVOCATE)
Associated vasculitis (AAV)

PDUFA Advisory Committee May 6, 2021. PDUFA date July 7, 2021.
$3.4 billion

DFFN – Diffusion Pharmaceuticals Inc.
Trans sodium crocetinate (TSC) - TCOM Study
Peripheral tissue oxygenation

Phase 1 Phase 1 data due 2Q 2021.
$85.9 million

EYEN – Eyenovia Inc.
MydCombi
Mydriasis - pupil dilation

PDUFA PDUFA date October 28, 2021.
$147.3 million

HRMY – Harmony Biosciences Holdings Inc.
WAKIX (pitolisant)
Myotonic dystrophy

Phase 2 Phase 2 trial to be initiated 1H 2021.
$1.6 billion

LLY – Eli Lilly and Company
Tanezumab
Osteoarthritis

PDUFA Advisory Committee March 25, 2021 voted 19 to 1 against whether the proposed REMS will ensure its benefits outweigh risks.
$176.9 billion

NKTX – Nkarta Inc.
NKX101
Acute myeloid leukemia / myelodysplastic syndromes

Phase 1 Phase 1 interim data due by end of 2021.
$1.1 billion

PHIO – Phio Pharmaceuticals Corp.
PH-762
Melanoma

Phase 1 Phase 1 trial to be initiated 4Q 2021.
$37.1 million

RLAY – Relay Therapeutics Inc.
RLY-4008
Solid tumors

Phase 1 Phase 1 initial data due 2H 2021.
$3 billion

TRVI – Trevi Therapeutics Inc.
Nalbuphine ER - PRISM
Prurigo Nodularis

Phase 2/3 Phase 2b/3 enrolment to be completed in 3Q 2021 with top-line data due 4Q 2021.
$52 million