BPC May 28 update

BioMarin BMRN releases updated hemophilia data - shares fall 5%

Price and Volume Movers

Celgene Corporation (NASDAQ: CELG) announced the FDA approved Revlimid in combination with rituximab (lenalidomide) for the treatment of adult patients with previously treated follicular lymphoma (FL) or marginal zone lymphoma (MZL) following Priority Review designation. The approval came one month before its assigned PDUFA date of June 27, with such early decisions not uncommon for drugs under priority review.

Allergan plc (NYSE: AGN) also announced that the FDA has approved its supplemental New Drug Application (sNDA) for Vraylar (cariprazine) to treat depressive episodes associated with bipolar I disorder (bipolar depression) in adults.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) released three-year data from its Phase 1/2 trial and interim data from its Phase 3 trial of valoctocogene roxaparvovec, for adults with severe hemophilia A. In its Phase 1/2 trial, after three years the Annualized Bleed Rate (ABR) went from a mean of 16.5 and median 16.3 to an impressive mean of 0.7 and a median zero, a 96% reduction in mean ABR over three years. Questions were raised regarding the durability of the therapy, with levels of Factor VII falling off after three years. Durability was also questioned with the release of interim Phase 3 data. The company will meet with regulatory agencies and intends to announce the timing for its planned EMA and FDA marketing applications in 3Q 2019. Shares closed down 5% to $84.50.

Kezar Life Sciences, Inc. (Nasdaq: KZR) announced the release of its abstract for the upcoming European League Against Rheumatism (EULAR) 2019 annual meeting taking place in from June 12-15. The abstract contained data from the first two cohorts of its Phase 1/2 open-label dose escalation trial of KZR-616 in patients with systemic lupus erythematosus (SLE). Both safety and efficacy data were disappointing, with shares closing down 41% to $9.90.

Rexahn Pharmaceuticals, Inc. (NYSE American: RNN) announced that it will move from the NYSE American exchange to Nasdaq, and expects to begin trading on Nasdaq under the symbol "REXN" on or about June 10, 2019.


Major price movers (stocks priced > $1.00, volume > 50k):

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


Armata Pharmaceuticals, Inc. (ARMP): $4.15; +32%.

Avadel Pharmaceuticals plc (AVDL): $1.79; +15%.

Motif Bio plc (MTFB): $2.10; +15%.

Sesen Bio, Inc. (SESN): $2.27; +12%.

PhaseBio Pharmaceuticals, Inc. (PHAS): $11.56; +10%.


Bellicum Pharmaceuticals, Inc. (BLCM): $2.06; -13%.

Teva Pharmaceutical Industries Limited (TEVA): $9.52; -12%.

Evolus, Inc. (EOLS): $14.51; -10%.

Tonix Pharmaceuticals Holding Corp. (TNXP): $1.85; -9%.

Novan, Inc. (NOVN): $2.06; -8%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AGN – Allergan plc
Bipolar I Depression

PDUFA FDA Approval announced May 28, 2019.
$56.1 billion

ARGX – argenx SE
Efgartigimod (ARGX-113)
Myasthenia gravis (MG)

Phase 3 Phase 3 data due 2H 2020.
$4.4 billion

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

Phase 3 Phase 3 interim data released May 28, 2019 - Factor VIII level =36 (23 to 26 weeks).
$12.2 billion

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

BLA Filing BLA filing due 4Q 2019.
$12.2 billion

CELG – Celgene Corporation
Relapsed or Refractory Follicular Lymphoma

Approved FDA Approval announced May 28, 2019.
$71.6 billion

CRBP – Corbus Pharmaceuticals Holdings Inc.
Lenabasum (resunab) - DETERMINE

Phase 3 Phase 3 data due 2021.
$310.7 million

KALV – KalVista Pharmaceuticals Inc.
Hereditary Angioedema (HAE)

Phase 2 Phase 2 data due late 2019.
$196.7 million

KZR – Kezar Life Sciences Inc.

Phase 1/2 Phase 1b updated data to be presented at ACR/ARP November 12, 2019. Phase 2 interim analysis 1H 2021 with final data 2H 2021.
$62.6 million

MYOK – MyoKardia Inc.
Heart failure

Phase 2a Phase 2a top-line data due 4Q 2019.
$2.6 billion

ORTX – Orchard Therapeutics plc
Metachromatic leukodystrophy (MLD)

Phase 2 Phase 2 data due by the end of 2019.
$1.1 billion

SAVA – Cassava Sciences Inc.
Alzheimer’s disease

Phase 2 Phase 2b trial initiation announced September 16, 2019.
$21.4 million

SPPI – Spectrum Pharmaceuticals Inc.
Non-small cell lung cancer (NSCLC) with exon 20 insertion mutation in EGFR or HER2

Phase 2 Phase 2 top-line data from Cohort 1 due 4Q 2019. Cohort 2 data due mid-2020.
$914.1 million