BPC September 07 update

Adaptimmune ADAP +29% collaboration with Genentech; Contract manufacturers' delay leaves CorMedix CRMD -23% NDA filing date uncertain

Price and Volume Movers

Adaptimmune Therapeutics PLC (NASDAQ: ADAP) announced a collaboration with Genentech to research, develop and commercialize cancer-targeted allogeneic T-cell therapies. Adaptimmune receives $150 million upfront, $150 million over the next five years and an additional $3 billion in milestone payments. Shares closed up 29% at $6.37.

CorMedix Inc. (NASDAQ: CRMD) announced its resubmission for its New Drug Application (NDA) for DefenCath has been delayed due to delays with its third-party contract manufacturer. The timeline to address the deficiencies at the facility is uncertain at this time. Shares closed down 23% at $5.01.

Pliant Therapeutics (NASDAQ: PLRX) reported interim results from its Phase 2a PET imaging clinical trial of PLN-74809 in patients with Idiopathic Pulmonary Fibrosis (IPF). Pilant said that across four dose levels, all patients achieved greater than 50% target engagement. Shares traded up as high as $24.17, but following a sell off closed down 0.8% at $19.16.

MacroGenics, Inc. (NASDAQ: MGNX) released after hours final overall survival (OS) results of the SOPHIA Phase 3 study in adult patients with metastatic HER2-positive breast cancer. Analysis showed that MARGENZA plus chemotherapy did not demonstrate a statistically significant advantage compared to trastuzumab plus chemotherapy. Shares were halted during the after hours, trading down 7% after hours at $20.15.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced on Monday that the FDA placed a clinical hold on the Phase 1/2 Phearless study of BMN 307, an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy for adults with phenylketonuria (PKU). The FDA's clinical hold was based on interim safety findings from a pre-clinical, non-GLP pharmacology study. Shares closed down 8% at $77.81.

Advancers

CompanyPriceChange
CRVS
Corvus Pharmaceuticals Inc.
$5.32+3.06  +135.40%
IPHA
Innate Pharma S.A.
$8.10+2.35  +40.87%
CYDY
Cytodyn Inc
$2.01+0.37  +22.56%
SURF
Surface Oncology Inc.
$7.10+1.09  +18.14%
ATNM
Actinium Pharmaceuticals Inc. (Delaware)
$7.83+1.15  +17.22%
EVLO
Evelo Biosciences Inc.
$8.34+1.03  +14.09%
MGTA
Magenta Therapeutics Inc.
$7.79+0.92  +13.39%
CNTB
Connect Biopharma Holdings Limited
$23.06+2.51  +12.21%
TRVN
Trevena Inc.
$1.36+0.14  +11.48%
FULC
Fulcrum Therapeutics Inc.
$30.83+3.14  +11.34%

Decliners

CompanyPriceChange
PTGX
Protagonist Therapeutics Inc.
$17.53-28.6  -62.00%
TCRR
TCR2 Therapeutics Inc.
$9.99-5.73  -36.45%
ELYM
Eliem Therapeutics Inc
$18.77-5.27  -21.92%
JANX
Janux Therapeutics Inc.
$27.39-6.96  -20.26%
ICVX
Icosavax Inc.
$33.06-6.4  -16.22%
IPSC
Century Therapeutics Inc.
$24.76-4.49  -15.35%
GRTS
Gritstone bio Inc.
$11.50-2.04  -15.07%
VERV
Verve Therapeutics Inc.
$59.88-9.99  -14.30%
INAB
IN8bio Inc.
$7.00-1.09  -13.47%
TNYA
Tenaya Therapeutics Inc.
$20.85-3.2  -13.31%

Pipeline Database Updates

Drug Price Stage Catalyst Market Cap

ADAP – Adaptimmune Therapeutics plc
AFP (ADP-A2AFP)
Hepatocellular carcinoma

$5.40
+0.11  +2%
Phase 1 Phase 1 data displayed one complete response and a disease control rate of 64% with stable disease for ≥16 weeks in two patients. Phase 1 top-line data has an acceptable safety profile. Dose escalation is complete September 5, 2021.
$842.9 million

ALNY – Alnylam Pharmaceuticals Inc.
Vutrisiran (HELIOS-A)
ATTR Amyloidosis

$190.34
+0.14  +0%
Phase 3 Positive results from subgroup analyses and exploratory endpoints compared with placebo at 9-month period. September 7, 2021
$22.6 billion

BMRN – BioMarin Pharmaceutical Inc.
BMN 307
Phenylketonuria (PKU)

$77.76
+1.26  +2%
Phase 1/2 Phase 1/2 clinical hold announced September 6, 2021.
$14.3 billion

CADL – Candel Therapeutics Inc.
CAN-2409 combined with Valacyclovir
Prostate cancer

$7.84
-0.49  -6%
Phase 3 Phase 3 enrollment completed September 7, 2021 with data due 2024.
$224.9 million

CRMD – CorMedix Inc.
DEFENCATH (Neutrolin)
Hemodialysis patients with central venous catheters

$4.67
-0.01  -0%
NDA Filing CRL announced March 1, 2021. No timeline for resubmitting NDA, delays in its third party contract manufacturer noted.
$177.9 million

CTXR – Citius Pharmaceuticals Inc.
Denileukin diftitox (E7777)
CTCL non-Hodgkin lymphoma

$2.08
0.00  0%
Phase 3 Phase 3 recruited last patient, top-line results due first half of 2022. A BLA to be filed with the FDA by the end of 2022.
$303.6 million

EDIT – Editas Medicine Inc.
AGN-151587 (EDIT-101) - BRILLIANCE
Leber Congenital Amaurosis type 10 (LCA10).

$63.20
+2.20  +4%
Phase 1/2 Phase 1/2 initial data due to be presented at the International Symposium on Retinal Degeneration planned for September 29, 2021.
$4.3 billion

GBT – Global Blood Therapeutics Inc.
OXBRYTA (Voxelotor)
Sickle cell disease - children age 4-11

$28.03
+1.68  +6%
PDUFA priority review PDUFA date under priority review December 25, 2021.
$1.7 billion

HZNP – Horizon Therapeutics Public Limited Company
TEPEZZA (Teprotumumab)
Thyroid Eye Disease (TED) - chronic

$109.71
+1.76  +2%
Phase 3 Phase 4 first patient has been enrolled September 7, 2021.
$24.8 billion

MGNX – MacroGenics Inc.
MARGENZA (margetuximab)
Metastatic breast cancer

$20.48
-0.51  -2%
Approved FDA approval announced December 16, 2020. Final overall survival data did not demonstrate MARGENZA plus chemotherapy had a statistically significant advantage compared to trastuzumab plus chemotherapy; September 7, 2021.
$1.3 billion

PLRX – Pliant Therapeutics Inc.
PLN-74809
Idiopathic pulmonary fibrosis

$18.24
-0.76  -4%
Phase 2a Phase 2a PET positive interim data results released September 7, 2021.
$655.5 million

PRQR – ProQR Therapeutics N.V.
QR-421a (Stellar)
Retinitis pigmentosa

$8.47
+0.23  +3%
Phase 1/2 Phase 1b/2 interim data showed BCVA stabilization was observed in QR-421a-treated eyes, versus decline observed in untreated eyes. No serious adverse events or inflammation was observed, noted September 9, 2021. Two-Phase 2/3 trials planned.
$568.1 million

TVTX – Travere Therapeutics Inc.
Sparsentan (DUPLEX)
Focal segmental glomerulosclerosis (FSGS)

$22.51
+0.07  +0%
Phase 3 Phase 3 trial met partial remission of proteinuria endpoint - February 2, 2021. Noted September 7, 2021 data to support an application for accelerated approval in 2022. Additional estimated glomerular filtration (eGFR) data potentially due due 1H 2022. Full top-line data due 1H 2023.
$1.4 billion