BPC January 10 update

BioCryst BCRX +28% ORLADEYO revenue; Pluristem PSTI +24% Deal

Price and Volume Movers

BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) announced preliminary, unaudited ORLADEYO (berotralstat) revenue for the fourth quarter and provided new guidance for 2022. ORLADEYO net revenue is expected to more than double in 2022 to no less than $250 million with peak sales of $1 billion. Shares closed up 28% at $14.78.

Pluristem Therapeutics, Inc. (NASDAQ: PSTI) and Tnuva Group have collaborated to develop cultured cell-based products for the food industry with a new company that will receive exclusive licensing rights to use Pluristem's proprietary technology in the field of cultured meat. Pluristem shares closed up 24% at $1.83.

Molecular Partners AG (NASDAQ: MOLN) and Novartis (NYSE: NVO) announced that Part A of their EMPATHY Phase 2 trial of ensovibep to treat COVID-19, met the primary endpoint. Novartis will exercise its option to in-license ensovibep, and Molecular Partners will receive a milestone payment of 150M CHF and be entitled to a 22% royalty on sales. Molecular shares closed up 30% at $21.50.

Amicus Therapeutics (NASDAQ: FOLD) released its yearly financials for 2021 and its guidance for 2022. Amicus plans for Galafold to generate $306 million in global revenue for 2021 with a 17% YoY growth. For 2022, the revenue estimate implied a 15 to 20% YoY growth. Shares closed down 17% at 9.45.

Notable JPM presentations for Tuesday, January 11, 2022: SWTX PTGX ALLO and KPTI.

Advancers

CompanyPriceChange
ARDS
Aridis Pharmaceuticals Inc.
$1.60+0.27  +20.30%
CINC
CinCor Pharma Inc.
$18.20+2.13  +13.25%
RVLP
RVL Pharmaceuticals plc
$1.20+0.11  +10.09%
ITCI
Intra-Cellular Therapies Inc.
$42.66+3.79  +9.75%
GMDA
Gamida Cell Ltd.
$3.24+0.28  +9.46%
HILS
Hillstream BioPharma Inc.
$3.36+0.23  +7.35%
KPTI
Karyopharm Therapeutics Inc.
$8.28+0.55  +7.12%
CNTX
Context Therapeutics Inc.
$2.10+0.13  +6.60%
LABD
Direxion Daily S&P Biotech Bear 3X Shares
$45.71+2.71  +6.30%
BDSI
BioDelivery Sciences International Inc.
$3.36+0.18  +5.66%

Decliners

CompanyPriceChange
IMMX
Immix Biopharma Inc.
$4.51-0.91  -16.79%
ACXP
Acurx Pharmaceuticals Inc.
$3.64-0.7  -16.13%
CGTX
Cognition Therapeutics Inc.
$3.50-0.64  -15.46%
HOOK
HOOKIPA Pharma Inc.
$1.50-0.26  -14.77%
CVAC
CureVac N.V.
$18.73-3.12  -14.28%
BFRI
Biofrontera Inc.
$3.47-0.53  -13.25%
BSGM
BioSig Technologies Inc.
$1.80-0.27  -13.04%
HCWB
HCW Biologics Inc.
$2.02-0.3  -12.93%
VRPX
Virpax Pharmaceuticals Inc.
$2.30-0.34  -12.88%
APM
Aptorum Group Limited
$1.48-0.21  -12.43%

Pipeline Database Updates

Drug Price Stage Catalyst Market Cap

ASND – Ascendis Pharma A/S
TransCon - (PaTHway)
Hypoparathyroidism

$107.97
-3.17  -3%
Phase 3 Phase 3 top-line data due 1Q 2022. NDA due in 3Q 2022.
$6.1 billion

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

$13.99
-0.32  -2%
BLA Filing Rolling BLA filing to be completed 2Q 2022.
$1.3 billion

AUTL – Autolus Therapeutics plc
AUTO1/22 - (CARPALL)
Pediatric Acute lymphoblastic leukemia (ALL)

$3.96
-0.18  -4%
Phase 1/2 Phase 1/2 data reported that all patients showed engraftment of single and double CAR positive populations, pointing to early CAR T cell persistence, noted December 13, 2021. Additional data due in 2H 2022.
$288.8 million

BMRN – BioMarin Pharmaceutical Inc.
Valoctocogene roxaparvovec (BMN 270) - (GENEr8-1)
Hemophilia A

$84.65
+0.20  +0%
BLA Filing BLA filing 2Q 2022.
$15.5 billion

CCCC – C4 Therapeutics Inc.
CFT7455
Hematologic malignancies

$23.40
-1.04  -4%
Phase 1/2 Phase 1/2 data due 1H 2022.
$1.1 billion

DCPH – Deciphera Pharmaceuticals Inc.
QINLOCK (ripretinib) - (INTRIGUE)
Gastrointestinal Stromal Tumors (GIST) - second-line

$8.04
-0.11  -1%
Phase 3 Phase 3 top-line data did not meet the primary endpoint of improved progression-free survival (PFS) compared with the standard of care, noted November 5, 2021. Phase 3 results to be presented at ASCO January 25, 2022.
$470.2 million

EPZM – Epizyme Inc.
TAZVERIK (Tazemetostat) and RITUXAN (rituximab) - EZH-1401 - LYSA
Follicular Lymphoma

$2.07
-0.12  -5%
Phase 2 Phase 2 trial is enrolling, interim results will be presented in 2022.
$215.1 million

EXEL – Exelixis Inc.
CABOMETYX (Cabozantinib) and OPDIVO (nivolumab) and YERYOY (Ipilimumab) - (COSMIC-313)
Renal Cell Carcinoma

$17.32
+0.29  +2%
Phase 3 Phase 3 data due 1H 2022.
$5.5 billion

KPTI – Karyopharm Therapeutics Inc.
XPOVIO (selinexor) - (SIENDO)
Endometrial cancer

$8.28
+0.55  +7%
Phase 3 Phase 3 recruitment completed on December 2, 2021. Phase 3 top-line data due in 1Q 2022. sNDA due in 1H 2022.
$625.5 million

MOLN – Molecular Partners AG
Ensovibep - (EMPATHY)
COVID-19

$30.07
+0.18  +1%
Phase 2/3 Phase 2b/3 part A met primary endpoint, noted January 10, 2022.
$970.7 million

NGM – NGM Biopharmaceuticals Inc.
NGM621 - (CATALINA)
Geographic atrophy

$14.55
-0.28  -2%
Phase 2 Phase 2 enrollment completed, noted November 4, 2021. Top-line data to be released 4Q 2022.
$1.1 billion

PFE – Pfizer Inc.
Darovasertib (IDE196), MEKTOVI (​binimetinib) and Crizotinib
Metastatic uveal melanoma (MUM)

$52.79
-1.26  -2%
Phase 1/2 Phase 1/2 data released April 16, 2021. 2/9 (22%) partial responses. Phase 1/2 data reported a 31% overall response rate of 31%, noted December 7, 2021. Phase 2 enrollment ongoing with clinical data expected 1H 2022.
$296.3 billion

PTCT – PTC Therapeutics Inc.
Translarna
Duchenne muscular dystrophy caused by nonsense mutations (nmDMD)

$38.82
-1.25  -3%
Phase 3 Received a Refusal to File letter February 23 2016. Appeal rejected October 17, 2016. Decision made to resubmit NDA during 1Q 2017 under protest. Further CRL October 25, 2017. NDA to be refiled pending data from dystrophin study 041 expected by mid-2022.
$2.7 billion

PTCT – PTC Therapeutics Inc.
Vatiquinone (MIT-E)
Mitochondrial Epilepsy

$38.82
-1.25  -3%
Phase 2/3 Phase 2/3 data due by year end 2022.
$2.7 billion

TCRR – TCR2 Therapeutics Inc.
Gavo-cel (TC-210)
Mesothelin-positive solid tumors

$3.47
-0.11  -3%
Phase 1/2 Phase 1/2 completion of the 3-patient cohort at the new dose level 3.5A using a split dosing approach. Phase 1 translational data expected 1H 2022. Phase 2 expansion cohort initiation anticipated 1H 2022. Phase 2 initial data expected 2H 2022.
$132.6 million