BPC October 04 update

Xenon XENE +103% Phase 2b data; Regenxbio RGNX -20% Phase 2 data

Price and Volume Movers

Xenon Pharmaceuticals, Inc. (NASDAQ: XENE) announced top-line results from its Phase 2b X-TOLE trial of XEN1101 to treat focal epilepsy. The trial met its primary endpoint and reported a reduction of focal seizure frequency by 52.8% compared to placebo. Xenon also announced after-hours that it will undergo a public offering for $250 million of its common stock. Shares closed up 103% at $31.50.

Regenxbio, Inc. (NASDAQ: RGNX) released initial data Friday from its ongoing Phase 2 AAVIATE trial of RGX-314 to treat wet age-related macular degeneration (wet AMD). Fourteen patients reported a mean Best Corrected Visual Acuity (BCVA) change of -2.8 letters when measured from Day 1 and -0.6 letters when measured from Week 1. Regenxbio noted the treatment was well tolerated; however, four serious adverse events (SAEs), including one death, were reported, all of which were not treatment-related. Shares closed down 20% at $32.33.

Progenity, Inc. (NASDAQ: PROG) announced that it has entered into a direct offering for 13,333,334 shares of its common stock at $1.50 per share. The gross proceeds from this offering are $20 million. Shares closed down 22% at $1.40.

Redhill Biopharma (NASDAQ: RDHL) released an analysis of its Phase 2/3 trial of oral opaganib to treat COVID-19. Data noted a 62% reduction in mortality compared to placebo. 77% of opaganib-treated patients reached room air by Day 14 compared to 63.5% treated with placebo. Shares closed up 12% at $4.88.

Advancers

CompanyPriceChange
AUPH
Aurinia Pharmaceuticals Inc
$28.00+5.91  +26.75%
PROG
Progenity Inc.
$2.96+0.46  +18.40%
CNTX
Context Therapeutics Inc.
$6.99+0.82  +13.29%
ATHA
Athira Pharma Inc.
$10.71+1.08  +11.21%
PTPI
Petros Pharmaceuticals Inc.
$1.89+0.19  +11.18%
EYEN
Eyenovia Inc.
$6.58+0.61  +10.22%
CMPS
COMPASS Pathways Plc
$38.89+3.58  +10.14%
KALA
Kala Pharmaceuticals Inc.
$2.05+0.15  +7.89%
RPTX
Repare Therapeutics Inc.
$24.46+1.7  +7.47%
MRTX
Mirati Therapeutics Inc.
$190.29+12.99  +7.33%

Decliners

CompanyPriceChange
MTCR
Metacrine Inc.
$1.63-2.2  -57.44%
OCUL
Ocular Therapeutix Inc.
$6.91-3.92  -36.20%
AGEN
Agenus Inc.
$4.01-1.12  -21.83%
SIOX
Sio Gene Therapies Inc.
$2.02-0.34  -14.41%
APLT
Applied Therapeutics Inc.
$13.74-1.95  -12.43%
VTYX
Ventyx Biosciences Inc.
$18.65-2.37  -11.27%
GRCL
Gracell Biotechnologies Inc.
$10.42-1.3  -11.09%
TPST
Tempest Therapeutics Inc.
$12.15-1.41  -10.40%
ADIL
Adial Pharmaceuticals Inc
$3.48-0.37  -9.61%
GRTX
Galera Therapeutics Inc.
$1.82-0.19  -9.45%

Pipeline Database Updates

Drug Price Stage Catalyst Market Cap

ARQT – Arcutis Biotherapeutics Inc.
Roflumilast Cream (ARQ-151) - (DERMIS-1)
Plaque psoriasis

$21.57
-0.21  -1%
NDA Filing NDA filed October 4, 2021.
$1.1 billion

BPTS – Biophytis SA
Sarconeos (BIO101) - SARA-INT
Sarcopenia

$6.09
-0.91  -13%
Phase 2b Phase 2b data displayed that the highest dose (350 mg bid) showed a clinically meaningful improvement in the 400-Meter Walk Test (400MWT), the primary endpoint of the study, including in sub-populations at higher risk of mobility disability, after 6 months of treatment. Safety profile at the doses of 175 mg bid and of 350 mg bid showed no Serious Adverse Events (SAE) related to treatment, noted October 4, 2021. Phase 3 trial of 350 mg to be initiated in 2022.
$76.6 million

CARA – Cara Therapeutics Inc.
KORSUVA (CR845/difelikefalin tablets)
Pruritus in patients with Atopic dermatitis

$12.70
-0.29  -2%
Phase 2 Phase 2 data reported approximately 64% of subjects had BSA<10. The primary endpoint, change from baseline in Itch Numerical Rating Scale (I-NRS) score, was not met with any of the dosed groups in the overall population, noted October 4, 2021. Phase 3 trial planned for end of 2021.
$636.1 million

CFRX – ContraFect Corporation
Exebacase (DISRUPT)
Serious infections caused by Staph aureus including MRSA

$3.83
-0.04  -1%
Phase 3 Phase 2 late breaking data displayed symptom resolution in 94.3% of the exebacase-treated group versus 87.9% of SOCA-alone patients. The median time to resolution was 3 days for exebacase-treated patients compared to 6 days for SOCA-alone patients, noted October 4, 2021. Phase 3 interim futility analysis due 2H 2021.
$150.6 million

ENTA – Enanta Pharmaceuticals Inc.
EDP-305 ARGON-2
Non-alcoholic steatohepatitis (NASH)

$73.97
+1.85  +3%
Phase 2b Phase 2b interim analysis showed 1.0 mg as the optimal dose. The monontherapy has been discontinued and priority is placed on the combinations, noted October 4, 2021.
$1.5 billion

LABP – Landos Biopharma Inc.
Omilancor (BT-11)
Ulcerative Colitis

$13.84
-0.93  -6%
Phase 2 Phase 2 data displayed clinical remission in 36.1% of the 880 mg group and 35.5% of the 440 mg group during the blinded maintenance phase. Both dose cohorts had 10% of patients report an AE of ulcerative colitis worsening, noted October 4, 2021. Phase 3 trial planned.
$555.2 million

LYRA – Lyra Therapeutics Inc.
LYR-210 - LANTERN
Chronic rhinosinusitis / Chronic Sinusitis

$7.84
-0.10  -1%
Phase 2 Phase 2 trial did not meet primary endpoint - December 7, 2020. Additional Phase 2 data of the PK study outcomes showed that Mometasone Furoate (MF) blood levels were constant over the 56 days, providing further evidence that LYR-210 delivers a consistent and steady daily dose of MF with accompanying rapid symptom relief during this time period, noted October 4, 2021.
$101.9 million

PTGX – Protagonist Therapeutics Inc.
Rusfertide (PTG-300)
Hereditary hemochromatosis

$30.96
-0.09  0%
Phase 2 Phase 2 trial placed on clinical hold, September 17, 2021. Phase 2 results to be released at The Liver Meeting on November 12-15, 2021.
$1.5 billion

RDHL – Redhill Biopharma Ltd.
YELIVA (Opaganib)
COVID-19

$5.82
+0.11  +2%
Phase 2/3 Phase 2/3 preliminary top-line data results did not meet its primary endpoint September 14, 2021. Phase 2/3 data showed 62% statistically significant reduction in mortality shown for moderately severe COVID-19 patients, noted October 4, 2021.
$271.9 million

RGNX – REGENXBIO Inc.
RGX-314 (AAVIATE)
Wet AMD using suprachoroidal delivery

$36.14
+1.11  +3%
Phase 2 Phase 2 safety results reported at Retina Society 54th Annual Scientific Meeting showed that it was well tolerated by 50 patients in the first 3 cohorts - noted October 4, 2021.
$1.5 billion

SIOX – Sio Gene Therapies Inc.
AXO-Lenti-PD - SUNRISE-PD
Parkinson's disease

$2.02
-0.34  -14%
Phase 1/2 Phase 1/2 24 month data to be presented at ESGCT October 19-22, 2021.
$147.3 million

SIOX – Sio Gene Therapies Inc.
AXO-AAV-GM1
GM1 gangliosidosis

$2.02
-0.34  -14%
Phase 1/2 Phase 1/2 interim safety and biomarker data demonstrated consistent dose-dependent improvements across biomarker measures, normalization of serum beta-galactosidase activity and GM1 ganglioside in CSF in the high-dose cohort, no overt disease progression in (6/7) patients treated across low- and high-dose cohorts. No SAE reported in patients (10/10), noted October 21, 2021.
$147.3 million

XENE – Xenon Pharmaceuticals Inc.
XEN1101 (X-TOLE)
Adult Focal Epilepsy

$30.86
+0.23  +1%
Phase 2 Phase 2b trial met its primary efficacy endpoint demonstrating a statistically significant and dose-dependent reduction from baseline in monthly focal seizure frequency when compared to placebo. Additional primary and secondary seizure reduction endpoints statistically significant across all dose groups, noted October 4, 2021.
$1.6 billion

ZSAN – Zosano Pharma Corporation
Qtrypta (M207)
Migraine

$0.59
-0.03  -4%
NDA Filing CRL announced October 21, 2020. NDA filing to be resubmitted following PK trial. PK trial data reported that treatment was generally well tolerated, consistent with previous studies. The preliminary data from the PK analysis showed that there were no outliers with unexpected high plasma concentrations of zolmitriptan, noted October 4, 2021. Granted Type C meeting with FDA.
$68.7 million