BPC October 06 update

Kiniksa KNSA meets mid-stage trial goals +18%; Corbus CRBP negative cystic fibrosis data -36%

Price and Volume Movers

Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) shares closed up 18% to $18.74 on news its Phase 2 trial of mavrilimumab in giant cell arteritis (GCA) achieved both the primary and secondary efficacy endpoints.

Corbus Pharmaceuticals Holdings, Inc. (NASDAQ:CRBP) announced its Phase 2b trial of lenabasum in patients with cystic fibrosis did not meet its primary endpoint. Shares fell 36% to $1.21.

Axovant Gene Therapies Ltd. (NASDAQ:AXGT) reported six-month follow-up data from cohort 2 of its SUNRISE-PD Phase 2 trial of AXO-Lenti-PD for the treatment of Parkinson's disease. Two evaluable patients in the cohort showed a 21-point mean improvement in the UPDRS Part III "OFF" score, which assesses motor function. However, because of COVID-19 and a patient refusal, two of the four patients in the cohort were not evaluated in UPDRS assessments. Shares closed down 26% to $4.15.

Vir Biotechnology, Inc. (Nasdaq: VIR) and GlaxoSmithKline plc (NYSE: GSK) announced it will enter Phase 3 trials for VIR-7831, in development for the early treatment of COVID-19 in patients who are at high risk of hospitalization. Initial Phase 3 results may be available at the end of 2020, with primary endpoint data due January 2021.

Gamida Cell Ltd. (Nasdaq: GMDA) announced that its Phase 3 trial of omidubicel, for patients in need of bone marrow transplant, met all three of its secondary endpoints. The company announced in May that it achieved its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment. Shares closed Tuesday up 10% to $4.60.

SpringWorks Therapeutics, Inc. (NASDAQ:SWTX) announced after hours that it has commenced an underwritten public offering of $150m of shares of its common stock.

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

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

ADVANCERS:

Celldex Therapeutics, Inc. (CLDX): $18.16; +19%.

Personalis, Inc. (PSNL): $28.20; +16%.

Beam Therapeutics Inc. (BEAM): $27.39; +15%.

Endo International plc (ENDP): $4.25; +14%.

ARCA biopharma, Inc. (ABIO): $5.18; +13%.

DECLINERS:

Corvus Pharmaceuticals, Inc. (CRVS): $4.17; -14%.

Iovance Biotherapeutics, Inc. (IOVA): $28.10; -12%.

Y-mAbs Therapeutics, Inc. (YMAB): $38.13; -9%.

RedHill Biopharma Ltd. (RDHL): $9.45; -8%.

Solid Biosciences Inc. (SLDB): $4.37; -8%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AFMD – Affimed N.V.
AFM13 + NK cells
CD30-positive lymphomas

Phase 1 Phase 1 initiation of dosing announced October 6, 2020.
$269.5 million

AXGT – Axovant Gene Therapies Ltd.
AXO-Lenti-PD - SUNRISE-PD
Parkinson's disease

Phase 1/2 Phase 1/2 6-month data released October 6, 2020. Two evaluable patients in Cohort 2 demonstrated a 21-point mean improvement in the UPDRS Part III “OFF” score, representing a 40% improvement from baseline average score. Two other patients not evaluated. Further data due at R&D Day October 30, 2020. Randomized trial to commence 2021.
$170.1 million

BBI – Brickell Biotech, Inc.
Sofpironium Bromide
Axillary hyperhidrosis

Phase 3 U.S. Phase 3 trial initiation announced October 6, 2020.
$26.4 million

CANF – Can-Fite Biopharma Ltd Sponsored ADR (Israel)
Piclidenoson (CF101)
Rheumatoid arthritis

Phase 3 Phase 3 interim analysis recommended trial to be discontinued - October 6, 2020.
$25.6 million

CANF – Can-Fite Biopharma Ltd Sponsored ADR (Israel)
Piclidenoson (CF101)
Psoriasis

Phase 3 Phase 3 trial ongoing.
$25.6 million

CRBP – Corbus Pharmaceuticals Holdings Inc.
Lenabasum
Cystic Fibrosis

Phase 2b Phase 2b trial did not meet primary endpoint - October 6, 2020.
$77.3 million

ETON – Eton Pharmaceuticals Inc.
ET-101
Seizures/migraine

NDA Filing NDA filing announced October 6, 2020.
$179.8 million

GMDA – Gamida Cell Ltd.
NiCord (omidubicel)
Acute myeloid leukemia

BLA Filing Phase 3 data met primary endpoint. Secondary endpoints also met. BLA filing due 4Q 2020.
$266.7 million

GSK – GlaxoSmithKline PLC
VIR-7831 / GSK418213
COVID-19 antibody

Phase 2/3 Initial Phase 3 results may be available at the end of 2020; results for the primary endpoint are expected January 2021.
$85.2 billion

KLDO – Kaleido Biosciences Inc.
KB195 (UNLOCKED)
Urea cycle disorder (UCD)

Phase 2 Phase 2 data due 2H 2021.
$232.8 million

KLDO – Kaleido Biosciences Inc.
KB295
Ulcerative colitis (UC)

Phase 2 Phase 2 top-line data due mid-2021.
$232.8 million

KNSA – Kiniksa Pharmaceuticals Ltd.
Mavrilimumab
Giant cell arteritis (GCA)

Phase 2 Phase 2 trial met endpoints - October 6, 2020. Data to be presented at ACR meeting November 9, 2020 at 11:30 a.m. ET.
$1.1 billion

NTRP – Neurotrope Inc.
Bryostatin-1
Alzheimer's disease

Phase 2 Phase 2 commencement of dosing announced October 6, 2020.
$25.7 million

PHAS – PhaseBio Pharmaceuticals Inc.
Bentracimab PB2452
Reversal agent for ticagrelor

Phase 3 Phase 3 commencement announced October 6, 2020.
$80.4 million