BPC January 08 update

​Applied Genetic Technologies AGTC data due Thursday pre-market; Applied Therapeutics APLT shares climb 34% on pivotal data

Price and Volume Movers

Applied Genetic Technologies Corporation (Nasdaq: AGTC) announced that it plans to release interim six-month data from the expansion group, and an update of enrollment for its ongoing Phase 1/2 clinical trial in patients with X-linked Retinitis Pigmentosa (XLRP) on January 9, 2020. A conference call will commence at 8am EST.

Mirati Therapeutics, Inc. (NASDAQ:MRTX) announced an underwritten public offering of $250m of shares of its common stock. Shares are trading down 8% to $96.85 after hours.

Applied Therapeutics Inc. (Nasdaq:APLT) shares closed up 34% to $36.42 following the release of top-line data from its pivotal Phase 2 ACTION-Galactosemia trial of AT-007, in adult Galactosemia patients. AT-007 treatment resulted in a statistically significant reduction in plasma galactitol vs placebo. At the highest dose tested (20mg/kg), AT-007 significantly reduced plasma galactitol 45-54% from baseline vs. placebo (p<0.01). The company will present full data at the Society for Inherited Metabolic Disorders Annual Meeting, April 26 - 29, 2020.

Verastem, Inc. (NASDAQ:VSTM) shares closed up 37% to $1.72. The company announced a global licensing agreement with Chugai Pharmaceutical Co., Ltd. Verastem will acquire rights to the RAF/MEK inhibitor CH5126766 (CKI27) from Chugai currently under development for the treatment of KRAS mutant solid tumors. The company will make an upfront payment of $3m and pay royalties to Chugai.

Merck (NYSE: MRK) announced that the FDA approved Keytruda for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Scynexis, Inc. (NASDAQ: SCYX) shares closed up 17% to $1.05 following the release of its second interim efficacy analysis of the Phase 3 open-label FURI trial evaluating oral ibrexafungerp in patients with difficult-to-treat mucocutaneous and invasive fungal infections. 12/21(57%) patients achieved a partial or complete response, in line with data from the first analysis where 11/20 (55%) achieved a response.

Intra-Cellular Therapies, Inc. (NASDAQ:ITCI) shares closed down 15% to $26.34 following the pricing of its public offering of 10m shares at a price of $29.50 per share for gross proceeds of $295m .

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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:

Clovis Oncology, Inc. (CLVS): $1.72; +37%.

Trillium Therapeutics Inc. (TRIL): $1.49; +25%.

Adamas Pharmaceuticals, Inc. (ADMS): $5.11; +23%.

Seneca Biopharma, Inc. (SNCA): $1.40; +21%.

Zafgen, Inc. (ZFGN): $1.49; +18%.

DECLINERS:

BioXcel Therapeutics, Inc. (BTAI): $13.34; -15%.

IVERIC bio, Inc. (ISEE): $7.61; -10%.

Neoleukin Therapeutics, Inc. (NLTX): $9.94; -9%.

AzurRx BioPharma, Inc. (AZRX): $1.20; -9%.

Protalix BioTherapeutics, Inc. (PLX): $4.11; -9%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AGTC – Applied Genetic Technologies Corporation
AAV-based gene therapy
X-linked Retinitis Pigmentosa (XLRP)

Phase 1/2 Phase 1/2 positive dose expansion data released January 9, 2020.
$152.5 million

APLT – Applied Therapeutics Inc.
AT-007
Galactosemia

Phase 2 Phase 2 data January 8, 2019 noted significant plasma galactitol reduction of ~50% (p<0.01). Full data to be presented at the Society for Inherited Metabolic Disorders Annual Meeting, April 26 – 29, 2020.
$904.4 million

BBIO – BridgeBio Pharma Inc.
BBP-009/Patidegib
High-Frequency Basal Cell Carcinoma

Phase 2 Phase 2 trial initiation announced January 8, 2020.
$4.2 billion

BPMC – Blueprint Medicines Corporation
Pralsetinib (BLU-667)
First-line RET-fusion non-small cell lung cancer (NSCLC)

NDA Filing Rolling NDA has commenced. To be completed 1Q 2020.
$3.3 billion

IMMP – Immutep Limited
Eftilagimod Alpha - Active Immunotherapy PAClitaxel (AIPAC)
Metastatic breast cancer (MBC)

Phase 2b Phase 2b PFS data due March 2020.
$68.7 million

IMMP – Immutep Limited
Eftilagimod alpha and Keytruda - TACTI-002
Non-small cell lung cancer; Head and neck cancer

Phase 2 Phase 2 data due 1Q 2020. Noted January 8, 2020 that enrolment in the head and neck cohort has been expanded due to meeting the required number of responses.
$68.7 million

KTOV – Kitov Pharma Ltd.
NT-219
Squamous cell carcinoma of the head & neck (SCCHN)

Phase 1 Phase 1/2 trial to be initiated in 2020.
$15.9 million

MRK – Merck & Company Inc. (new)
Keytruda KN-057
NMIBC Bladder cancer

Approved FDA Approval announced January 8, 2020.
$225.5 billion

MTEM – Molecular Templates Inc.
MT-5111
HER2-positive solid tumors

Phase 1 Phase 1 interim data due 2Q 2020.
$630.4 million

NBRV – Nabriva Therapeutics plc
Contepo
Complicated urinary tract infections (cUTI)

PDUFA CRL issued April 30, 2019. New PDUFA date June 19, 2020.
$135.2 million

SCYX – SCYNEXIS Inc.
SCY-078 - oral (FURI)
Invasive candidiasis

Phase 3 Phase 3 second interim analysis January 8, 2020. Overall response rate 56%.
$95.6 million

SLS – SELLAS Life Sciences Group Inc.
Galinpepimut-S
Acute Myeloid Leukemia (AML)

Phase 3 Phase 3 trial initiation announced January 8, 2020 with interim data due 4Q 2021.
$15.1 million

TYME – Tyme Technologies Inc.
SM-88
Pancreatic cancer

Phase 3 Phase 3 commencement of dosing announced January 8, 2020.
$185.7 million