BPC January 09 update

Applied Genetic Technologies AGTC shares surge 123% on early data; PTLA revenue miss -39% AH

Price and Volume Movers

Applied Genetic Technologies Corporation (Nasdaq: AGTC) shares surged to close up 123% to $9.27 following the release of positive interim six-month data from its ongoing Phase 1/2 trial in X-linked retinitis pigmentosa (XLRP). Patients demonstrated durable improvement in visual function. Improvements were observed in visual sensitivity for four of the evaluable eight patients. All nine patients treated centrally also had stable or improving visual acuity, 78% saw a 5 letter or more improvement.

Blueprint Medicines Corporation (NASDAQ:BPMC) announced that the FDA approved Ayvakit (avapritinib) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) shares are trading up 10% to $48.21 after hours following the release of data from Cohort 3 and longer-term data from Cohort 2 of the ongoing Phase 1/2 study of DTX301, for the treatment of ornithine transcarbamylase (OTC) deficiency. The company noted up to six responders were evident across all nine patients dosed in the trial.

Portola Pharmaceuticals, Inc. (NASDAQ:PTLA) shares are trading down 39% to $15.18 following its report of revenues for 4Q 2019. Andexxa net revenues of $28m for the quarter were well down on expectations.

Aptinyx Inc. (NASDAQ:APTX) announced after hours that it intends to sell shares of its common stock in an underwritten public offering. Shares are trading down 7% to $3.35 after hours.

Matinas BioPharma Holdings, Inc. (NYSE AMER: MTNB) shares are trading down 5% after hours to $1.81 after it also announced that it has commenced an offering of its common stock.

VBI Vaccines Inc. (NASDAQ: VBIV) announced that its Phase 3 Constant trial assessing its hepatitis B (HBV) vaccine, Sci-B-Vac met both the primary and secondary endpoints. Shares closed up 5% to $1.43.

Can-Fite BioPharma Ltd. (NYSE: CANF) shares closed down 20% to $2.78 following news warrants have been exercised to purchase up to an aggregate of 22,278,540 ordinary shares represented by 742,618 American Depositary Shares (ADS) having exercise prices ranging from $12.90 to $78.75 per ADS issued by Can-Fite, at a reduced exercise price of $3.25 per ADS.

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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:

Trillium Therapeutics Inc. (TRIL): $2.89; +94%.

La Jolla Pharmaceutical Company (LJPC): $5.25; +35%.

Capricor Therapeutics, Inc. (CAPR): $1.75; +32%.

Applied Therapeutics, Inc. (APLT): $44.13; +25%.

AzurRx BioPharma, Inc. (AZRX): $1.42; +18%.

DECLINERS:

Regulus Therapeutics Inc. (RGLS): $1.21; -20%.

Zafgen, Inc. (ZFGN): $1.22; -18%.

Solid Biosciences Inc. (SLDB): $3.79; -14%.

Seneca Biopharma, Inc. (SNCA): $1.22; -13%.

Kaleido BioSciences, Inc. (KLDO): $5.42; -11%.

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

BPMC – Blueprint Medicines Corporation
Avapritinib BLU-285
PDGFRA exon 18 mutant Gastrointestinal stromal tumors (GIST)

Approved FDA Approval announced January 9, 2020.
$3.3 billion

CGEN – Compugen Ltd.
COM701
Solid tumors

Phase 1 Phase 1 initial data due 2H 2020.
$406.2 million

EGRX – Eagle Pharmaceuticals Inc.
Ryanodex
Exertional heat stroke (EHS)

NDA Filing NDA resubmission announced January 9, 2020.
$792.7 million

ENTA – Enanta Pharmaceuticals Inc.
EDP-938
Respiratory Syncytial Virus

Phase 2b Phase 2b data due 3Q 2020.
$1.1 billion

MGNX – MacroGenics Inc.
Margetuximab and MGA012 (MAHOGANY)
Gastric cancer

Phase 2 Phase 2/3 initial data due 2H 2020.
$558.1 million

MRNA – Moderna Inc.
mRNA-1647
CMV vaccine (Cytomegalovirus Infection)

Phase 2 Phase 2 interim data due 2H 2020.
$7.2 billion

NTLA – Intellia Therapeutics Inc.
NTLA-2001
transthyretin amyloidosis (ATTR)

Phase 1 IND filing mid-2020 with dosing to commence 2H 2020.
$689.7 million

ORMP – Oramed Pharmaceuticals Inc.
ORMD-0801
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2 data due 1Q 2020.
$91.9 million

OTIC – Otonomy Inc.
OTIVIDEX
Ménière's disease

Phase 3 Additional Phase 3 trial data due 3Q 2020.
$109.2 million

RARE – Ultragenyx Pharmaceutical Inc.
DTX301
Ornithine Transcarbamylase (OTC) Deficiency

Phase 1/2 Phase 1/2 third cohort data released January 9, 2020. 6/9 responders across whole trial. Prophylactic steroid cohort to begin 1H 2020 with data due 2H 2020.
$3.3 billion

REGN – Regeneron Pharmaceuticals Inc.
Garetosmab
Fibrodysplasia ossificans progressiva (FOP)

Phase 2 Phase 2 trial did not meet primary endpoint p=0.07 - January 9, 2020. Patient-reported flare-ups were reduced by 50% (nominal p=0.03).
$39.2 billion

RGNX – REGENXBIO Inc.
RGX-314
Wet age-related macular degeneration (wet AMD)

Phase 2 Pivotal trial to commence 2H 2020.
$1.7 billion

SBBP – Strongbridge Biopharma plc
COR-003 (levoketoconazole) - LOGICS
Endogenous Cushing’s syndrome

Phase 3 Phase 3 top-line data expected 2Q or 3Q 2020.
$164.8 million