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.


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

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


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%.


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
X-linked Retinitis Pigmentosa (XLRP)

Phase 1/2 Phase 1/2 further data due 2H 2020.
$152 million

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

Approved FDA Approval announced January 9, 2020.
$4.2 billion

CGEN – Compugen Ltd.
COM701 and Opdivo
Solid tumors

Phase 1 Phase 1 presentation at AACR April 27, 2020 noted disease control rates of 69% (11/16) for monotherapy and 75% (9/12) for combination arm. Initial data from monotherapy expansion cohort due 1H 2021.
$1.4 billion

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

CRL CRL announced August 10, 2020.
$597.2 million

ENTA – Enanta Pharmaceuticals Inc.
Respiratory Syncytial Virus

Phase 2b Phase 2b data due 3Q 2021.
$962 million

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

Phase 2 Phase 2/3 initial data due 2H 2020.
$1.5 billion

MRNA – Moderna Inc.
CMV vaccine (Cytomegalovirus Infection)

Phase 2 Phase 2 first interim analysis is expected 3Q 2020
$28.4 billion

NTLA – Intellia Therapeutics Inc.
transthyretin amyloidosis (ATTR)

Phase 1 Phase 1 trial to commence dosing 2H 2020.
$1.2 billion

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

Phase 2 Phase 2 interim data presented at ADA 2020 Scientific Sessions - mean 6.9% reduction in liver fat content.
$93.4 million

OTIC – Otonomy Inc.
Ménière's disease

Phase 3 Phase 3 data due 1Q 2021.
$160.6 million

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

Phase 1/2 Phase 1/2 data from Cohort 4 due by the end of 2020. Phase 3 trial to commence 1H 2021.
$5.2 billion

REGN – Regeneron Pharmaceuticals Inc.
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).
$64.6 billion

Wet AMD using using subretinal delivery

Phase 2 Phase 2 trial to commence enrollment 3Q 2020 with interim data from first cohort due by the end of 2020.
$1.3 billion

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

Phase 3 Phase 3 top-line data expected September 2020.
$222.6 million