BPC February 07 update

Solid Biosciences SLDB and Sangamo SGMO shares slide following respective gene therapy updates

Price and Volume Movers

Evolus, Inc. (NASDAQ: EOLS) shares closed up 20% to $28.91 for a cumulative five-day gain of 77% following last Friday’s announcement of the FDA approval of Jeuveau for the improvement in the appearance of glabellar lines. 

Solid Biosciences (Nasdaq: SLDB) shares plunged following the release of preliminary data from IGNITE DMD, its Phase 1/2 dose-ascending trial evaluating SGT-001 microdystrophin gene transfer for the treatment of Duchenne muscular dystrophy (DMD). Initial three-month biopsy data showed low levels of microdystrophin protein expression. Two of the three patients showed microdystrophin undetectable via western blot. The other patient had microdystrophin detected via western blot but below the 5% level of quantification. Shares closed the session down 68% to $7.19.

Sangamo Therapeutics, Inc. (NASDAQ: SGMO) shares slid, closing down 31% to $8.31 following the report of preliminary findings from its Phase 1/2 Champions trial evaluating SB-913, for the treatment of patients with Mucopolysaccharidosis Type II (MPS II). MPS II, is a genetic disorder caused by a deficiency of iduronate-2-sulfatase (IDS), an enzyme required to break down or recycle the toxic buildup of glycosaminoglycans (GAGs). Without IDS enzyme activity, GAGs accumulate in cells throughout the body, leading to widespread tissue and organ damage. The goal of SB-913 treatment is to enable a patient's liver to produce a continuous supply of the enzyme. However, as their CEO, Sandy Macrae noted“you can see a small increase in the enzyme but we don’t think that would be enough to make a difference.” 

Incyte (Nasdaq:INCY) announced the FDA has extended the review period for the supplemental New Drug Application (sNDA) for ruxolitinib (Jakafi) for the treatment of patients with acute graft-versus-host disease (GVHD) who have had an inadequate response to corticosteroids. The new PDUFA target action date is May 24, 2019. The FDA extended the action date to allow time to review additional data submitted by Incyte in response to the FDA’s information requests.  

Axovant Sciences (NASDAQ: AXON) noted in it earnings release that it plans to change its name to Axovant Gene Therapies and its stock symbol to “AXGT”. Common stock will begin trading under the new ticker at the opening of trading on February 14, 2019. The former ticker symbol “AXON” will remain effective through market close on February 13, 2019.

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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:

OHR Pharmaceutical, Inc. (OHRP): $2.77; +33%.

Melinta Therapeutics, Inc. (MLNT): $1.85; +24%.

Correvio Pharma Corp. (CORV): $4.01; +19%.

Gemphire Therapeutics Inc. (GEMP): $1.27; +18%.

Atossa Genetics Inc. (ATOS): $1.70; +14%.

DECLINERS:

Avadel Pharmaceuticals plc (AVDL): $2.53; -22%.

Trillium Therapeutics Inc. (TRIL): $1.28; -14%.

AzurRx BioPharma, Inc. (AZRX): $2.07; -13%.

Vaxart, Inc. (VXRT): $2.00; -12%.

MacroGenics, Inc. (MGNX): $22.87; -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 dose escalation data due 3Q 2019. Expansion data due 4Q 2019.
$82.7 million

AGTC – Applied Genetic Technologies Corporation
AAV Gene Therapy
CNGB3 Achromatopsia

Phase 1/2 Phase 1/2 six-month data for the dose escalation portion due 2H 2019.
$82.7 million

ALNY – Alnylam Pharmaceuticals Inc.
Lumasiran (ALN-GO1) ILLUMINATE-A
Primary Hyperoxaluria Type 1 (PH1)

Phase 3 Phase 3 data due late-2019.
$8.7 billion

ALNY – Alnylam Pharmaceuticals Inc.
Givosiran
Acute hepatic porphyrias

NDA Filing Phase 3 trial met primary endpoint. Rolling NDA has been initiated.
$8.7 billion

ALNY – Alnylam Pharmaceuticals Inc.
ALN-HBV02 (VIR-2218))
Chronic hepatitis B virus (HBV)

Phase 1/2 Phase 1/2 data due 2019.
$8.7 billion

ALNY – Alnylam Pharmaceuticals Inc.
ALN-CC5 (cemdisiran)
IgA nephropathy

Phase 2 Phase 2 trial to be initiated.
$8.7 billion

INCY – Incyte Corporation
Ruxolitinib
Graft versus host disease

PDUFA priority review PDUFA date under priority review extended by three months to May 24, 2019.
$15.8 billion

LXRX – Lexicon Pharmaceuticals Inc.
Sotagliflozin
Type 2 Diabetes

Phase 3 Phase 3 data due 4Q 2019.
$593 million

MCRB – Seres Therapeutics Inc.
SER-109 - ECOSPOR III
Recurrent C. Difficile infection

Phase 3 Phase 3 trial initiation announced June 12, 2017. Noted February 7, 2019 that the trial continues to enrol but is evaluating modification of the trial to expedite clinical results.
$283.2 million

MEIP – MEI Pharma Inc.
ME-344
HER2-negative Breast Cancer

Phase 1 Phase 1 data due mid-2019.
$207.8 million

MEIP – MEI Pharma Inc.
ME-401
Relapsed/refractory Follicular Lymphoma/Chronic Lymphocytic Leukemia (CLL)

Phase 1b Phase 2 trial to commence 1Q 2019.
$207.8 million

MEIP – MEI Pharma Inc.
Voruciclib
B-Cell Malignancies

Phase 1 Phase 1 data due 2019.
$207.8 million

MEIP – MEI Pharma Inc.
Pracinostat in combination with Vidaza
High-risk Myelodysplastic Syndrome (MDS)

Phase 2 Phase 2 updated data due by the end of 2019.
$207.8 million

MYOV – Myovant Sciences Ltd.
Relugolix - LIBERTY 1 and LIBERTY 2
Menstrual bleeding associated with uterine fibroids

Phase 3 Phase 3 LIBERTY 1 top-line data due 2Q 2019. LIBERTY 2 data due 3Q 2019.
$1.3 billion

MYOV – Myovant Sciences Ltd.
Relugolix - HERO
Advanced prostate cancer

Phase 3 Phase 3 data due 4Q 2019.
$1.3 billion

MYOV – Myovant Sciences Ltd.
Relugolix - SPIRIT 1
Endometriosis-associated pain

Phase 3 Phase 3 data 1Q 2020.
$1.3 billion

MYOV – Myovant Sciences Ltd.
Relugolix - SPIRIT 2
Endometriosis-associated pain

Phase 3 Phase 3 data 1Q 2020.
$1.3 billion

PULM – Pulmatrix Inc.
Pulmazole (PUR1900)
Allergic bronchopulmonary aspergillosis in patients with asthma.

Phase 2a Phase 2 trial to be initiated 2Q 2019 with data due 2Q 2020.
$11.7 million

SGEN – Seattle Genetics Inc.
Enfortumab vedotin - EV201
Urothelial cancer

BLA Filing Phase 2 pivotal data March 28, 2019 noted 44% ORR. BLA to be filed later in 2019.
$11.1 billion

SGEN – Seattle Genetics Inc.
Tisotumab Vedotin (innovaTV 204 )
Cervical Cancer

Phase 2 Phase 2 completion of enrolment announced March 29, 2019.
$11.1 billion

SGEN – Seattle Genetics Inc.
(Tucatinib ONT-380) + Herceptin and Xeloda - HER2CLIMB trial
HER2+ Metastatic Breast Cancer (MBC)

Phase 2 Phase 2 data due 2019.
$11.1 billion

SGMO – Sangamo Therapeutics Inc.
SB-318
MPS Type 1

Phase 1/2 Phase 1/2 longer-term safety and biochemical data due in 2019.
$1.3 billion

SGMO – Sangamo Therapeutics Inc.
SB-913 - CHAMPIONS
MPS Type 2

Phase 1/2 Phase 1/2 updates during 2019.
$1.3 billion

SLDB – Solid Biosciences Inc.
SGT-001
Duchenne Muscular Dystrophy

Phase 1/2 Phase 1/2 negative initial data released February 7, 2019. Additional data due later in 2019.
$326.2 million

SNY – Sanofi
Olipudase alfa
Acid sphingomyelinase deficiency (ASMD)

Phase 2/3 Phase 3 data due 4Q 2019.
$102.9 billion

SNY – Sanofi
Sutimlimab (BIVV009) - Cardinal
Cold agglutinin disease

Phase 3 Phase 3 data due 4Q 2019.
$102.9 billion

SNY – Sanofi
Sutimlimab (BIVV009) - Cadenza
Cold agglutinin disease

Phase 3 Phase 3 data due 4Q 2019.
$102.9 billion

SNY – Sanofi
Isatuximab
Refractory Multiple Myeloma

BLA Filing Phase 3 data February 5, 2019 met primary endpoint. BLA filing 2Q 2019.
$102.9 billion

VBIV – VBI Vaccines Inc.
VBI-1901
Recurrent glioblastoma multiforme (rGBM)

Phase 1/2 Phase 1/2a six-month PFS and OS data due 1H 2019.
$126.2 million