BPC January 31 update

​Fortress Biotech FBIO surges 40% on partnership with Alexion ALXN; AVEO slides 60% on tivozanib NDA filing roadblock

Price and Volume Movers

Fortress Biotech, Inc. (NASDAQ: FBIO) shares closed up 40% to $2.07. The company announced that Caelum Biosciences, founded by Fortress, has signed an agreement with Alexion Pharmaceuticals, Inc (NASDAQ: ALXN) to advance the development of CAEL-101 for light chain (AL) amyloidosis. Alexion will make payments to Caelum totalling up to $60m. The collaboration also provides for potential additional payments of up to $500m.  Alexion will acquire a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 2 data.

AVEO Oncology (NASDAQ: AVEO) shares slumped to close down 60% to $0.70 following news the FDA has recommended the company not to submit its New Drug Application (NDA) for tivozanib with the preliminary overall survival (OS) results from the Phase 3 TIVO-3 trial. The company now plans to make a NDA filing decision following the availability of more mature OS results. Further interim OS data are due 4Q 2019.

Aquestive Therapeutics, Inc. (NASDAQ: AQST) announced its licensee, Sunovion Pharmaceuticals Inc., received a Complete Response Letter (CRL) from the FDA for the New Drug Application (NDA) for apomorphine sublingual film (APL-130277), for the treatment of OFF episodes (the re-emergence or worsening of Parkinson's symptoms otherwise controlled by medications) experienced by people with Parkinson's disease (PD). The FDA requested additional information and analyses, but no new clinical studies are required. Shares closed down 14% to $6.13.

Leap Therapeutics, Inc. (Nasdaq:LPTX) announced it has commenced an underwritten public offering of its common stock and warrants to purchase common stock. Shares are trading down 15% after hours to $1.66 in addition to a 10% loss during normal trading hours.

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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:

SCYNEXIS, Inc. (SCYX): $1.02; +42%.

Novus Therapeutics, Inc. (NVUS): $4.30; +41%.

Onconova Therapeutics, Inc. (ONTX): $3.10; +27%.

GTx, Inc. (GTXI): $1.09; +21%.

AC Immune SA (ACIU): $4.12; +13%.

DECLINERS:

Achaogen, Inc. (AKAO): $1.51; -12%.

Ovid Therapeutics Inc. (OVID): $2.83; -11%.

Aerpio Pharmaceuticals, Inc. (ARPO): $3.48; -10%.

Selecta Biosciences, Inc. (SELB): $1.57; -10%.

Allena Pharmaceuticals, Inc. (ALNA): $7.01; -7%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ABEO – Abeona Therapeutics Inc.
ABO-102
Sanfilippo syndrome type A (MPS IIIA)

Phase 1/2 Phase 1/2 safety and tolerability data to be presented at WORLDSymposium February 6, 2019.
$207.5 million

ACHN – Achillion Pharmaceuticals Inc.
ACH-5228
Healthy volunteers

Phase 1 Phase 1 multiple ascending dose trial to be completed 2019.
$342.7 million

AQST – Aquestive Therapeutics Inc.
APL-130277
Parkinson’s Disease with motor fluctuations

CRL CRL issued January 30, 2019 through partner Sunovion (private company).
$103.6 million

AVEO – AVEO Pharmaceuticals Inc.
TIVO-3 - tivozanib
Third line treatment of patients with renal cell cancer

Phase 3 Noted January 31, 2019 that FDA recommended against filing an NDA with current OS data. Additional interim OS analysis due August 2019 with further OS data 4Q 2019.
$117.4 million

CBIO – Catalyst Biosciences Inc.
Marzeptacog alfa
Hemophilia

Phase 2 Phase 2 final data released July 7, 2019. Primary endpoint met.
$100.4 million

CELG – Celgene Corporation
liso-cel (JCAR017) - TRANSCEND OUTREACH
Relapsed and/or refractory diffuse large B-cell lymphoma (DLBCL)

Phase 2 Phase 2 trial initiated 4Q 2018.
$64.7 billion

CELG – Celgene Corporation
OTEZLA - ADVANCE
Plaque psoriasis

Phase 3 Phase 3 trial is initiating.
$64.7 billion

ENTX – Entera Bio Ltd.
EB613
Osteoporosis

Phase 2 Phase 2 trial initiation announced July 2, 2019.
$37.7 million

EXEL – Exelixis Inc.
IMspire170 - cobimetinib and atezolizumab
First-line BRAF wild-type metastatic or unresectable locally advanced melanoma

Phase 3 Phase 3 data did not meet primary endpoint - June 20, 2019.
$6.5 billion

EXEL – Exelixis Inc.
IMspire150 - cobimetinib, atezolizumab and vemurafenib - TRILOGY
BRAF V600 mutation-positive metastatic or unresectable locally advanced melanoma

Phase 3 Phase 3 data due 2019.
$6.5 billion

LLY – Eli Lilly and Company
(MK-3475-189/KEYNOTE-189)
First Line Metastatic Non-squamous Non-small Cell Lung Cancer (NSCLC)

Approved FDA label expansion announced January 31, 2019.
$105.3 billion

PTCT – PTC Therapeutics Inc.
RG7916 - SUNFISH
Spinal Muscular Atrophy (SMA)

NDA Filing NDA filing due 2H 2019.
$2.5 billion

RHHBY – Roche Holding Ltd ADR (Sponsored)
Tecentriq + chemo - IMpassion 031
Neoadjuvant triple negative breast cancer

Phase 3 Phase 3 interim analysis passed.
$229.2 billion

RHHBY – Roche Holding Ltd ADR (Sponsored)
Tecentriq + Avastin - IMbrave 150
Hepatocellular carcinoma

Phase 3 Phase 3 data due 2019.
$229.2 billion