BPC November 28 update

Catalyst Pharmaceuticals CPRX FDA approval for Firdapse; ACAD +21% on positive reaction to offering

Price and Volume Movers

Catalyst Pharmaceuticals, Inc. (NASDAQ: CPRX) received approval from the FDA for Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. LEMS is a rare autoimmune disorder that affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients. This is the first FDA approval of a treatment for LEMS. As of 7pm EST shares of the company are still in a trading halt.

ACADIA Pharmaceuticals Inc. (Nasdaq: ACAD) shares closed up 21% to $20.53 following its announcement of the pricing of its public offering of 16,176,471 shares at a price to the public of $17 per share, the same price as Tuesday’s close. Gross proceeds of $275m were noted with an option to raise a further $41.2m. The company had initially indicated Tuesday that it would offer shares to the value of $200m with an option to raise a further $30m. Wednesday’s positive stock move suggests that investors were buoyed by both the pricing and ability to raise more funds than initially indicated.

Celltrion, Inc. and Teva Pharmaceutical Industries Ltd. (NYSE: TEVA) announced that the FDA has approved Truxima, a biosimilar to Rituxan (rituximab) for the treatment of adult patients in three non–Hodgkin’s lymphoma indications.

-

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

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

ADVANCERS:

Vaxart, Inc. (VXRT): $3.20; +20%.

Fortress Biotech, Inc. (FBIO): $1.30; +19%.

Tyme Technologies, Inc. (TYME): $3.20; +18%.

Forty Seven, Inc. (FTSV): $18.38; +13%.

Zymeworks Inc. (ZYME): $13.50; +13%.

DECLINERS:

Arsanis, Inc. (ASNS): $3.06; -26%.

Protagonist Therapeutics, Inc. (PTGX): $6.30; -10%.

Tenax Therapeutics, Inc. (TENX): $2.25; -8%.

Mersana Therapeutics, Inc. (MRSN): $5.40; -8%.

OncoMed Pharmaceuticals, Inc. (OMED): $1.25; -6%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ASND – Ascendis Pharma A/S
TransCon
Hypoparathyroidism

Phase 2 Phase 2 trial to be initiated 1Q 2019 with top-line data due early 2020.
$2.7 billion

ASND – Ascendis Pharma A/S
TransCon hGH
Growth hormone deficiency in children

Phase 3 Phase 3 data due 1Q 2019.
$2.7 billion

ASND – Ascendis Pharma A/S
TransCon CNP
Achondroplasia

Phase 1 Phase 2 trial to be intiated mid-2019.
$2.7 billion

CPRX – Catalyst Pharmaceuticals Inc.
Firdapse
Lambert-Easton Myasthenic Syndrome (LEMS)

Approved FDA approval announced November 28, 2018.
$240.4 million

MBIO – Mustang Bio Inc.
MB-102
Acute myeloid leukemia

Phase 1 Phase 1 data presented at AACR Special Conference on Tumor Immunology and Immunotherapy.
$77.4 million

OBSV – ObsEva SA
Nolasiban (OBE001) - IMPLANT 4
Improving IVF outcomes

Phase 3 Phase 3 initiation announced November 28, 2018.
$645.8 million

PSTI – Pluristem Therapeutics Inc.
PLX-R18
Incomplete hematopoietic recovery following hematopoietic cell transplantation (HCT)

Phase 1 Phase 1 second cohort enrolment is underway.
$98.4 million

PTI – Proteostasis Therapeutics Inc.
PTI-428 + Symdeko
Cystic fibrosis

Phase 2 Phase 2 data due 1Q 2019.
$244.4 million

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

Phase 3 Phase 3 open-label trial planned.
$35.2 million

TEVA – Teva Pharmaceutical Industries Limited American Depositary Shares
TRUXIMA
Rituxan (rituximab) biosimilar

Approved FDA Approval announced November 28, 2018.
$18.8 billion

TLSA – Tiziana Life Sciences plc
Foralumab
NASH (healthy human volunteers)

Phase 1 Phase 1 trial initiation announced November 28, 2018.
$109.1 million

XLRN – Acceleron Pharma Inc.
ACE-083
Facioscapulohumeral muscular dystrophy

Phase 2 Phase 2 Part 2 data due 2H 2019.
$2.1 billion

XLRN – Acceleron Pharma Inc.
ACE-083
Charcot-Marie-Tooth disease (CMT)

Phase 2 Phase 2 Part 2 preliminary data due by the end of 2019.
$2.1 billion