BPC August 07 update

​Biogen BIIB Alzheimer’s disease drug awarded priority review +10%; PTCT Roche FDA approval for spinal muscular atrophy

Price and Volume Movers

Biogen (Nasdaq: BIIB) and Eisai, Co., Ltd. announced that FDA has accepted their Biologics License Application (BLA) for aducanumab for Alzheimer’s disease with a PDUFA date under Priority Review of March 7, 2021. Shares closed up 10% to $305.71.

PTC Therapeutics, Inc. (NASDAQ:PTCT) and Roche (OTCQX:RHHBY) announced that the FDA has approved Evrysdi (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older. The approval decision came ahead of the assigned PDUFA date of August 24.

Kazia Therapeutics Limited (NASDAQ:KZIA) shares closed up 50% to $6.40. Shares rose reacting to news that the company was awarded Rare Pediatric Disease Designation (RPDD) by the FDA for paxalisib (formerly GDC-0084) for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG). RPDD is granted to drugs in development for life-threatening diseases which primarily affect children that have an incidence of less than 200,000 new cases per annum in the United States. Such companies may request a Priority Review Voucher (PRV) at the time of a marketing application to the FDA, shortening the review from 12 months to 6 months. It should be noted, however, that paxalisib is in the very early stage of development, with Phase 1 data due later this year.

Adamas Pharmaceuticals, Inc. (NASDAQ:ADMS) shares closed up 45% to $3.78 following better than expected second quarter earnings. Revenue from its approved drug Gocovri were $18m for the quarter, an increase of 41% as compared to the same period last year.

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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:

Acer Therapeutics Inc. (ACER): $5.41; +21%.

SIGA Technologies, Inc. (SIGA): $7.68; +18%.

Zosano Pharma Corporation (ZSAN): $1.56; +15%.

Kura Oncology, Inc. (KURA): $20.41; +14%.

Agenus Inc. (AGEN): $3.75; +13%.

DECLINERS:

ADiTx Therapeutics, Inc. (ADTX): $5.85; -23%.

Capricor Therapeutics, Inc. (CAPR): $6.53; -17%.

GW Pharmaceuticals plc (GWPH): $109.37; -17%.

CohBar, Inc. (CWBR): $1.76; -15%.

Marinus Pharmaceuticals, Inc. (MRNS): $1.79; -14%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ABUS – Arbutus Biopharma Corporation
AB-729
Hepatitis B (HBV)

Phase 1 Phase 1a/b data released September 15, 2020 noted favorable safety and tolerability profile.
$264 million

ACRS – Aclaris Therapeutics Inc.
ATI-450
Rheumatoid arthritis (RA)

Phase 2a Phase 2a data due 1H 2021.
$108.4 million

ALT – Altimmune Inc.
NASOSHIELD
Anthrax

Phase 1b Phase 1b data due 4Q 2020.
$464.7 million

BIIB – Biogen Inc.
Aducanumab
Alzheimer’s disease

PDUFA priority review PDUFA date under priority review March 7, 2021.
$43.5 billion

CRNX – Crinetics Pharmaceuticals Inc.
CRN00808 - ACROBAT EVOLVE
Acromegaly

Phase 2 Phase 2 data due 4Q 2020.
$518.6 million

HTBX – Heat Biologics Inc.
COVID vaccine
COVID-19 vaccine

Phase 1 Phase 1 trial planned.
$220.6 million

JNCE – Jounce Therapeutics Inc.
Vopratelimab and JTX-4014 - SELECT
Second-line non-small cell lung cancer (NSCLC) patients

Phase 2 Phase 2 data due 2021.
$337 million

JNCE – Jounce Therapeutics Inc.
Vopratelimab and Ipilimumab - EMERGE
Non-small cell lung cancer and bladder cancer

Phase 2 Phase 2 interim analysis due early 2021.
$337 million

KZIA – Kazia Therapeutics Limited
Paxalisib (GDC-0084)
Diffuse Intrinsic Pontine Glioma

Phase 1 Phase 1 initial efficacy data due 2H 2020.
$49.1 million

OCUL – Ocular Therapeutix Inc.
OTX-TKI
Wet Age-related Macular Degeneration (AMD)

Phase 1 Phase 1 update due fall of 2020.
$581.7 million

PTCT – PTC Therapeutics Inc.
Risdiplam (RG7916)
Spinal Muscular Atrophy (SMA) Type 1

Approved FDA Approval announced August 7, 2020.
$3.2 billion

PULM – Pulmatrix Inc.
PUR1800
Acute exacerbation of chronic obstructive pulmonary disease (AECOPD)

Phase 1b Phase 1b trial to commence 2H 2020.
$43.7 million

REPL – Replimune Group Inc.
RP2 and nivolumab
Solid tumors

Phase 1 Phase 1 initial data due by the end of 2020.
$999.2 million

SRRK – Scholar Rock Holding Corporation
SRK-015 - TOPAZ
Spinal muscular atrophy (SMA)

Phase 2 Phase 2 interim efficacy data due 4Q 2020.
$542.8 million