BPC December 16 update

​Axsome AXSM positive late-stage data +71%; ​Wave Life WVE falls 55% on discontinuation of DMD trial

Price and Volume Movers

Axsome Therapeutics, Inc. (NASDAQ: AXSM) shares closed up 71% to $79.80 following news its Phase 3 Gemini trial of AXS-05 met the primary endpoint in patients with major depressive disorder (MDD), demonstrating a highly statistically significant reduction in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score compared to placebo, with mean reductions from baseline of 16.6 points for AXS-05 and 11.9 points for placebo (p=0.002). Axsome plans to file a New Drug Application in 2H 2020.

Wave Life Sciences Ltd. (Nasdaq: WVE) announced that it will discontinue development of suvodirsen for patients with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 51 skipping, based on its interim analysis of the Phase 1 open-label extension (OLE) trial. Shares closed down 55% to $16.79.

Novartis (NYSE: NVS) announced that it did not meet the primary endpoint in its Phase 3 Luster trials of its DP2 antagonist, fevipiprant (QAW039), in patients with asthma. Gossamer Bio Inc (NASDAQ: GOSS) which also has a DP2 antagonist, GB-001, saw its shares fall in response to the data, closing down 37% to $15.96. Data from its asthma trial are due next quarter.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) shares closed up 4% to $83.50 on news its Phase 3 trial of vosoritide in children with achondroplasia, the most common form of disproportionate short stature, met the primary endpoint, with a placebo-adjusted change from baseline in growth velocity after one year of treatment with vosoritide of 1.6 cm/yr (p<0.0001).

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) announced that following its pre-New Drug Application (NDA) meeting with the FDA of maralixibat for the treatment of pruritus associated with Alagille syndrome, it intends to file a rolling NDA in 3Q 2020. Shares closed up 111% to $17.28.

BeiGene, Ltd. (NASDAQ: BGNE) announced that it did not meet the primary endpoint of superiority in complete response (CR) in its Phase 3 Aspen trial comparing Brukinsa (zanubrutinib) to ibrutinib for the treatment of Waldenström’s macroglobulinemia (WM). Shares closed down 6% to $165.09.

Roche (OTCQX:RHHBY) and Spark Therapeutics, Inc. (NASDAQ:ONCE) announced that the UK Competition and Markets Authority has unconditionally cleared Roche's pending acquisition of Spark pursuant for $114.50 per share.

Aptose Biosciences Inc. (NASDAQ: APTO) announced after hours that it has commenced an underwritten public offering of its common shares.

MEI Pharma, Inc. (Nasdaq: MEIP) also announced it plans to offer shares of its common stock in an underwritten public offering. Shares are trading down 24% after hours to $1.75.

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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:

VistaGen Therapeutics, Inc. (VTGN): $1.01; +44%.

Clearside Biomedical, Inc. (CLSD): $2.22; +23%.

La Jolla Pharmaceutical Company (LJPC): $4.07; +21%.

Oncolytics Biotech Inc. (ONCY): $1.34; +21%.

BioXcel Therapeutics, Inc. (BTAI): $8.19; +21%.

DECLINERS:

Mereo BioPharma Group plc (MREO): $1.17; -20%.

Capricor Therapeutics, Inc. (CAPR): $1.10; -15%.

Brickell Biotech, Inc. (BBI): $1.41; -15%.

Matinas BioPharma Holdings, Inc. (MTNB): $1.61; -13%.

DiaMedica Therapeutics Inc. (DMAC): $3.30; -12%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AKRO – Akero Therapeutics Inc.
AKR-001
Nonalcoholic steatohepatitis (NASH)

Phase 2a Phase 2a data due 1Q 2020 (MRI‐PDFF).
$689.7 million

AVDL – Avadel Pharmaceuticals plc
AV001
Neonate Patients Requiring Total Parental Nutrition (TPN)

Approved FDA Approval announced December 16, 2019.
$399.8 million

AXSM – Axsome Therapeutics Inc.
AXS-05 -GEMINI
Major Depressive Disorder

NDA Filing Phase 3 data released December 16, 2019 met primary endpoint. NDA filing due 2H 2020.
$3.3 billion

BGNE – BeiGene Ltd.
Zanubrutinib (BGB-3111) - ASPEN
Waldenström’s macroglobulinemia

Phase 3 Phase 3 top-line data did not meet primary endpoint - December 16, 2019.
$12.7 billion

BMRN – BioMarin Pharmaceutical Inc.
Vosoritide
Achondroplasia

Phase 3 Phase 3 top-line data met primary endpoint - December 16, 2019.
$16.7 billion

DCPH – Deciphera Pharmaceuticals Inc.
Ripretinib DCC-2618
Gastrointestinal Stromal Tumors (GIST) - fourth-line

PDUFA priority review PDUFA date under priority review August 13, 2020.
$2.7 billion

EIGR – Eiger BioPharmaceuticals Inc.
Lonafarnib
Hutchinson-Gilford Progeria Syndrome (HGPS)

NDA Filing NDA rolling submission commenced December 16, 2019. To be completed 1Q 2020.
$276.3 million

GILD – Gilead Sciences Inc.
Cilofexor (GS-9674) and firsocostat (GS-0976) - ATLAS
Nonalcoholic steatohepatitis (NASH)

Phase 2 Phase 2 trial did not meet primary endpoint - December 16, 2019.
$92.2 billion

MIRM – Mirum Pharmaceuticals Inc.
Maralixibat
Alagille Syndrome (ALGS)

NDA Filing Rolling NDA to be filed 3Q 2020.
$461.1 million

NVS – Novartis AG
QAW039 (fevipiprant) - LUSTER-1
Asthma

Phase 3 Phase 3 trial did not meet primary endpoint - December 16, 2019.
$204 billion

PFE – Pfizer Inc.
XTANDI (ARCHES)
Metastatic hormone sensitive prostate cancer

Approved FDA Approval announced December 16, 2019.
$191.9 billion

RGLS – Regulus Therapeutics Inc.
RGLS4326
Autosomal dominant polycystic kidney disease (ADPKD)

Phase 1 Phase 1 initiation of dosing of the second cohort announced February 13, 2020. To be completed mid-2020 with data thereafter.
$16.7 million

WVE – Wave Life Sciences Ltd.
Suvodirsen (WVE-210201)
DMD Exon 51

Phase 1 Phase 1 development to be discontinued following interim analysis - December 16, 2019.
$282.2 million