The Daily Biotech Pulse: Novartis Program Placed On Partial Clinical Hold, Adcom Snub For Amag, Amgen Earnings

Here's a roundup of top developments in the biotech space over the last 24 hours.

Scaling The Peaks

(Biotech stocks hitting 52-week highs on Oct. 29)

• Cabaletta Bio Inc CABA
• Edwards Lifesciences Corp EW
• ESSA Pharma Inc EPIX
• GENMAB A/S/S ADR GMAB
• GlaxoSmithKline plc GSK
• Horizon Therapeutics PLC HZNP
• Novo Nordisk A/S NVO
• ResMed Inc. RMD
• Vertex Pharmaceuticals Incorporated VRTX

Down In The Dumps

(Biotech stocks hitting 52-week lows on Oct. 29)

• Acorda Therapeutics Inc ACOR
• Agile Therapeutics Inc AGRX
• Atara Biotherapeutics Inc ATRA
• Auris Medical Holding Ltd EARS
• Axcella Health Inc AXLA
• CytomX Therapeutics Inc CTMX
• Diffusion Pharmaceuticals Inc DFFN
• Eloxx Pharmaceuticals Inc ELOX
• Helius Medical Technologies Inc HSDT
• Hoth Therapeutics Inc HOTH
• MacroGenics Inc MGNX
• Midatech Pharma PLC-ADR MTP
• Onconova Therapeutics Inc ONTX(announced common stock offering)
• Orgenesis Inc ORGS
• Orthofix Medical Inc OFIX (reported disappointing third-quarter results and guidance)
• Tetraphase Pharmaceuticals Inc TTPH

Stocks In Focus

Roche Says U.S., European Regulators Accept Filing of Drug To Treat CNS Inflammatory Disorder

Roche Holdings AG Basel ADR RHHBY said the European Medicines Agency has validated its Marketing Authorization Application for satralizumab for the treatment of adult and adolescent patients with neuromyelitis optica spectrum disorder, granting it Accelerated Assessment. The company said the FDA has also accepted the BLA for satralizumab.

Both decisions are expected in 2020.

Satralizumab is being developed by CHUGAI PHARMACE/ADR CHGCY in which Roche has a majority stake.

Novartis' Spinal Muscular Dystrophy Gene Therapy Placed On Partial Clinical Hold

Novartis AG NVS said the FDA placed a partial hold on clinical trials for intrathecal administration of AVXS-101 following a communication by its AveXis subsidiary to health authorities and clinical trial investigators findings from a small, AveXis-initiated pre-clinical study.

Animal findings from the study showed dorsal root ganglia mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss, Novartis said.

Novartis, however, clarified the partial hold does not impact Zolgensma or AVS-101 intravenous clinical trial.

AveXis is studying AVXS-101 intrathecal administration in patients with spinal muscular dystrophy Type 2.

The stock slipped 1.12% to $87.40 in pre-market trading.

FDA Panel Votes In Favor Of Withdrawing Approval For Amag's Makena To Reduce Preterm Birth

AMAG Pharmaceuticals, Inc. AMAG said FDA's Bone, Reproductive and Urologic Drugs Advisory Committee, which met to discuss its confirmatory trial for Makena, a treatment approved to reduce preterm birth in pregnant women, who have had a previous spontaneous preterm birth, voted 9-7, recommending withdrawal of approval of Makena.

"We are disappointed with the nearly split vote on this key question and we are committed to working with the FDA to identify feasible ways to generate additional efficacy data on Makena while retaining current access to the therapy for at-risk pregnant women," the company said.

Eynenovia Advances Clinical Program For Farsightedness Into Late-Dtage Development

Eyenovia Inc EYEN said it's advancing the development of its MicroLine program for the improvement in near vision in patients with presbyopia into Phase 3 development. The company also said it's deferring development activities for its MicroProst and MicroTears programs, as it's prioritizing MicroLine along with MicroPine and MicroStat programs.

The stock was seen advancing 5.96% to $3.20 in pre-market trading.

Earnings

Amgen, Inc.'s AMGN third-quarter revenues dipped 3% to $5.7 billion and non-GAAP earnings per share slid 1% to $3.66. The EPS exceeded the consensus estimate of $3.53. The company raised its 2019 revenue and EPS guidance.

EXACT Sciences Corporation EXAS said third-quarter revenues jumped 85% to $281.8 million, as Cologuard volume climbed 89%. The net loss narrowed from 37 cents to 31 cents. For 2019, the company expects revenues of $802 million to $810 million, shy of the $810.87 million consensus estimate.

The stock was slipping 8.40% to $83 in pre-market trading.

Cardiovascular Systems Inc CSII reported 14.6% year-over-year revenue growth for its fiscal year first quarter. The net loss, however, widened from 9 cents per share to 17 cents per share, while the consensus estimate had called for a loss of 8 cents per share.

The company reiterated its fiscal-year 2020 outlook.

PTC Therapeutics, Inc.'s PTCT third-quarter revenues climbed about 33% to $71.4 million and net loss per share remained unchanged at $1.16, wider than the 76 cents per share expected by analysts.

The stock slipped 3.97% to $37.75 in after-hours trading.

Verastem Inc VSTM reported revenues of $9 million and a non-GAAP net loss per share of 35 cents per share compared to a loss per share of 26 cents per share last year. The company reiterated its full-year guidance.

The stock was declining 4.80% to $1.19 in pre-market trading.

On The Radar

Adcom Meeting

The Bone, Reproductive and Urologic Drug Advisory Committee will discuss Agile Therapeutics Inc AGRX's NDA for Twirla – a low-dose combo hormonal contraceptive patch – to be used for birth control.

Earnings

United Therapeutics Corporation UTHR (before the market open)
ArQule, Inc. ARQL (before the market open)
Iradimed Corp IRMD (before the market open)
ACADIA Pharmaceuticals Inc. ACAD (after the market close)
Vertex Pharmaceuticals (after the market close)
AtriCure Inc. ATRC (after the market close)
Cerus Corporation CERS (after the market close)
Exelixis, Inc. EXEL (after the market close)
Retrophin Inc RTRX (after the market close)
Masimo Corporation MASI (after the market close)
Immunomedics, Inc. IMMU (after the market close)