The Daily Biotech Pulse: Pfizer Earnings, Roche's Spinal Muscular Dystrophy Treatment, Moderna COVID-19 Vaccine Update

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 April 27)

• AstraZeneca plc AZN
• Alnylam Pharmaceuticals, Inc. ALNY
• Arcturus Therapeutics Ltd ARCT
• Avadel Pharmaceuticals PLC AVDL
• BioMarin Pharmaceutical Inc. BMRN
• Bio-Rad Laboratories, Inc. BIO
• BioSig Technologies Inc BSGM
• Black Diamond Therapeutics Inc BDTX
• Cytosorbents Corp CTSO
• DexCom, Inc. DXCM
• Erytech Pharma SA ERYP
• Exelixis, Inc. EXEL
• Halozyme Therapeutics, Inc. HALO
• I-Mab ADR IMAB
• Imara Inc IMRA
• Incyte Corporation INCY
• Kala Pharmaceuticals Inc KALA
• Keros Therapeutics Inc KROS
• Masimo Corporation MASI
• Novo Nordisk A/S NVO
• OraSure Technologies, Inc. OSUR
• Quidel Corporation QDEL
• REDHILL BIOPHAR/S ADR RDHL
• Repligen Corporation RGEN
• Seattle Genetics, Inc. SGEN
• Translate Bio Inc TBIO
• Ultragenyx Pharmaceutical Inc RARE
• Vaxart Inc VXRT
• Vermillion, Inc. VRML
• West Pharmaceutical Services Inc. WST
• Y-mAbs Therapeutics, Inc YMAB
• Zentalis Pharmaceuticals Inc ZNTL
• Zynex Inc. ZYXI

None of the Nasdaq- or NYSE-listed biotech/medical device/diagnostics company hit 52-week lows Monday.

Stocks In Focus

Syndax Presents Positive Pre-clinical & Clinical Data For Blood Cancer Drug

Syndax Pharmaceuticals Inc SNDX presented positive preclinical and initial clinical data for SNDX-5613, its potent, highly selective oral menin inhibitor, with the data suggesting the investigational asset can induce response in patients with genetically-defined acute leukemias.

"Notably, clinical activity was achieved rapidly after a single, 28-day cycle, a highly encouraging sign in this population of patients who face a particularly poor prognosis with few effective treatment options," said Briggs Morrison, CEO of Syndax.

The company said it will present additional findings from this trial in the fourth quarter.

It also said SNDX-5613 was recently granted Orphan Drug Designation for the treatment of adult and pediatric acute myeloid leukemia.

In after-hours trading, Syndax shares added 20.33% to $13.85.

Moderna Files IND Application For Phase 2 Study of Coronavirus Vaccine

Moderna Inc MRNA said it has submitted an IND application to the FDA for its mRNA vaccine candidate mRNA-1273 against the novel coronavirus – SARS-CoV-2 – for evaluating it in a Phase and late-stage studies if supported by the Phase 1 study conducted by the National Institute of Allergy and Infectious Diseases.

The company expects the study to begin in the second quarter.

The stock gained 7.18% to $51.50 in after-hours trading.

Related Link: A Deep Dive On Coronavirus Vaccine Plays Moderna, Inovio

Roche Reports Positive Results For Pivotal Study of its Spinal Muscular Dystrophy Treatment

Roche Holdings AG's Basel RHHBY presented data from FIREFISH Part 2 – a pivotal global study that is evaluating its risdiplam in infants, ages 1-7 months, with symptomatic Type 1 spinal muscular atrophy, which showed the primary endpoint was met. The company noted that 29% of infants could sit without support for five seconds by month 12, as assessed by the Gross Motor Scale of Bayley Scales of Infant and Toddler Development Third Edition.

Additionally, the company noted that 43.9% of infants were able to hold their head upright, 31.7% were able to roll to the side and 4.9% infants were able to stand with support.

Risdiplam is an orally administered survival motor neuron-2 splicing modifier for SMA.

Roche is leading the development of risdiplam and is working with the SMA Foundation and PTC Therapeutics, Inc. PTCT on this.

The regulatory application for the investigational asset was accepted with priority designation by the FDA, with an expected PDUFA date of Aug. 24.

Separately, Roche also released new analyses of Phase 3 OPERA I and OPERA II studies as well as open-label extensions, of its Ocrevus, which showed the treatment reduced the risk of disease and disability progression in relapsing multiple sclerosis and primary progressive multiple sclerosis.

Avadel to Offer $100M Worth of ADSs

Avadel Pharmaceuticals PLC AVDL said it commenced an underwritten public offering of $100 million shares, in the form of ADSs. All the ADSs are being offered by Avadel.

The stock slipped 0.90% to $11.05 in after-hours trading.

Earnings

Novartis AG's NVS first-quarter net sales from continuing operations rose 11% to $12.28 billion, EPS climbed 19% to 96 cents and core EPS increased 29% to $1.56, ahead of the $1.36 per share consensus estimate. The company expects 2020 sales to grow mid- to high-single digits and core operating income to increase in high-single to low-double digits.

The stock added 0.90% to $90.40 in after-hours trading.

On The Radar

Clinical Readouts

Iovance Biotherapeutics Inc IOVA is scheduled to present at the American Association for Cancer Research, or AACR, 2020 Virtual Annual Meeting Phase 1 data for tumor-infiltrating lymphocytes in non-small cell lung cancer.

Earnings

Merck & Co., Inc. MRK (Q1 Revenues of $12.1 billion, up 11%, and non-GAAP EPS $1.50 Vs. consensus of $11.46 billion and $1.34; lowered full-year guidance)

Pfizer Inc. PFE (Q1 Revenues of $12 billion, down 7%, and adjusted EPS of 80 cents Vs. consensus of $11.87 billion and 73 cents; reaffirmed 2020 guidance)