The Daily Biotech Pulse: Zolgensma Data, Roche Q1 Results, Brainsway IPO

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 April 16)

• Eidos Therapeutics Inc EIDX(reported fourth quarter results)
• Biohaven Pharmaceutical Holding Co Ltd BHVN
• Fulgent Genetics Inc FLGT

Down In The Dumps

(Biotech stocks hitting 52-week lows on April 16)

• ABIOMED, Inc. ABMD
• Aethlon Medical, Inc. AEMD
• Guardion Health Sciences Inc GHSI
• Meridian Bioscience, Inc. VIVO
• Vaxart IncVXRT
• Wave Life Sciences Ltd WVE (reported additional Phase 1 results for its DMD candidate and the design for a Phase 2/3 trial)

See Also: The Week Ahead In Biotech: Conferences, Clinical Trial Readouts, Earnings And IPOs

Stock In Focus

Novartis' Spinal Muscular Atrophy Gene Therapy Shows Efficacy and Safety In Late-stage Trial

Novartis AG NVS's AveXis unit released interim data from the Phase 3 trial, dubbed STRIVE, of Zolgensma, which is being evaluated for spinal muscular dystrophy Type 1, which showed prolonged event-free survival, an early and rapid increase in CHOP-INTEND scores and significant milestone achievement compared to untreated natural history. The data was consistent with the readout from the pivotal START trial.

The STRIVE trial is an open label single-arm, single-dose, multi-center trial that evaluated safety and efficacy of one-time intravenous infusion of Zolgensma in patients with SMA Type 1 who are less than six months of age at the time of gene therapy.

As of September, 21 of the 22 or 95 percent of patients were alive and event-free. The median age was 9.5 months, with 6 of the 7 patients who could have achieved 10.5 months of age or older surviving event free. However, natural history indicated 50 percent of the babies with SMA Type 1 will not survive or will require permanent ventilation by the time they reach 10.5 months of age.

The results were presented at the 2019 Muscular Dystrophy Association Clinical and Scientific Conference in Orlando, Florida.

Natera To Offer $100M Worth Of Shares

Genetic testing company Natera Inc NTRA announced a proposed follow-on public offering of $100 million worth of shares. The company, however, said the offering is subject to market and other conditions and there can be no assurance regarding the completion as well as the size of the offering.

The stock fell 6.69 percent to $19.25 in after-hours trading.

Adverum To Present New Preclinical Data On Gene Therapy Candidate

Adverum Biotechnologies Inc ADVM said it will present at the American Society of Gene and Cell Therapy conference new preclinical data based on animal testing of its single injection 2x10^12 vg of ADVM-022 gene therapy, which demonstrated long-term durability and safety.

The gene therapy candidate provided durable expression of aflibercept with no deleterious effects on retina structure and function observed out to 30 months, the company said,

At the Association for Research in Vision and Ophthalmology conference, the company said it will present a poster on preclinical data characterizing immunological response following sequential intravitreal administration of AAV2.7m8 gene therapy and its degree of effect on transduction in the contralateral eye.

Roche Q1 Sales Rise, Raises Guidance

Roche Holdings AG Basel ADR RHHBY reported 8-percent sales growth for the first quarter, as volume growth more than offset the price declines. The company raised its 2019 sales growth guidance from low-single digit to mid-single digit growth to mid-single digit growth.

On The Radar

Earnings

Abbott LaboratoriesABT Q1 Adj. EPS $0.63 Beats $0.61 Estimate, Sales $7.5B Beat $7.47B Estimate

IPO

Brainsway, a maker of medical devices that use magnetic stimulation to treat depression and obsessive-compulsive disorder, priced its 2.5-million initial public offering at $11, at the upper bound of the estimated price range of $9-$11. The shares will be listed on the Nasdaq under the ticker symbol "BWAY."