The Daily Biotech Pulse: Acorda Swings To Profit, Achaogen Offering, Bioblast Strikes Sanfilippo Syndrome Deal

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 Feb. 14)

• argenx SE – ADR ARGX
• Orthofix Medical Inc OFIX
• OUTLOOK THERAPEUTICS, INC. OTLK(reported a narrower net loss per share for fiscal-year first-quarter)
• Vericel Corp VCEL
• Wright Medical Group NV WMGI

Down In The Dumps

(Biotech stocks hitting 52-week lows on Feb. 14)

• CELLECT BIOTECH/S ADR APOP
• Endologix, Inc. ELGX
• MediciNova, Inc. MNOV
• MOTIF BIO PLC/S ADR MTFB(received complete response letter for its NDA for iclaprim for treating acute bacterial skin and skin structure infections)
• Organogenesis Holdings Inc ORGO
• Ovid Therapeutics Inc OVID
• Scpharmaceuticals Inc SCPH
• Xeris Pharmaceuticals Inc XERS(priced its common stock offering)

Stock In Focus

Acorda Posts an Unexpected Profit For Q4

Acorda Therapeutics Inc ACOR reported fourth-quarter revenues of $69.152 million compared to $188.398 million in the year-ago period. The sharp decline was due to the loss of exclusivity for its Ampyra, as generics entered the market. Non-GAAP net income fell from 61 cents per share to 45 cents per share.

The stock rallied 9.25 percent to $17 in after-hours trading.

Bioblast Strikes Partnership For Developing Rare Disease Drug

Israeli biotech Bioblast Pharma Ltd ORPN announced a clinical collaboration with Team Sanfilippo, a non-profit medical research foundation, to evaluate the safety and efficacy of Bioblast's proprietary Trehalose solution for treating patients with Sanfilippo syndrome.

Under the terms of the deal, the foundation will conduct an open-label study in up to 20 Sanfilippo patients to evaluate the safety and efficacy. If successful, Bioblast may determine to continue the development of Trehalose toward a pivotal study, while maintaining the commercial rights.

Sanfilippo Syndrome, or mucopolysaccharidosis III is a rare autosomal recessive lysosomal storage disease. It is caused by a deficiency in one of the enzymes needed to break down the glycosaminoglycan heparan sulfate, and results in developmental disability and the loss of previously learned skills in children.

The stock rallied 28.18 percent to $1.41 in after-hours trading.

See Also: Gilead Analysts React To Failed NASH Trial: Citigroup, Wells Fargo Downgrade Biotech

Achaogen To Offer Shares

Achaogen Inc AKAO shares, which have been on a downtrend since June last year following the mixed FDA verdict for its broad-spectrum antibiotic Plazomicin, said it has commenced an underwritten public offering of its common shares. All the shares earmarked for the offered are to be sold by the company.

The company expects to use the net proceeds for the commercialization of Plazomicin, with the brand name Zemdri, in the U.S, the regulatory approval process for Plazomicin in Europe and the development of its product candidate C-Scape.

The stock slumped 22.54 percent to $1.10 in after-hours trading.

Bristol-Myers Squibb Combo Cancer Drug Produces Response in Prostate Cancer

Bristol-Myers Squibb said the results from an interim analysis of the Phase 2 CheckMate-650 trial evaluating its Opdivo in combination with Yervoy in patients with metastatic castration-resistant prostate cancer showed an ORR of 25 percent.

The response was from among 32 asymptomatic or minimally symptomatic patients whose disease has progressed after second-generation hormone therapy and who have not received chemotherapy, with a median follow-up of 11.9 months.

The response rate was 10 percent in 30 patients whose disease progressed after taxane-based chemotherapy, with a median follow-up of 13.5 months.

Bayer's Prostate Cancer Drug Safe and Effective In Phase 3 Trial

Bayer AG BAYRY said its investigational drug Darolutamide along with androgen deprivation therapy, or ADT, significantly extended metastasis-free survival compared to placebo, plus ADT, in non-metastatic castration-resistant prostate cancer. Darolutamide, plus ADT, produced a 59 percent reduction in the risk of metastasis or death in a Phase 3 study dubbed ARAMIS. The median metastasis-free survival was 40.4 months compared to 18.4 months for the placebo arm.

OHR Pharma Q1 Loss Narrows

OHR Pharmaceutical Inc OHRP, which has agreed to merge with privately-held NeuBase Therapeutics, reported a loss of 32 cents per share for the first quarter compared to a loss of $1.48 per share last year.

The shareholder vote on the NeuBase transaction us due in the second quarter of 2019.

The stock slipped 3.76 percent to $2.05 in after-hours trading.

On The Radar

PDUFA Dates

The FDA is set to rule on Bausch Health Companies Inc BHC's NDA for Duobrii, being evaluated for plaque psoriasis. The application was handed down a CRL in June 2018 following which a resubmission was done in Aug. 2018.

Clinical Trial Results

ASCO GU Cancers Symposium Presentations

Immunomedics, Inc. IMMU is due to present Phase 1/2 data (already released Feb. 11) for its urothelial cancer pipeline candidate Sacituzumab govitecan.

Seattle Genetics, Inc. SGEN will release Phase 1 data for its urothelial cancer candidate Enfortumab vedotin, codenamed EV-101.

Rexahn Pharmaceuticals, Inc. RNN is scheduled to release updated preliminary Phase 2a data for RX-3117, its bladder cancer candidate, at 12:15 pm PST.

Nektar Therapeutics NKTR will release updated Phase 1/2 data for its NKTR-214 in combination with Bristol-Myers Squibb Co BMY, being evaluated for a host of cancers, including urothelial carcinoma, melanoma, renal cell carcinoma and non-small cell lung cancer.

IPO

Stealth Biotherapeutics, which focuses on therapies for mitochondrial dysfunction, priced its 6.5 million ADSs, each representing 12 ordinary shares, at $12, at the lower end of the estimated price range of $12-$14. The shares will be listed on the Nasdaq under the ticker symbol MITO.