The Daily Biotech Pulse: Biohaven In-Licenses Drug, Retrophin's Debt Offering, ProQR And Bluebird Report Positive Trial Results

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 Sept. 4)

• Aptinyx Inc APTX
• Becton Dickinson and Co BDX
• BioCryst Pharmaceuticals, Inc. BCRX(announced positive results for the Zenith-1 trial that evaluated its BCX7353 for treating hereditary angioedema patients)
• DexCom, Inc. DXCM
• EXACT Sciences Corporation EXAS
• Genomic Health, Inc. GHDX
• Haemonetics Corporation HAE
• Inspire Medical Systems Inc INSP
• Masimo Corporation MASI
• Myriad Genetics, Inc. MYGN
• Neurocrine Biosciences, Inc. NBIX
• Omeros Corporation OMER
• ProQR Therapeutics NV PRQR
• Repligen Corporation RGEN
• STAAR Surgical Company STAA
• Uniqure NV QURE

Down In The Dumps

(Biotech Stocks Hitting 52-week lows on Sep. 4)

• Chimerix Inc CMRX
• Cytori Therapeutics Inc CYTX
• INTEC PHARMA ORD NTEC
• Oncolytics Biotech, Inc. (USA) ONCY

Related Link: Attention Biotech Investors: Mark Your Calendar For These September PDUFA Dates

Stocks In Focus

Merck, Partner Eisai Announce Approval of Liver Cancer Drug In China

Merck & Co., Inc. MRK and Eisai announced that the China National Medical Products Administration approved Lenvima, a kinase inhibitor, as a single agent treatment for unresectable hepatocellular carcinoma.

Biohaven In-licenses Neurological Drug From AstraZeneca

Biohaven Pharmaceutical Holding Co Ltd BHVN said it has entered into an exclusive worldwide license agreement with AstraZeneca plc (ADR) AZN for acquiring the development and commercialization rights to AZD3241, an oral myeloperoxidase inhibitor that has progressed through the Phase 2 trial. Biohaven plans to commence Phase 3 trial for evaluating the candidate for multiple system atrophy, a fatal neurogenerative disease.

The deal terms provide for Biohaven paying AstraZenexa an upfront cash payment and shares in Biohaven, and also further development and commercial milestone payments, and up to double-digit sales based royalties.

Agios Names CEO as Incumbent Transitions to Chairman Role

Agios Pharmaceuticals Inc AGIO announced long-time CEO David Schenkein will transition to the role of Chairman, effective Feb. 1, 2019. The company named board member Jacqualyn Fouse as its next CEO.

Pfizer Prices Debt Offering

Pfizer Inc. PFE announced pricing of its debt offering consisting of six tranches of notes – a $300 million floating rate note offering due 2023, a $1 billion note offering due 2021, a $1 billion note offering due 2023, a $1 billion offering due 2028, a $700 million note offering due 2038 and a $1 billion note offering due 2048.

The company expects to close the offering Sept. 7.

ProQR Therapeutics Reports Positive Results For Genetic Blindness Drug

ProQR released interim analysis of Phase 1/2 data for its QR-110 for treating Leber's congenital amaurosis, a form of genetic blindness, which showed rapid and sustained improvement in vision in patients, as measured by visual acuity and the mobility course performance. It was also well-tolerated with no serious adverse events.

Bluebird Bio Says Study Confirms Efficacy, Safety of Neurodegenerative Drug

bluebird bio Inc BLUE released updated Phase 2/3 data on its Lenti-D to treat childhood cerebral adrenoleukodystrophy, or CALD, at the Society for the Study of Inborn Errors of Metabolism 2018 Symposium, which showed that the updated data was consistent with the previously reported Phase 2/3 Starbeam results.

The company also said it reached a general agreement with the FDA and the European Medicines Agency on the clinical development program to support future marketing applications for Lenti-D in CALD.

Retrophin to Offer $200 Million Worth of Debt

Retrophin Inc RTRX intends to offer $200 million aggregate principal amount of convertible senior notes due 2025 in an underwritten offering. The company intends to use the net proceeds to pay a portion of its outstanding 4.50 percent senior convertible notes due 2019.

The stock fell 9.33 percent to $29.15 in after-hours trading.

Tesaro Lung Cancer Drug Found Effective In Mid-stage Trial

TESARO Inc TSRO said it has initiated the second stage of the JASPER study that evaluates the clinical benefit of Zejula in combination with an anti-PD-1 antibody as a first-line treatment of non-small cell lung cancer.

The decision was based on achieving the protocol defined response criteria in the initial cohort of 16 treated patients with high PD-L1 expression, of which 14 were evaluable for a response.

"Nine of the 14 patients had objective responses by RECIST criteria at the time of the analysis, with all 14 patients experiencing tumor shrinkage," the company said.

The stock climbed 8.7 percent to $36 in after-hours trading.

Aduro's Multiple Myeloma Drug Found Safe, Effective In Pre-clinical Study

Aduro BioTech Inc ADRO's presentation of pre-clinical data for its anti-APRIL antibody BION-1301 at the 5th European Congress of Immunology in Amsterdam showed that the candidate being evaluated for multiple myeloma was well tolerated.

"In addition, pharmacological activity of BION-1301 binding to APRIL (A Proliferation-Inducing Ligand), a ligand for the receptors BCMA (B cell maturation antigen) and TACI (transmembrane activator and cyclophilin ligand interactor), was established in a dose-dependent fashion," the company said.

The stock rallied 8.84 percent to $8 in after-hours trading.

On The Radar

Earnings

• Misonix, Inc. MSON

PDUFA Date

Roche Holdings AG Basel ADR RHHBY's non-squamous non-small cell lung cancer, or NSCLC, candidate Tecentriq awaits FDA decision. Tecentriq is tested in combination with Avastin, paclitaxel and carboplatin (chemotherapy), for the initial (first-line) treatment of metastatic non-squamous NSCLC.

Clinical Trial Results

• Spectrum Pharmaceuticals, Inc. SPPI is set to release abstract of the updated Phase 2 data on Poziotinib, its pipeline candidate for non-small cell lung cancer with exon 20 insertion mutation in EGFR, or HER2. Detailed data is to be presented at the World Conference on Lung Cancer on Sep. 24.
• Sangamo Therapeutics Inc SGMO highlights presentation at annual symposium of the Society for the Study of Inborn Errors of Metabolism evaluating SB-913, which showed early data from Cohorts 1, 2, enrollment of Cohort 3 recently completed; "Total urinary GAGs declined by 51%, dermatan sulfate by 32%, and heparan sulfate by 61% in Cohort 2 at 16 weeks"; "SB-913 has been generally well-tolerated with no treatment related serious adverse events in patients up to 32 weeks"