FDA approves first treatment for rare form of skin cancer

The U.S. Food and Drug Administration today granted accelerated approval to Bavencio (avelumab) for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC), including those who have not received prior chemotherapy. This is the first FDA-approved treatment for metastatic MCC, a rare, aggressive form of skin cancer.【阅读全文】

Biotech giant plans $25M Tampa investment, 450 jobs

Amgen Inc. will open a capability center in Tampa, creating hundreds of high-skill jobs.

Amgen (NASDAQ: AMGN), a biotechnology firm headquartered in Thousand Oaks, California, will invest $25 million in the 136,000-square-foot center, which will occupy four floors of Corporate Center One at 2202 N. Westshore Blvd. The center is expected to open in October and will employ 450 people by the end of 2018, a press release from the Tampa Hillsborough Economic Development Corp. said.【阅读全文】

BioLineRx buys Agalimmune to expand cancer pipeline

BioLineRx has bought Agalimmune to expand its immuno-oncology pipeline. The takeover adds the synthetic alpha-gal immunotherapy AGI-134 to BioLineRx’s pipeline as the drug nears the start of a first-in-human trial in solid tumors.

Tel Aviv, Israel-based BioLineRx is paying $6 million (€5.6 million) upfront—split evenly between cash and stock—to buy Agalimmune. In return for the outlay, BioLineRx is gaining AGI-134, a preclinical-stage asset it thinks could make immunotherapies effective against solid tumors that evade the attacks of today’s therapeutics.【阅读全文】

Merrimack Initiates Phase 1 Study of MM-310 in Solid Tumors

Merrimack Pharmaceuticals, Inc. (NASDAQ: MACK) today announced the enrollment of its first patient in a Phase 1 study of MM-310 in solid tumors. MM-310 is an antibody-directed nanotherapeutic (ADN) that encapsulates a novel taxane and targets the EphA2 receptor, a protein which surveys suggest is overexpressed in 50-100% of many major tumor types, including prostate, ovarian, bladder, gastric, pancreatic and lung cancers.【阅读全文】

Pharma development budgets grow as Roche remains a believer

Roche was once again the sector’s biggest spender on drug development last year, its R&D bill touching $10bn for the first time. The Swiss company also remained one of the most enthusiastic investors in science among the world’s biggest pharma groups, data collated by EvaluatePharma show.

However, in terms of budget growth, Abbvie has increased spend on its development labs more than any other major over the past five years, percentage-wise. The company is working desperately to replace Humira, which in the meantime is generating plenty of cash to support its growing R&D investment.【阅读全文】

Bristol failure makes small dent in CXCR4-blocking approach

Bristol-Myers Squibb’s discontinuation of a phase I/II study of ulocuplumab highlights an unusual mechanism of action – CXCR4 inhibition – that has quietly been pursued as part of several companies’ oncology strategies.

Biolinerx and Polyphor have similarly acting agents as lead projects, while Lilly is relying on one as potentially its fifth-most lucrative pipeline asset. The approach aims to trigger stem cell mobilisation, and while the Bristol failure raises obvious doubts at least one other group, X4 Pharmaceuticals, is entirely focused on exploring the CXCR4 pathway【阅读全文】

Atossa Genetics Opens Enrollment in Study of Endoxifen

Atossa Genetics Inc. (NASDAQ: ATOS) today announced that it has opened enrollment of a Phase 1 study of endoxifen, which is an active metabolite of tamoxifen, an FDA approved drug for breast cancer. The objectives of this placebo-controlled, repeat dose study of 48 healthy female volunteers is to assess the pharmacokinetics of proprietary formulations of both oral and topical endoxifen dosage forms over 28 days, as well as to assess safety and tolerability.

The study is being conducted on behalf of Atossa by CPR Pharma Services Pty Ltd., Thebarton, SA, Australia. 【阅读全文】

Kineta RLR Agonist Demonstrates Tumor Regression and an Adaptive Immune Response in Colon Carcinoma Model

Kineta, Inc., a biotechnology company focused on the translational development of novel therapies in immuno-oncology, today announced the discovery and characterization of several "hit to lead" small molecule compounds that activate interferon response factor 3 (IRF3) via RIG-I like receptor (RLR) pathways and demonstrate immune-mediated tumor regression in a murine colon carcinoma mouse model. Furthermore, mice in the study who demonstrated complete tumor regression to initial drug treatment were resistant to tumor re-challenge confirming an adaptive immune response in these animals. The findings were presented on March 22nd at the Keystone Symposia on Cancer Immunology and Immunotherapy: Taking a Place in Mainstream Oncology conference.【阅读全文】

Shire’s rare blood disorder drug gets US fast track

An experimental therapy in development by Shire for patients with the rare blood disorder hereditary thrombotic thrombocytopenic purpura (hTTP) has been granted a fast track designation by the US Food and Drug Administration.

The company is targeting its therapy SHP655 (recombinant ADAMTS13) towards treatment of acute episodes of hTTP in patients with a constitutional deficiency of the von Willebrand factor-cleaving (VWF) protease ADAMTS13.【阅读全文】

Ultragenyx looks ahead after triheptanoin failure

Ultragenyx’s phase II miss with triheptanoin in transporter type-1 deficiency syndrome might not affect an ongoing phase III trial in a slightly different indication – but it does not do much to boost confidence in the project.

Meanwhile, the company can console itself that it will soon have pivotal data from another candidate, KRN23, in X-linked hypophosphataemia, and is planning a filing of the enzyme-replacement therapy rhGUS in the first half of 2017. Sales of these products are likely to be smaller than those for triheptanoin, according to EvaluatePharma consensus, but Ultragenyx might benefit from not putting all of its rare disease eggs in one basket.【阅读全文】