FDA grants priority review for Biogen’s tofersen to treat ALS

27 July | R&D

The US FDA has granted priority review for Biogen’s New Drug Application (NDA) for the investigational drug, tofersen, to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). A decision from the regulatory agency on approval for the drug is anticipated on 25 January next year.  The FDA also plans to hold an advisory committee meeting for the NDA. The application for the drug comprised data from a Phase I clinical trial in healthy subjects, a Phase I/II trial analysing ascending dose levels, the Phase III VALOR trial and an open-label extension (OLE) trial. It also included the latest one-year combined data from VALOR and the OLE study.

5 August | DEALS

Amgen to acquire biopharma firm ChemoCentryx for $3.7bn

Amgen has signed a definitive agreement to acquire biopharmaceutical company ChemoCentryx for $52 for each share in cash or a total enterprise cost of nearly $3.7bn. With the takeover, Amgen will gain access to ChemoCentryx ’s Tavneos (avacopan), a therapy for serious autoimmune diseases. This therapy complements Amgen’s expertise in inflammation and nephrology. An orally administered selective inhibitor of complement component 5a receptor, Tavneos received approval from the US Food and Drug Administration (FDA) in October last year. It is approved as an adjunctive therapy along with standard therapy for treating adults with severe active ANCA-associated vasculitis, particularly granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA).

20 July | R&D

Avista and Roche to develop gene therapy vectors for eyes

Avista Therapeutics, a University of Pittsburgh Medical Center (UPMC) spinout, has entered a collaboration with Roche for developing new AAV gene therapy vectors for eyes. Avista is entitled to receive $7.5m in upfront payment from Roche. The alliance plans to leverage the single-cell adeno-associated virus (AAV) engineering (scAAVengr) platform technology of Avista to develop intravitreal AAV capsids for a Roche-defined profile. According to the deal, Roche holds the right to analyse and licence new capsids from Avista. Roche will use these new capsids to carry out preclinical, clinical and marketing activities for gene therapy programmes. These capsids will be different from the internal pipeline of Avista.

4 August | R&D

Poseida and Roche to develop cell therapies for hematologic cancers

Poseida Therapeutics and Roche have signed a strategic partnership and license agreement to develop allogeneic CAR-T cell therapies for hematologic malignancies. According to the deal, Poseida will offer either exclusive rights or options to Roche for developing and marketing various allogeneic CAR-T programmes in its portfolio targeting hematologic malignancies. Poseida could get research, development, launch and net sales milestones and other payments totalling up to $6bn. The programmes include P-BCMA-ALLO1, an allogeneic CAR-T to treat multiple myeloma and P-CD19CD20-ALLO1, an allogeneic dual CAR-T to treat B-cell malignancies. P-BCMA-ALLO1 is being analysed in a Phase I clinical trial while an investigational new drug (IND) application for P-CD19CD20-ALLO1 is anticipated next year.

1 August | COVID-19

Moderna signs contract to supply Covid-19 booster vaccines to the US

Moderna has entered a new supply contract with the US Government to deliver 66 million doses of its Covid-19 vaccine booster candidate, mRNA-1273.222, this year. The contract comprises a $1.74bn award to produce and supply these vaccine doses and options to further procure up to 234 million additional doses of the company’s booster candidates. A bivalent booster candidate, the mRNA-1273.222 vaccine comprises Spikevax along with the Omicron BA.4/5 strain messenger RNA (mRNA). On the US exercising options, the doses could total 300 million. The US Department of Health and Human Services, and Administration for Strategic Preparedness and Response (ASPR) and others are supporting the deal through federal funds.

22 July | REGULATORY

Sangamo’s cell therapy receives EC orphan medicinal product designation

Sangamo Therapeutics has received orphan medicinal product designation (OMPD) from the European Commission (EC) for its Investigational autologous Chimeric Antigen Receptor Regulatory T Cell (CAR-Treg) cell therapy, TX200, for solid organ transplantation. TX200 has been developed for reducing the transplant rejection risk by suppressing inflammation in the local area and promoting immunological tolerance. The cell therapy is being evaluated in patients with HLA-A2 negative receiving a mismatched kidney from a living donor with HLA-A2 positive kidney. In March, the company dosed the first participant in the Phase I/II STEADFAST clinical study, which is designed to evaluate the safety and tolerability of TX200.

In Brief

EMA task force begins review of Veru’s sabizabulin for Covid-19

The Emergency Task Force (ETF) of the EMA has commenced the review of Veru’s sabizabulin to treat hospitalised Covid-19 patients at increased acute respiratory distress syndrome (ARDS) risk. The review will facilitate the use of the therapy for emergency usage in countries in the EU.

US FDA approves Daiichi Sankyo-AstraZeneca’s Enhertu for lung cancer

The US FDA has granted approval for Daiichi Sankyo and AstraZeneca’s Enhertu (fam-trastuzumab deruxtecan-nxki) to treat unresectable or metastatic non-small cell lung cancer (NSCLC) in adults, whose tumours have activating HER2 (ERBB2) mutations and have previously received systemic therapy.

GentiBio and BMS partner to develop inflammatory bowel disease therapies

GentiBio and Bristol Myers Squibb (BMS) have signed a multi-year partnership agreement for developing new engineered Treg therapies for inflammatory bowel disease (IBD) patients.  

These therapies will be developed for re-instating immune tolerance and repairing tissue in IBD.

FDA grants Orphan Drug status to Aro Biotherapeutics’ Pompe disease therapy

The US FDA has granted Orphan Drug Designation for Aro Biotherapeutics’ ABX1100 to treat Pompe disease. An investigational Centyrin-small interfering ribonucleic acid (siRNA) conjugate, ABX1100 acts on the glycogen synthase 1 (Gys1) gene in the muscle.

25 July | REGULATORY

EC extends marketing authorisation for Bavarian Nordic’s smallpox vaccine

The EC has extended Bavarian Nordic’s smallpox vaccine Imvanex (MVA-BN or Modified Vaccinia Ankara-Bavarian Nordic) marketing authorisation to offer protection from monkeypox. A non-replicating smallpox vaccine, Imvanex was developed in partnership with the US Government.The move to expand the marketing authorization for Imvanex to include protection from monkeypox and disease caused by the vaccinia virus comes after the EMA CHMP provided a positive opinion.

26 July | DEALS

Ginkgo to acquire Zymergen for $300m 

Ginkgo Bioworks has signed a definitive agreement for the acquisition of biotechnology company Zymergen in an all-stock deal worth nearly $300m. According to the agreement, 5.25% of the pro forma merged business will be owned by the stockholders of Zymergen following the acquisition. The deal to combine two complementary companies with identical visions is anticipated to expedite the development of the innovative horizontal synthetic biology platform of Ginkgo.

In brief

EMA task force begins review of Veru’s sabizabulin for Covid-19

The Emergency Task Force (ETF) of the EMA has commenced the review of Veru’s sabizabulin to treat hospitalised Covid-19 patients at increased acute respiratory distress syndrome (ARDS) risk. The review will facilitate the use of the therapy for emergency usage in countries in the EU.

US FDA approves Daiichi Sankyo-AstraZeneca’s Enhertu for lung cancer

The US FDA has granted approval for Daiichi Sankyo and AstraZeneca’s Enhertu (fam-trastuzumab deruxtecan-nxki) to treat unresectable or metastatic non-small cell lung cancer (NSCLC) in adults, whose tumours have activating HER2 (ERBB2) mutations and have previously received systemic therapy.

GentiBio and BMS partner to develop inflammatory bowel disease therapies

GentiBio and Bristol Myers Squibb (BMS) have signed a multi-year partnership agreement for developing new engineered Treg therapies for inflammatory bowel disease (IBD) patients. These therapies will be developed for re-instating immune tolerance and repairing tissue in IBD.

FDA grants Orphan Drug status to Aro Biotherapeutics’ Pompe disease therapy

The US FDA has granted Orphan Drug Designation for Aro Biotherapeutics’ ABX1100 to treat Pompe disease. An investigational Centyrin-small interfering ribonucleic acid (siRNA) conjugate, ABX1100 acts on the glycogen synthase 1 (Gys1) gene in the muscle.