Biopharma and life sciences companies from across the globe provide updates on their businesses and pipelines.
France-based Corlieve Therapeutics and its lead program for temporal lobe epilepsy will be acquired by Massachusetts-based uniQure N.V. Corlieve’s lead gene therapy, now known as AMT-260, uses miRNA silencing technology to target suppression of aberrantly expressed kainate receptors in the hippocampus of patients with temporal lobe epilepsy, a disease that affects approximately 1.3 million people across the United States and Europe. Of those, about 800,000 are unable to adequately control acute seizures with currently approved anti-epileptic therapies. Patients with refractory TLE experience increased morbidity, excess mortality, and poor quality of life.
Under the terms of the agreement, uniQure will pay €46.3 million ($55.39 million) in an upfront payment of cash to acquire Corlieve. Additional finances include up to €43.7 million in development milestones through Phase I/II and €160 million in milestones associated with Phase III development and approvals of AMT-260 in the U.S and European Union.
“As leaders in the field for miRNA gene therapy for neurological conditions, uniQure is the ideal long-term partner for us, and we look forward to working together to advance our program rapidly to the clinic for the benefit of the patients we serve,” Richard Porter, founder and chief executive officer of Corlieve said in a statement. Upon the closing of the transaction, Porter will assume the role at uniQure of General Manager of the Corlieve subsidiary.
Elsewhere around the globe:
AstraZeneca – AstraZeneca and Merck won approval for Koselugo, the first medication approved in the European Union to treat pediatric patients with neurofibromatosis type 1 (NF1), a rare and debilitating genetic condition. In NF1, tumors can develop on the nerve sheaths, which can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction. The approval by the European Commission was based on positive results from the SPRINT Stratum 1 Phase II trial, which showed Koselugo demonstrated an objective response rate (ORR) of 66% in pediatric patients with NF1 PN when treated with Koselugo as twice-daily oral monotherapy. Koselugo has been approved in the US and several other countries for the treatment of pediatric patients with NF1 and symptomatic, inoperable PN.
Coriolis Pharma – Germany-based Coriolis announced the expansion of its advanced therapy medicinal products (ATMP) development facilities under biosafety level S2 following renovation of an existing building. The new facilities will increase Coriolis’ total floor space to 7,800 m2. The new labs will host the formulation development of ATMPs, cell culture activities, particle characterization and identification, analytical ultracentrifugation and a lyophilization development center for ATMPs.
Avacta Group – Based in the U.K., Avacta Group plc has entered into a non-exclusive distribution agreement with Calibre Scientific Inc., for Avacta’s AffiDX SARS-CoV-2 antigen lateral flow test for professional use in the UK and European Economic Area.
Biotage – Sweden’s Biotage launched Biotage PhyPrep, an automated platform for plasmid DNA purification. The new platform is expected to improve lab work flow and take advantage of the PhyTip columns’ dual flow chromatography technology, to yield supercoiled, endotoxin-free, transfection-grade plasmid DNA, the company said.
Iksuda Therapeutics – Antibody drug conjugate developer Iksuda, based in the U.K., expanded its research collaboration and License Agreement with LegoChem Biosciences, Inc. to explore additional ADC programs in order to leverage that company’s proprietary ADC platform technology. The two companies partnered in 2020 on three ADC programs using LCB’s ADC linker/toxin platform. Under the expanded agreement, Iksuda has been granted rights for three additional targets. Iksuda also gains access to LCB’s innovative and recently discovered novel DNA-modifying payload, supplementing LCB’s proprietary tumor-activated DNA toxins.
ProBioGen – Germany-based ProBioGen and Netherlands-based LAVA Therapeutics NV announced the closing of a second cell line development agreement to support a bispecific gamma-delta T cell engager (TCE) candidate in LAVA’s pipeline. ProBioGen will use its CHO.RiGHT expression platform to generate stable high-titer cell lines in order to develop novel gamma-delta bsTCE.
AbCellera – Vancouver-based AbCellera secured a new site for the construction of a Good Manufacturing Practices (GMP) manufacturing facility that will be used to produce therapeutic antibodies. The 130,000-square-foot facility will expand AbCellera’s capabilities in bringing new antibody therapies to clinical trials. The new GMP facility will be built on what is currently a vacant two-acre site, and the planned building will house laboratory, office, GMP protein biologic manufacturing, and warehouse space. The announcement follows the company’s previous groundbreaking on a new global headquarters in Vancouver. The 380,000-square-foot headquarters will be within walking distance of the new GMP manufacturing facility. The facility is scheduled to be ready for GMP manufacturing in early 2024. When it operates at full capacity, the site will employ hundreds scientific personnel.
Poxel SA – France’s POXEL announced that Twymeeg (Imeglimin hydrochloride) tablets for type 2 diabetes has been approved in Japan.
TWB – France’s TWB launched two new service offerings developed in partnership with innovative start-ups in its consortium. The first offering developed with ALTAR, involves adapting microorganisms to the requirements of industrial companies via natural selection. The second, which was developed with the start-up iMEAN, delivers a modelling program capable of streamlining strain engineering in industrial biotechnologies.
Ilya Pharma – Based in Sweden, Ilya Pharma has received approval from the Swedish Medical Product Agency and Swedish Ethical Review Authority to initiate a Phase II clinical study of its ILP100 gene therapy for the treatment of difficult diabetic wounds. ILP100h represents a new-in-class drug candidate, engineering Limosilactobacillus reuteri bacteria to produce and release the chemokine CXCL12 at the actual wound site. After delivery, ILP100 enhances the healing properties of immune cells in the wound. Phase I results released earlier this year indicate that ILP100-treated wounds in healthy subjects healed on average 6 days and, for the highest dose, 11 days faster than controls.
Immunicum AB – The first patient has been enrolled in the Phase I ALISON study assessing DCP-001, the company’s lead cancer relapse vaccine candidate, in High-Grade Serous Ovarian Cancer (HGSOC) patients following primary standard of care treatment. HGSOC is a unique type of epithelial cancer that is characterized by the loss of function of the tumor suppressor protein, p53, which can lead to chemotherapy resistance and disease relapse. This is the first study using Immunicum’s cancer relapse vaccine approach to target a solid tumor indication and will evaluate the safety, feasibility and immunogenicity of DCP-001.
Abivax SA – France-based Abivax announced topline results from a Phase IIa study assessing ABX464 in combination with methotrexate (MTX) for the treatment of active moderate to severe rheumatoid arthritis that the company is calling “excellent.” The study hit its primary endpoint showing that a once-daily 50mg dose of the combination treatment was safe and tolerable. Although the study was not designed to demonstrate efficacy, Abivax said the group that received the 50mg dose showed “statistically significant differences for the key secondary endpoint ACR20 compared to placebo” at 12 weeks. ACR20 is the key primary efficacy endpoint required by the FDA for licensure of new drugs in rheumatoid arthritis. Based on data from the IIa study, Abivax will initiate a Phase IIb study assessing the combination in early 2022. ABX464 is a small molecule for once-daily administration with a first-in-class mechanism of action that was centered on the upregulation of the anti-inflammatory microRNA, miR-124. ABX464 was already shown to be efficacious and safe in Phase IIa and Phase IIb clinical studies for the treatment of ulcerative colitis.
