BioSpace Global Roundup, April 2
Takeda– Pharma giant Takeda, based in Osaka, Japan, received a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for an extension of the marketing authorization of Adcetris (brentuximab vedotin) in combination with CHP (cyclophosphamide, doxorubicin, prednisone) as a treatment for adult patients with previously untreated systemic anaplastic large cell lymphoma. The positive opinion is based on positive results from the Phase III ECHELON-2 study. Adcetris is an antibody-drug conjugate (ADC) directed at CD30, which is expressed on the surface of several types of lymphomas, including sALCL. Takeda now awaits approval from the European Commission for the combination treatment.
Ilya Pharma – Sweden’s Ilya Pharma received a BIO-X grant that will support development of two novel chemokine-based gene therapy candidates, ILP100 and ILP101, for Inflammatory Bowel Disease. This will represent a major breakthrough since ILP100 and ILP101 are the first drugs based on genetically engineered lactobacilli. The bacteria act as on-site mini-bioreactors to express chemokines in therapeutic quantities. ILP100 and ILP101 represent both a novel drug delivery technology and novel investigational new products and are classified as advanced therapy medicinal products by the EMA and as low risk candidates under the gene therapy umbrella by the FDA. Ilya Pharma is aiming for progression to clinic by the end of 2022.
Sanofi -- The European Medicines Agency’s CHMP adopted a positive opinion for Paris-based Sanofi’s Sarclisa (isatuximab). The CHMP recommends Sarclisa in combination with pomalidomide and dexamethasone (pom-dex) for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on the last therapy. The CHMP positive opinion is based on data from ICARIA-MM, the first randomized Phase III trial to evaluate an anti-CD38 monoclonal antibody (mAB) in combination with pom-dex. The European Commission will review the CHMP recommendation and a final decision on the Marketing Authorization Application for Sarclisa in the E.U. is expected in the coming months.
Isofol Medical – Sweden’s Isofol said the new gene expression data based on a validated method from TATAA Biocenter AB, which is significantly more sensitive than the pre-commercial method, shows that a larger proportion of patients, including those with low ABCC3 expression, could have significant better effect of arfolitixorin than currently used prodrugs (leucovorin) on PFS and ultimately on overall survival. The data from the optimized gene expression method validated by TATAA Biocenter AB, compared with data based on the pre-commercial gene expression method, shows an increase of the proportion of patients having lower ABCC3 gene expression, 66.6% to 75%, respectively, the company said. The progression free survival benefit for those with a high ABCC3 gene expression increased by 1.1 months, now showing a 4.7 month difference from those with a low ABBC3 gene expression, the company said.
Oxford BioTherapeutics – U.K.-based Oxford BioTherapeutics (OBT) said Boehringer Ingelheim has selected a second asset to advance into formal IND enabling studies. The asset discovery was enabled by the company’s proprietary OGAP target discovery platform. It is one of several assets that have emerged from OGAP including several assets in preclinical and clinical development by OBT. OBT and BI’s collaboration employs OBT’s OGAP target discovery platform to identify novel drug discovery targets that can be substrates for innovative antibody-based therapeutics.
Neurimmune – Swiss pharma company Neurimmune and Germany’s Ethris GmbH forged a partnership to develop mRNA-encoded, neutralizing anti-SARS-CoV-2 antibodies administered by inhalation for the treatment of Covid-19. The collaboration brings together Neurimmune’s expertise in developing human antibodies based on high-throughput immunoglobulin sequence analyses from COVID-19 patients who have recovered from the disease with Ethris’ proprietary and unique pulmonary SNIM RNA therapeutics platform. Both companies will work together to rapidly develop an immunotherapy designed to produce therapeutic antibodies directly in the lungs of affected patients.
GENFIT – France’s GENFIT continues to monitor the COVID-19 outbreak, the company said it does not think the virus will impact its late-stage NASH and liver fibrosis trial assessing elafibranor. The company said it is continuing the extension phase of the Phase III RESOLVE-IT trial. However, GENFIT is pausing its Phase I trials due to the pandemic.
ProQR Therapeutics – Netherlands-based ProQR announced positive findings from an interim analysis of its Phase I/II STELLAR trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations. QR-421a is an investigational RNA therapy.
Optibrium – University of Cambridge spinout Optibrium reached a significant milestone in its contribution to the Open Source Malaria initiative. The team has successfully completed Phase II of a global challenge aimed at developing and testing novel antimalarial compounds. During this phase, predictive models from Phase I were combined with generative methods to design novel compounds. The compounds were subsequently validated by testing their activity against the target. Out of four compounds proposed in this phase, only Optibrium/Intellegens’ entry demonstrated potency against the target.
Mind Medicine – Switzerland’s Mind Medicine signed a multi-year collaboration with a leading psychedelics group at University Hospital Basel in Switzerland. Under the agreement, MindMed gains exclusive worldwide rights to data, compounds, and patent rights associated with the laboratory's research with LSD and other psychedelic compounds. MindMed is particularly interested in the data and outcome of an ongoing placebo-controlled Phase II trial of high dose LSD for the treatment of anxiety.
Theranexus– France-based Theranexus said its Phase II Parkinson’s disease trial was a success. The company said data supported efficacy of its drug candidate THN102 for Parkinson's disease. THN102 met the primary efficacy endpoint and significantly increased the proportion of patients no longer suffering from daytime sleepiness for the duration of the treatment.
Horizon Therapeutics – The company announced pooled efficacy data from the Phase II and III clinical trials of Tepezza (teprotumumab-trbw) showing that the recently approved medicine effectively reduces proptosis (eye bulging) in patients with Thyroid Eye Disease (TED) regardless of age, gender and smoking status. This new analysis was conducted to determine if there are any differences in proptosis response based on patient demographic characteristics, including age, gender and smoking status.