BioSpace's Global Biopharma Roundup: March 7

World Map made up of colorful Pills

The European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended conditional marketing authorization for Akcea Therapeutics’ Waylivra, a treatment for confirmed familial chylomicronemia syndrome (FCS).

Approval in Europe for Waylivra is something that has eluded Akcea in the United States. The company, a subsidiary of Ionis Pharmaceuticals, is hoping to snag full regulatory approval across Europe for Waylivra. As part of the conditional marketing authorization, Akcea and Ionis will conduct a non-interventional post-authorization safety study (PASS) based on a registry, the company said.

“This positive CHMP opinion is an important step forward as we work to bring Waylivra to people living with FCS who currently have no treatment options. Once approved, Waylivra will be the first and only therapy for people living with the devastating challenges of FCS. We are now anticipating approval in Europe in the coming months. We will build on the strong infrastructure we have in place for Tegsedi (inotersen) in Europe as we prepare for the launch of Waylivra,” Paula Soteropoulos, chief executive officer of Akcea Therapeutics, said in a statement. “

Portola Pharmaceuticals – The EMA’s CHMP also gave the nod to Portola Pharmaceutical’s marketing authorization application for Ondexxya (andexanet alfa). Ondexxya will now be reviewed for conditional approval as a treatment for the reversal of the anticoagulant effects of the Factor Xa inhibitors apixaban or rivaroxaban in patients experiencing uncontrolled or life-threatening bleeding. The EC is expected to issue a decision in early May 2019. Andexanet alfa was approved by the FDA in May 2018 and is marketed by Portola in the U.S. under the trade name Andexxa [coagulation factor Xa (recombinant), inactivated-zhzo].

BioNTech – Ugur Sahin, founder and CEO of Germany-based BioNTech AG, received the German Cancer Award for 2019. The award is one of the most prestigious distinctions in cancer medicine in Germany. Sahin was recognized in the category Translational Research for his seminal work on individualized cancer immunotherapies and in particular the development and clinical testing of mRNA-based cancer vaccines that are tailored to each patient´s cancer mutation profile.

eTheRNA Immunotherapies – Belgium-based eTheRNA immunotherapies completed the high-dose cohort of its Phase 1b study (E011-MEL) evaluating ECI-006 (TriMix with tumor-specific antigens) in an adjuvant setting in melanoma patients after surgical removal of lesions. The E011-MEL study confirms the feasibility of developing mRNA immunotherapy for direct injection intranodally, in addition to a previously used dendritic cell infusion-based approach, the company said. ECI-006 is a TriMix-based immunotherapy boosted with mRNA encoding melanoma tumor-specific antigens.

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Ayala Pharmaceuticals, Inc. – Israel-based Ayala provided new data for AL101, a gamma-secretase inhibitor being evaluated as a potential therapy for adenoid cystic carcinoma (ACC) with Notch activating mutations, will be presented at the 2019 American Association for Cancer Research (AACR) Annual Meeting in Atlanta. The preclinical data will show that AL101 monotherapy has a significant effect on tumor growth inhibition in a mouse model using implanted ACC patient-derived xenograft (PDX) tumors. The effect of AL101 was only seen in PDX tumors with Notch activating mutations and not on PDX tumors without such mutations. These data support the clinical development of AL101 as a targeted monotherapy for ACC tumors bearing Notch activating mutations.

Tubulis – Germany-based Tubulis announced pre-clinical data evaluating its sophisticated dual platform for the rapid screening and production of highly stable and efficient antibody-drug conjugates (ADCs). In its announcement, the company said the results highlight the technology’s ability to create a wide variety of ADCs with superior plasma stability and the potential for higher efficacy by overcoming limitations that have inhibited the success of many ADCs to-date. In the presentation, Tubulis demonstrated in cell culture and mouse tumor studies that its novel proprietary ADC development approach rapidly produces ADC candidates with equal or superior efficacy compared to established linker technologies. The candidates furthermore show antibody-like stability in classical in vitro assays as well as indicate the potential to reduce the dosing regimen due to improved payload delivery in in vivo models.

Verona Pharma – Part one of a two-part mid-stage clinical trial of a dry powder inhaler formulation of ensifentrine in patients with moderate-to-severe chronic obstructive pulmonary disease hit the mark for United Kingdom-based Verona Pharma plc. The positive data support initiation of the second part of the Phase 2 trial to evaluate the ensifentrine DPI formulation in patients with moderate-to-severe COPD over one week of twice-daily treatment. Interim efficacy and safety data from this single dose study showed a statistically significant and clinically meaningful increase in lung function as measured by forced expiratory volume in one second compared to placebo. Ensifentrine, also known as RPL554, is an investigational first-in-class, inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4 designed to have bronchodilator as well as anti-inflammatory properties.

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