Mymetics, a pioneer and leader in the research and development of virosome-based vaccines against life threatening and life disabling diseases, announced that the pre-clinical proof-of-concept study for allergies, with their partner Anergis, has met the success criteria.

The mice proof-of-concept immunogenicity study evaluated the effects of the Bet v 1 COPs (Anergis’ proprietary birch pollen allergy peptides) using the five subcutaneous injection schedule used in former AllerT clinical trials. The development of AllerT (Bet v 1 COPs plus aluminum hydroxide) was discontinued by Anergis in 2017 following completion of a Phase 2 clinical trial showing evidence of sensitization to the peptides and a 7% reduction in seasonal allergy symptoms vs placebo (p=0.0047).

In the mice study, AllerT was compared to Bet v 1 COPs linked to Mymetics’ virosomes (the “Bet v 1 COP-virosomes”). Anergis reported that the administration of AllerT led to the development of Bet v 1 specific IgEs (p<0.001) associated with a more pronounced TH2 than TH1 response. In contrast, in the mice receiving the Bet v 1 COP-virosomes, no development of Bet v 1 specific IgEs were observed (p<0.001 vs AllerT). With the same dose of Bet v 1 COPs, there was a strong boost of immunogenicity with a TH1 antibody response, which was a hundred times greater than with aluminum hydroxide (p<0.001). The Bet v 1 COP-virosomes were well tolerated.

This study is part of the research collaboration agreement between Anergis and Mymetics which was signed in April 2018. The success criteria were met and Anergis has now a time limited option to enter into an exclusive license agreement with Mymetics for the use of virosomes in the field of allergies.

Ronald Kempers, CEO of Mymetics, stated: “The results from Anergis are very encouraging and show that our virosomes can have a broad application in vaccines, not only for prevention of infectious diseases but also in the field of immunotherapy for allergies.”

Source: Mymetics

Het Zorginstituut nodigt geneesmiddelenfabrikanten uit om bij te dragen aan de halfjaarlijkse update van de Horizonscan Geneesmiddelen. Deze scan brengt patiënten, behandelaars, ziekenhuizen, zorgverzekeraars en overheidsorganen vroegtijdig op de hoogte van ontwikkelingen op het gebied van geneesmiddelen. Verwacht je de aankomende twee jaar geneesmiddelen op de markt te brengen? Of uitbreidingen van bestaande indicaties? Dan is deze oproep van het Zorginstituut relevant voor jou.

Met deze uitvraag verzamelt het Zorginstituut gegevens voor de scan die in juni 2019 live gaat. De deadline voor aanlevering is 11 januari 2019. Producten verschijnen overigens sowieso op de scan, ongeacht of de fabrikant informatie aanlevert. Het Zorginstituut gebruikt hiervoor informatie van “(Horizon)scanners” en werkgroepen van experts. Door zelf informatie aan te leveren, draag je als bedrijf bij aan de volledigheid en accuraatheid van de scan. HollandBIO juicht deze mogelijkheid dan ook toe.

Meer weten of bijdragen?

Op hun website licht het Zorginstituut de uitvraag en werkwijze verder toe (deadline 11 januari!). Voor vragen, nadere toelichting en het insturen van input neem je contact op met het Zorginstituut via horizonscan@zinl.nl. De huidige Horizonscan is te raadplegen via www.horizonscangeneesmiddelen.nl.

ProQR, a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for QR-421a. QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of the vision loss associated with Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.

Usher syndrome is the leading cause of combined deafness and blindness. Exon 13 mutations in the USH2A gene targeted by QR-421a cause vision loss in approximately 16,000 individuals in the Western world. ProQR plans to start enrolling patients in a Phase 1/2 trial named STELLAR in the coming months with preliminary data expected in mid-2019.

“We are pleased to be advancing QR-421a, our second therapy for an inherited retinal disease, into the clinic and continuing the development of our portfolio of transformative RNA medicines for severe genetic rare diseases,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “QR-421a has shown promising activity in both the optic cup and zebra fish models and we are excited about the potential to make a meaningful impact for Usher syndrome patients.

Source: ProQR

Editas Medicine, a leading genome editing company, announced the U.S. Food and Drug Administration (FDA) has accepted the Company’s Investigational New Drug (IND) application for EDIT-101, an experimental CRISPR genome editing medicine being investigated for the treatment of Leber Congenital Amaurosis type 10 (LCA10).

“The FDA’s acceptance of our IND for EDIT-101 is a significant moment in the field of genome editing, and importantly, a critical milestone for patients, as we are now one step closer to a treatment for LCA10,” said Katrine Bosley, President and Chief Executive Officer, Editas Medicine. “This moment is a truly exciting one for us, and we look forward to embarking on our next chapter as a clinical stage company, harnessing the power of CRISPR technology to transform the lives of people with serious diseases around the world.”

With the IND acceptance, Editas Medicine has earned a $25 million milestone payment from Allergan as part of the alliance between the companies to discover and develop experimental ocular medicines targeting serious, vision-threatening diseases. Editas Medicine and its partner, Allergan Pharmaceuticals International Limited (Allergan), a wholly-owned subsidiary of Allergan plc, expect to enroll 10 to 20 patients in a Phase 1/2 open label, dose escalation study to evaluate the safety, tolerability, and efficacy of EDIT-101.

The Editas Medicine-Allergan Alliance
In March 2017, Editas Medicine and Allergan Pharmaceuticals International Limited (Allergan) entered a strategic alliance and option agreement under which Allergan received exclusive access and the option to license up to five of Editas Medicine’s genome editing programs for ocular diseases, including EDIT-101. Under the terms of the agreement, Allergan is responsible for development and commercialization of optioned products, subject to Editas Medicine’s option to co-develop and share equally in the profits and losses of two optioned products in the United States. In August 2018, Allergan exercised its option to develop and commercialize EDIT-101 globally for the treatment of LCA10. Additionally, Editas Medicine exercised its option to co-develop and share equally in the profits and losses from EDIT-101 in the United States. Editas Medicine is also eligible to receive development and commercial milestones, as well as royalty payments on a per-program basis. The agreement covers a range of first-in-class ocular programs targeting serious, vision-threatening diseases based on Editas Medicine’s unparalleled CRISPR genome editing platform, including CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a).

Source: Editas Medicine