Ferrer, an international pharmaceutical company focused on pulmonary vascular and interstitial lung diseases and neurological disorders, and Treeway, a clinical-stage biotechnology company, announced they have entered into a licence agreement for the development and commercialisation of an oral formulation of edaravone (TW001/FNP122)[i] for Amyotrophic Lateral Sclerosis (ALS) in certain territories, including Europe and some Asian countries. Under the terms of the licence agreement, Ferrer is responsible for advancing the development and commercialisation of the product and will make royalty payments to Treeway at certain milestones.

The deal leverages Ferrer’s strong capabilities and expertise in neurology to underscore both companies’ commitment to expanding global access to TW001/FNP122 for the treatment of ALS. 

Inez de Greef, CEO at Treeway comments: “Guided by patients, we have developed TW001/FNP122 through the early stages of development. We are therefore delighted that Ferrer has committed to advance our lead product to the required pivotal phase III trial and make it available for ALS patients in large parts of the world.”

Oscar Pérez, Ferrer’s Chief BD&L Officer, adds: “Driven by our purpose of bringing transformative products to patients over the world, we are pleased to enter the agreement with Treeway and look forward to patients benefiting from improved treatment options. TW001/FNP122 is the perfect fit for Ferrer’s strategy to bring new molecules to late-stage clinical development, registration and market entry in the field of neurological disorders and strengthen our pipeline for the coming years.” 

The agreement comes into play with immediate effect.

Source: Press release (Treeway)

Read more (In Dutch):
https://fd.nl/bedrijfsleven/1416633/biotechbedrijf-treeway-is-stap-dichter-bij-medicijn-tegen-als-juj1cafpsCxg

Cergentis, a genomics company that develops and commercializes kits and services based on its proprietary Targeted Locus Amplification (TLA) technology, today announced the appointment of Joris Schuurmans as Chief Executive Officer and Dirk Pollet and Maja Sanders as Supervisory Board members. Novalis Biotech Incubation will invest in Cergentis’ growth potential.

Incoming CEO Joris Schuurmans has extensive experience in the molecular diagnostics and biopharmaceutical industry. He held senior executive positions at Biocartis and MyCartis, and recently lead SkylineDx’ melanoma program prior to transitioning to his role at Cergentis.

Joris Schuurmans, newly appointed CEO of Cergentis, commented: “I am very excited by the opportunity to join Cergentis at this pivotal stage. The company has developed an impressive array of products and services in the field of genetic engineering, and is extremely well-positioned to enter the molecular diagnostics field with products that elucidate DNA variants in tumors that cannot be well-analyzed with current technologies. We are all excited to embark on our mission to improve the diagnosis and treatment of cancer patients.”

Dr. Jan Groen, Chairman of the Board: “A heartfelt gratitude to outgoing CEO Jan Dekker, who laid the foundation for Cergentis to take the next step to accelerate the development and commercialization of its unique TLA technology for cancer diagnostics. We are pleased to welcome Joris as new CEO. Joris has an excellent international track record in the biotech industry. We are also very pleased with the appointment of our two new Board members Maja and Dirk. Both are industry veterans and experienced Board members.”

Dr. Dirk Pollet is currently CEO of Enzyre, and heralded the acquisition of Multiplicom by Agilent as the CEO of Multiplicom. Maja Sanders currently holds supervisory roles at various non-profit and for-profit organizations, and has built up extensive managerial experience in the private equity industry.

Dr. Jan Groen, Chairman of the Board: “Dirk and Maja are valuable additions to the Board and we are pleased they join us in these exciting times. As Cergentis has already established a strong position it the support of drug development, we are looking forward to working together to support the company in further expansion in the cancer diagnostics field.”

Novalis Biotech Incubation to invest in Cergentis

Novalis Biotech Incubation (www.novalis.is), a Belgium venture capital investment fund, is investing in Cergentis.

Wim Van Criekinge, Co-founder of Novalis, and Board member of Cergentis added: “We are pleased to invest in Cergentis. Their proprietary Target Locus Amplification (TLA) technology is unique and allows the most advanced genome characterization to date”.

Joris Schuurmans, CEO of Cergentis: “We are delighted that Novalis has decided to invest in Cergentis. We appreciate their trust in our company and their support in our mission to improve the quality of genetic research and human healthcare worldwide.”

Source: Cergentis (press release)

AveXis, a Novartis company, today announced the European Commission (EC) granted conditional approval for Zolgensma^® (onasemnogene abeparvovec) for the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene. The approval covers babies and young children with SMA up to 21 kg according to the approved dosing guidance.

In Europe each year, approximately 550–600 infants are born with SMA, a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.^1,2,3 Zolgensma is a one-time gene therapy designed to address the genetic root cause of the disease by replacing the function of the missing or nonworking SMN1 gene. Administered during a single, intravenous (IV) infusion, Zolgensma delivers a new working copy of the SMN1 gene into a patient’s cells, halting disease progression. According to Pediatric Neuromuscular Clinical Research (PNCR) natural history study of SMA, almost all patients under the age of five years of age will be under 21kg with some patients at 6, 7 or 8 weighing below 21 kg.⁴ AveXis is planning a product presentation that allows for treatment of patients weighing up to 21 kg and is working with the European Medicines Agency (EMA) to finalize supply timelines. 

“The EC approval of Zolgensma is a significant milestone for the SMA community, and further underscores the substantial clinical value of the only gene therapy for SMA, bringing new hope to those impacted by this rare, but devastating disease.” said Dave Lennon, president of AveXis. “Even under the current pandemic conditions, the urgent need to treat SMA has resulted in access pathways in France and Germany for Zolgensma, a potentially life-saving medicine delivered in a single dose. Additionally, we have met with more than 100 stakeholder organizations across Europe to discuss our “Day One” access program to enable rapid access with customizable options designed to work within local pricing and reimbursement frameworks.”

SMA is a significant burden to the healthcare system in Europe with cumulative estimated healthcare costs per child ranging between €2.5 to €4 million within the first 10 years alone.⁵ Zolgensma is a transformative and highly innovative one-time gene therapy for a devastating and progressive genetic disease and is consistently priced worldwide under a value-based framework, however final pricing and reimbursement decisions are determined at the local level. Designed to work within existing, local pricing and reimbursement frameworks, the “Day One” access program offers ministries of health and reimbursement bodies a variety of flexible options that can be implemented immediately to support swift access and broad reimbursement.

The “Day One” access program ensures the cost of patients treated before national pricing and reimbursement agreements are in place align with the value-based prices negotiated following clinical and economic assessments. The program maintains the integrity of the local pricing and reimbursement frameworks with a variety of customizable options including:

• Retroactive rebates ensuring early access costs are aligned with negotiated prices following local clinical and economic assessment processes
• Deferred payments and installment options allowing reimbursement bodies to manage budget impact during the early access phase
• Outcomes-based rebates negotiated following clinical and economic assessments can be applied to patients treated during the early access period
• Robust training for treating institutions on administration and follow-up care
• Access to RESTORE, a global registry of patients who have been diagnosed with SMA that draws upon existing country registries

Immediate access to Zolgensma, aligned to the label, is available in France through the ATU framework and expected shortly in Germany.

“Today’s approval brings tangible progress in harnessing the transformational power of gene therapy,” said Dr. Eugenio Mercuri, Professor, Pediatric Neurology, Catholic University, Rome, Italy. “The approval of Zolgensma represents an important new way for physicians to treat patients with SMA. The results we have seen for Zolgensma to date from the STR1VE clinical trial show an impressive survival rate at the conclusion of the study, with the majority of patients achieving functional milestones, like sitting without support, that wouldn’t have been reached in untreated infants.”

“SMA Europe receives with deep excitement the news on the approval by the European Commission, of a gene therapy for treating a part of our community,” said Mencía de Lemus, President of SMA Europe. “Many hopes have been put into this much awaited therapy. It will be now be up to all stakeholders involved to ensure that treating doctors, together with parents, can take the best therapeutic option based on the benefit that each of them can provide to each individual. Gathering more data on how Zolgensma impacts in the lives of patients will be extremely important to better understand the potential of this new therapy on improving lives of those living with SMA.”

The EC approval is based on the completed Phase 3 STR1VE-US and Phase 1 START trials that evaluated the efficacy and safety of a one-time IV infusion of Zolgensma in symptomatic SMA Type 1 patients <6 months of age at dosing, who had one or two copies of the SMN2 backup gene, or two copies of the SMN2 backup gene, respectively. STR1VE-EU, a comparable Phase 3 study is ongoing. Zolgensma demonstrated prolonged event-free survival; rapid motor function improvement, often within one month of dosing; and, sustained milestone achievement, including the ability to sit without support, crawl and walk independently – milestones never achieved in untreated Type 1 patients.⁶

Additional supportive data included interim results from the ongoing SPR1NT trial, a Phase 3, open-label, single-arm study of a single, one-time IV infusion of Zolgensma in pre-symptomatic patients (<6 weeks at age of dosing) genetically defined by bi-allelic deletion of SMN1 with 2 or 3 copies of SMN2. These data demonstrate rapid, age appropriate major milestone gain, reinforcing the critical importance of early intervention in SMA patients.⁵

The most commonly observed side effects after treatment were elevated liver enzymes and vomiting. Acute serious liver injury and elevated aminotransferases can occur. Patients with pre-existing liver impairment may be at higher risk. Prior to infusion, physicians should assess liver function of all patients by clinical examination and laboratory testing. And, they should administer systemic corticosteroid to all patients before and after treatment, and then continue to monitor liver function for at least 3 months after infusion.⁶ There is limited experience in patients 2 years of age and older or with body weight above 13.5 kg. The safety and efficacy of Zolgensma in these patients have not been established.

AveXis has an exclusive, worldwide license with Nationwide Children’s Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Généthon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

Source: Avexis (press release)