6-7 - News

Germany-based Bayer will pay $2bn initially to gain full rights to AskBio’s gene therapy platform, which includes an intellectual property portfolio and an established contract development and manufacturing organisation (CDMO).

On top of the upfront payment, Bayer will also pay up to an additional $2bn dependent on success milestones. In a statement, Bayer said that 75% of the potential success-based milestone payments are expected to be due over the course of the next five years, with the remaining amount due soon after that.

AskBio’s adeno-associated gene therapy platform has shown promise in a number of diseases, with the company’s lead research programmes focused on Pompe disease, Parkinson’s disease and congestive heart failure.

The company’s co-founder Jude Samulski is a gene therapy pioneer, whose early work included demonstrating that AAV could be cloned for therapeutic purposes.
Samulski also created two AskBio subsidiaries, Bamboo Therapeutics and Chatham Therapeutics, which focused on Duchenne muscular dystrophy and haemophilia respectively.

Since AskBio launched in 2001, the company has generated hundreds of proprietary third generation AAV capsids and promoters. These could potentially offer improved efficacy, immune response and tissues and
organ specificity.

In a statement issued on 21 October, the Department of Justice said that Purdue has agreed to plead guilty in a federal court to one count of dual-object conspiracy to defraud the US and violate the Food, Drug and Cosmetic Acts, as well as two counts of conspiracy to violate the Federal Anti-Kickback Statute.

Collectively, Purdue will face a $3.54bn criminal penalty as well as be expected to forfeit an additional $2bn. The company will also pay $2.8bn to settle the civil claims and an additional $225m in civil charges will be forfeited by the Sackler family, who privately own Purdue.

Purdue is expected to pay $225m of the $2bn forfeiture on the effective date of the company’s bankruptcy, which it filed for last year.

As part of the bankruptcy agreement, a new company is set to be formed which will be governed by a new board selected by the claimants and approved by the bankruptcy court.
No members of the Sackler family or the company’s executives or employees will face criminal charges as part of the settlement.

The new company will provide opioid overdose reversal medication, as well as addiction treatment medications at no or low cost.

Galápagos and development partner Servier announced that the drug failed to improve outcomes in the phase 2 ROCCELLA study of knee osteoarthritis patients and was unable to hit all secondary endpoints in the trial.

The drug was tested as three different once-daily oral doses in 932 patients with knee osteoarthritis over 52 weeks of treatment, however no dose was able to produce a significant difference compared to placebo.

Last year, Gilead announced a $5.1bn deal for access to Galápagos’ late-stage pipeline, and gained rights to GLPG1972 in the process.

At that time, Gilead agreed to an option fee for GLPG1927 worth $250m if the drug proved promising in the targeted osteoarthritis indication, as well as a further $200m related to endpoints in the phase 2b study and $550m in related milestones.

Following the disappointing results, Galápagos has lost out on the funds, with more pressure piled onto the other focal drug of the Gilead alliance – filgotinib.

However, earlier this year the US Food and Drug Administration (FDA) rejected Gilead’s submission for filgotinib for rheumatoid arthritis.

The regulatory authority said that it required additional data from two ongoing safety studies of the investigational drug, and raised concerns regarding the ‘overall benefit/risk profile of the filgotinib 200mg dose’.

The decision to advance clinical testing of prasinezumab is based on the results from the phase 2 PASADENA study. Although the study did not meet its primary objective of slowing the progression of motor and non-motor symptoms over the first year of treatment, the drug showed signs of efficacy on secondary and exploratory measures.

This included a reduction in disease progression in both prasinezumab arms compared to placebo. In addition, prasinezumab reduced the decline in motor function by 35% when compared to placebo after one year of treatment, using the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III.

Patients treated with prasinezumab also demonstrated a significant delay in time to clinically meaningful worsening of motor progression versus placebo over one year.
Prasinezumab is a monoclonal antibody designed to target alpha-synuclein, a protein found in neurons that can build up and spread from cell to cell. This results in the neuronal dysfunction and loss that ultimately leads to Parkinson’s disease.

In 2013, Roche and Prothena entered a collaboration agreement to develop and commercialise antibodies that target alpha-synuclein.

Mayzent (siponimod), a sphinogosine 1-phosphate receptor modulator, is the first oral disease-modifying treatment recommended by both NICE and the Scottish Medicines Consortium (SMC) for SPMS with active disease.

Novartis’ drug was first approved by the US Food and Drug Administration (FDA) in 2019 to delay disease progression in patients with SPMS. Mayzent then received approval from the European Commission (EC) earlier this year, again for the treatment of adults with active SPMS.

SPMS is typically characterised by an irreversible decline of neurological functions and there remains a high unmet need for safe and effective treatments to help delay the disability progression in this form of MS. According to NICE, around 11,000 people with SPMS will now be eligible to receive Mayzent treatment.

Initially, NICE turned down Mayzent due to a lack of clinical evidence for its clinical benefit in SPMS and also a lack of cost-efficiency evidence.

However, following changes made by Novartis to the costing of the drug in response to consultation on the initial draft guidance, NICE felt that the pharma company had addressed earlier concerns around Mayzent’s cost-effectiveness.

Regulators will need to approve the drug before it could be made more widely available.

People with Alzheimer’s experience a build-up of the protein amyloid in their brains at an early stage in the disease process. Aducanumab is an antibody designed to target this protein.

In May 2019, phase 3 clinical trials of aducanumab, ENGAGE and EMERGE were halted by Biogen after early indications suggested the drug would not be beneficial for those in the early stages of Alzheimer’s disease.

A larger amount of data that became available after the trials stopped led to new analysis, however, and resulted in Biogen filing for market approval with the US Food and Drug Administration (FDA) in August.
Clinical data from patients with Mild Cognitive Impairment due to Alzheimer’s disease and mild Alzheimer’s disease demonstrated that treatment with aducanumab resulted in the removal of amyloid beta and improved clinical outcomes.

The proposal was accepted by the FDA under its priority review process and a decision is expected by March 2021.
Following the EMA’s acceptance of the application to review the drug for use in people with Alzheimer’s disease in the EU, Biogen is planning to make the drug available to eligible participants from its earlier trials of aducanumab, as part of a new study.

Bayer pays an initial $2bn for gene therapy firm AskBio

Purdue to pay over $8bn to settle opioid-related criminal claims

Galápagos mid-stage trial failure raises concerns for Gilead deal

Roche and Prothena set to advance Parkinson’s disease candidate into phase 2b

Novartis’ MS therapy Mayzent approved by NICE

EMA reviewing Biogen’s potential Alzheimer’s drug for use in the EU