Biogen, Kite Pharma gain EMA hurry-up reviews

Biogen HQ

Biogen’s ($BIIB) Alzheimer’s candidate aducanumab and Kite’s ($KITE) much-hyped experimental CAR-T blood cancer drug KTE-C19 are some of the first drugs to gain a speedy review under a new system from the European Medicines Agency.

The EMA’s PRIME system (aka the Priority Medicines initiative)--which follows a similar philosophy of the FDA’s "breakthrough” tag--speeds up the review of new meds in disease areas with unmet medical need and allows companies more regulatory help along the way.

First up, Kite Pharma’s chimeric antigen receptor candidate KTE-C19 will be assessed under PRIME in patients with refractory diffuse large B-cell lymphoma (DLBCL).

Kite's drug is an investigational immunotherapy in which a patient's T cells are genetically modified to express a CAR designed to target the antigen CD19, a protein expressed on the cell surface of B cell lymphomas and leukemias.

There have been some stellar results in certain small trials in some blood cancer patients, which has caused much excitement ahead of the upcoming ASCO conference this week. Kite is in a race with Novartis ($NVS) and Juno ($JUNO), among others, to be the first to market this new class of oncology drug.

“We are honored to be among the first sponsors selected by the CHMP and CAT to participate in an innovative program that fosters development of therapies for patients with serious diseases that have no or only unsatisfactory therapeutic options,” said Arie Belldegrun, chairman, president, and CEO of Kite.

“The potential for KTE-C19 to address the substantial unmet medical need of patients with chemorefractory DLBCL is now recognized by designations from the U.S. and EU regulatory agencies. We look forward to working closely with the CHMP and CAT as we advance our multi-center ZUMA studies and move toward the KTE-C19 marketing authorization application process.”

The FDA has already granted its breakthrough therapy designation to KTE-C19 for patients with DLBCL, primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL) last year.

And it’s not the only biotech to gain this new accelerated review, as Biogen has also announced its experimental Alzheimer’s drug aducanumab will also be looked at under the PRIME program.

“Alzheimer’s disease is a debilitating condition affecting a growing number of patients and their loved ones, and there is an urgent need for new effective treatment for this disease,” said Alfred Sandrock, EVP and chief medical officer at Biogen.

“Aducanumab’s acceptance into the PRIME program is a significant benefit to its development and to the European Alzheimer’s disease community. We look forward to collaborating with the EMA on development plans and potential accelerated assessment of aducanumab with the hope of bringing effective treatment to patients as soon as possible.”

Aducanumab is currently being tested in two global Phase III studies, ENGAGE and EMERGE, which are designed to evaluate its safety and efficacy in slowing cognitive impairment and the progression of disability in people with early Alzheimer’s disease.

The drug is a human recombinant mAb derived from a de-identified library of B cells collected from healthy elderly subjects with no signs of cognitive impairment, or cognitively impaired elderly subjects with unusually slow cognitive decline. Biogen licensed aducanumab from Swiss biotech Neurimmune.

Aducanumab is believed to target forms of beta amyloid including soluble oligomers and insoluble fibrils deposited into the amyloid plaque in the brains of AD patients. Based on preclinical and interim Phase Ib data, treatment with aducanumab has been shown to reduce amyloid plaque levels, according to Biogen.

But the drug is a risk--not least because almost every new AD candidate in the past decade has failed in late-stage studies--with many in Big Pharma stepping back from the area or re-assessing their R&D programs for AD.

And Biogen has already hit roadblocks in testing, and just last year its 6-mg dose of aducanumab failed to deliver what the Big Biotech and anxious analysts were hoping to see, falling short of clinical significance on two key measures of efficacy that would have pointed toward a clear path ahead in a pivotal study.

And the safety data on the drug also worsened, with a heightened indication of toxicity that could spell trouble for the drug's future. It did, however, release positive data for a Phase I trial in March of last year, but longer term testing up until the end of the decade will need to be done to prove long-term efficacy and remove concerns over safety.

The specific criteria for the Priority Medicines initiative, which began in earnest earlier this year, require early clinical evidence showing that a new drug offers a “therapeutic advantage” over existing treatments--or benefits patients without treatment options.

On a practical level, this designation also provides appointment of a rapporteur, early dialogue and scientific advice at key development milestones, and the potential to qualify products for accelerated review earlier in the application process.

This does not, however, guarantee success or that these drugs will be pushed through the EMA immediately--but it may shave a few months off the regulator’s normal review time once a drug has been sent for assessment, and potentially help a company better align its trials and data with what the EMA will want to see to give an approval.

- see more about EMA’s PRIME program
- check out Kite’s release
- read Biogen’s statement

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