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First EU PRIME Designations Go To Biogen, ChemoCentryx, Kite, NovImmune

Executive Summary

The first four drugs European Medicines Agency selects for its new priority review program include Biogen's Alzheimer's disease treatment aducanumab. Fourteen applicants did not garner PRIME status.

Biogen's aducanumab for early Alzheimer's disease is one of the first four drugs to be accepted into the European Medicines Agency's PRIME (priority medicines) scheme, which offers early, proactive and enhanced support for the development of products that address unmet medical needs, as well as the possibility of accelerated assessment.

The other three products now eligible for EMA support under PRIME are ChemoCentryx Inc.'s CCX168 for valvulitis, Kite Pharma Inc.'s KTE-C19 for lymphoma, and NovImmune SA's NI-0501 for hemophagocytic lymphohistiocytosis (HLH).

Biogen said that aducanumab's acceptance into the scheme was "a significant benefit to its development and to the European Alzheimer's disease community," and that it looked 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."

PRIME is intended mainly for products at an early stage of development, either at the proof-of-concept stage or, for smaller firms, at proof-of-principle stage. Biogen's aducanumab is further advanced – it is entering global Phase III studies – but the company said it was accepted into PRIME on the basis of its Phase Ib placebo-controlled study in patients with prodromal or mild Alzheimer's disease.

Two Phase III trials with the drug are now under way and recruiting for patients: ENGAGE and EMERGE, which are being conducted in centers in the US, Canada, Europe, Australia and Asia to assess the efficacy and safety of aducanumab in slowing cognitive impairment and the progression of disability in people with early Alzheimer's.

Biogen Chief Medical Officer Alfred Sandrock acknowledged in January this year that recruitment into the studies could take some time because of the inherent challenges in recruiting patients with early Alzheimer's and the limited availability of PET scanners. He added that there had been substantial interest in the aducanumab program because of the "positive" Phase I data released last year (Also see "Biogen Senses 'Interesting Opportunities' If Financial Markets Remain Challenging" - Pink Sheet, 27 Jan, 2016.).

Launched March 7, PRIME is intended to foster R&D into new medicines that have the potential to address an unmet medical need, i.e., that offer a major therapeutic advantage over existing treatments, or benefit patients with no current treatment options, using existing regulatory tools such as accelerated assessment.

Eligibility for the PRIME scheme will depend on the availability of adequate non-clinical and exploratory data to justify a potential major public health interest prior to the initiation of confirmatory clinical studies at proof of concept stage (i.e., prior to confirmatory clinical studies).

The CHMP examined a total of 18 substances submitted for PRIME as of April 6 and turned down 14. Of the four products that were recommended for assessment under the scheme, two already have breakthrough designation in the US, a mechanism that, like PRIME, is also intended to ensure timely patient access to innovative new drugs.

Details of the other three substances entering PRIME are as follows:

  • ChemoCentryx' CCX168 is an orphan drug for active ANCA-associated valvulitis. The company said that positive results from the European Phase II CLEAR (C5aR inhibitor on Leukocytes Exploratory ANCA-associated Renal Vasculitis) trial were announced in January 2016. "With these results, combined with information from our ongoing CLASSIC (Clinical ANCA vasculitis safety and efficacy study of inhibitor of C5aR) trial, we plan to conduct our US and European regulatory meetings in the middle of 2016, and then to initiate a Phase III registration trial by the end of 2016," it added.
  • Kite Pharma's KTE-C19 received US breakthrough status in December 2015. It is being tested in adults with diffuse large B-cell lymphoma (DLBCL) who have not responded to prior therapy, or who have had disease progression after autologous stem cell transplant (ASCT). The company announced at the end of May that updated data from the Phase I portion of the KTE-C19 ZUMA-1 study in chemorefractory aggressive disease would be presented at the ASCO meeting on June 3-7, as would a poster on the study design for ZUMA-4, an ongoing Phase II study in children and young adults with previously treated acute lymphoblastic leukemia.
  • NovImmune's NI-0501 is an anti-interferon-gamma MAb for primary hemophagocytic lymphohistiocytosis (HLH). The product has US and EU orphan status and was also granted US breakthrough designation in March this year on the basis of clinical data from a Phase II trial in children with primary HLH. Preliminary data from the Phase II study were presented at the American Society of Hematology meeting in Orlando, US, last year. HLH is a hyperinflammatory syndrome characterized by uncontrolled and aberrant activation of the immune system and a life-threatening cytokine storm presenting with non-remitting fever, pancytopenia, coagulopathy and hemophagocytosis, potentially leading to death, NovImmune noted.

The 14 candidates turned down for PRIME at the CHMP meeting were for various indications in oncology, infectious diseases, pneumology-allergology, vaccines, cardiovascular, and ophthalmology. Three of them were advanced therapy medicines.

The EMA said that it assessed all PRIME applications taking account of the available treatments for the target disease, the stage of development of the product, and the data presented. It did not say why the 14 had been turned down, although an EMA official told a conference in London recently that two-thirds of the 18 applications were lacking a pediatric investigation plan (PIP) or a PIP waiver request.

"The fact that a medicine is not accepted in PRIME does not mean that its development should not be pursued," the agency said. "Medicines that are not granted PRIME access can still benefit patients by providing alternative treatment options for a disease."

The EMA added that another 14 applications for PRIME have been received and are now being processed. In future, it said, it will release information on PRIME on a monthly basis following each plenary meeting of the CHMP.

The information will include the substance name, the substance type (e.g., biological, chemical, advanced therapy), the therapeutic area, the kind of data supporting the applications (usually non-clinical and exploratory clinical), and the type of applicant – generally SME (small/medium-sized company) or "other."

[Editor's note: This article is also published in Scrip Regulatory Affairs. The Pink Sheet brings selected complementary coverage from sister publications to our readers.]

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