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Cancer Institute Head Named Acting FDA Director

March 18, 2019

National Cancer Institute Director Norman “Ned” Sharpless will serve as acting commissioner of the FDA following Scott Gottlieb’s departure next month.

An oncologist who holds numerous medical patents, Sharpless brings a broad background that includes cofounding two clinical stage biotech firms, G1 Therapeutics and HealthSpan Diagnostics.

Sharpless is also chief of the National Institute on Aging’s Laboratory of Genetics and Genomics’ Aging Biology and Cancer Section.

During his tenure at the NCI, Sharpless advocated for increased modernization of clinical trials as well as increased funding for academic investigators. He also oversaw several collaborative ventures with the FDA, including research and development for childhood cancer drugs.

European Panel Offers Advice on Complex Drug Trials
Drug sponsors hoping to make use of complex clinical trials that test for multiple diseases or test multiple treatments for single diseases need to explain the “overarching hypothesis” behind their protocol designs, according to a key European regulatory committee.

Complex — often called adaptive — trial protocols should “describe how the objectives specific to each sub-protocol contribute to the whole trial objective(s),” the Heads of Medicine Agencies (HMA) clinical trials advisory committee said in recommendations designed to get drugmakers and regulators thinking about the next evolution in drug development. HMA is a network of the heads of regulatory authorities for drugs in the European Economic Area, which collaborates with the European Medicines Agency and the European Commission.

Efficiency or speed “cannot be the only justification” for an adaptive design, the advisory committee said, adding that sponsors “should discuss the rationale of the specifically proposed clinical trial design and not the advantages of complex clinical trials in general.”

The 15-page document lays out eight broad goals for complex trial design:

  1. Clear descriptions and justifications for the unorthodox approaches;
  2. Scientific integrity;
  3. Quality “trial conduct” that also maximizes clinical feasibility;
  4. Patient safety;
  5. Data integrity;
  6. Continual risk/benefit analyses at critical steps through clinical trials;
  7. New and valid companion diagnostics; and
  8. Data transparency.

Read the HMA committee recommendations here: https://bit.ly/2HnhLqr.

Health Canada Launches Searchable Trials Database
Canadian regulators have opened a new website that will allow the public to search clinical trial data on drugs and devices.

The website will start with “a small number of products” that sponsors have volunteered data on, but the site ultimately will offer the public trial records for every drug and device submitted for approval, with a goal of having the data available within 120 days of a sponsor’s submission.

The database will begin with drug products, and devices should be added by 2021. It’s not clear when the regulations will formally take effect: Health Canada has promised to publish new rules this week.

Biogen Takes on Vision Disorder Portfolio with Nightstar Acquisition
Biogen has reached an agreement to buy London-based Nightstar Therapeutics for approximately $800 million.

Under the terms of the agreement, expected to close by mid-2019, Biogen will acquire a portfolio that includes the smaller drugmaker’s lead asset, a treatment for the rare degenerative X-linked retinal disorder choroideremia currently undergoing clinical trials. The condition, which currently has no approved treatments, disproportionately affects males and eventually causes blindness through a loss of function in the CHM gene, which encodes the enzyme REP-1.

A Phase I/II trial demonstrated that the product, NSR-REP1, could slow declines in visual acuity, and is currently being evaluated in a late-stage trial projected to read out by the second half of 2020.

Biogen also will take on trials of Nightstar’s treatment targeting a second rare, inherited retinal disease with no approved treatments, X-linked retinitis pigmentosa (XLRP).

Both therapies are estimated to generate up to $200 million in annual sales.

FDA Pulls Fast-Track Designation for Tonix’s PTSD Treatment
The FDA revoked its breakthrough therapy designation for Tonix’s posttraumatic stress disorder drug Tonmya (cyclobenzaprine HCl sublingual tablets), citing disappointing interim data from a clinical trial.

The agency said it withdrew the designation because data from the sponsor’s HONOR study didn’t support continued fast-tracking. The study, which tested the drug in military-related PTSD, was halted last July because of “inadequate separation from placebo on the primary endpoint at week 12,” the drugmaker said.

Tonix said that the agency’s decision to void its breakthrough designation would not interrupt the company’s drug application plan.