3 Things In Biotech You Should Learn Today: August 7, 2017

by: Zach Hartman

Roche's lung cancer drug heading for first-line approval?

TG Therapeutics lays out a plan with the FDA for multiple sclerosis.

AbbVie and Enanta score approval in hepatitis C infection.

Welcome to another edition of "3 Things In Biotech You Should Learn Today," a daily digest highlighting recent news from the biotechnology and pharmaceutical industries. The goal here is to help forward your due diligence and identify new potential opportunities.

So let's get started!

Roche's lung cancer drug heading for first-line approval?

Patients with non-small cell lung cancer (NSCLC) harboring rearrangements in the ALK gene have experienced an explosion of new therapeutic options, with two generations of approved small molecule inhibitors available to treat in the first line and in salvage therapy.

Pfizer's (NYSE:PFE) crizotinib was the first of these therapies to receive approval, but while it does help patients, development of resistance is almost inevitable. Furthermore, crizotinib does not pass the blood-brain barrier, so lung cancer that metastasizes to the brain often cannot be treated with this agent.

Roche's (OTCQX:RHHBF) alectinib and Novartis's (NYSE:NVS) ceritinib first showed significant benefit in patients with relapse following crizotinib therapy, including in those who develop resistant mutations and who have brain metastases.

Since then, one of the big movements in the space has been exploring these "second-generation" kinase inhibitors in the first line. NVS got the first approval here, thanks to good results from ASCEND-4, but RHHBF has not been far behind with positive findings from ALEX and J-ALEX.

Now RHHBF has announced that its supplemental NDA to the FDA has been submitted, and the FDA has given first-line alectinib priority review status. A decision is expected November 30.

Looking forward: If RHHBF does not get this approval, I will be flabbergasted. ALEX and J-ALEX give alectinib a potentially serious edge when it comes to competition, because these studies looked at alectinib compared with crizotinib in the first line setting, whereas ASCEND-4 compared ceritinib and chemotherapy. For ALK-positive lung cancer, crizotinib is the standard of care, full stop, so doctors will want to know that the drug they're giving is better, as assessed in a head-to-head trial. So it could be a huge inroad for RHHBF even though it is quite a ways behind NVS on that approval for first-line therapy.

TG Therapeutics lays out a plan with the FDA for multiple sclerosis

The clinical pipeline for TG Therapeutics (NASDAQ:TGTX) currently relies heavily on the success of its glycoengineered CD20 antibody ublituximab, which is being explored in hematologic malignancies and inflammatory conditions.

One of those diseases is multiple sclerosis, specifically the relapsing forms. CD20 therapy was recently brought to market by RHHBF with ocrelizumab, and now TGTX is looking to head into the home stretch of clinical development in this space.

On August 1, TGTX announced that it had reached an agreement with the FDA regarding a special protocol assessment (SPA) for two of its studies to be conducted in relapsing multiple sclerosis, ULTIMATE I and ULTIMATE II.

The SPA in essence outlines an agreement between the FDA and the company stating that the FDA recognizes that if the study meets its primary endpoint, that its statistical and experimental methods have been deemed sufficient for consideration of drug approval. In essence, the FDA has provided assistance to developing a true pivotal study.

Looking forward: It's difficult to gauge the impact of receiving an SPA, but it's definitely not bad news. Essentially, the FDA is giving TGTX the golden ticket to approval if it meets the objectives of the study. Fewer questions about its trial at the time of regulatory submission would hopefully mean an easier regulatory pathway. If this SPA helps to avoid a negative complete response letter down the line, then it will definitely have been worth the effort on the part of TGTX to have this discussion.

AbbVie and Enanta score approval in hepatitis C infection

AbbVie (NYSE:ABBV) has been a big player in the hepatitis C space for several years now with blockbuster combination therapies like Exviera. These approvals have been achieved in conjunction with work from its partner since 2006, Enanta (NASDAQ:ENTA).

Their newest entry into the space is a doublet therapy branded Mavyret, a ribovarin-free combination of glecaprevir and pibrentasvir for HCV infection across all six genotypes.

Recently, the companies announced that Mavyret has been approved by the FDA on the basis of data from nine different registrational studies covering all the major genotypes and patients with cirrhosis or chronic kidney disease. As a result, the approval covers therapy in adults with all six genotypes, and they may have no cirrhosis or mild cirrhosis, including patients with mild kidney disease.

Looking forward: The wide net gamble here looks like it paid off. Mavyret is the first eight-week treatment for genotypes 1 through 6, and genotype 1 in particular can be a challenge, necessitating further treatment options. In the heated world of treatment for these liver viruses, this should give ABBV and ENTA a pretty serious edge in the marketplace. Time will tell how well they're able to break in, though.

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