3 Things In Biotech You Should Learn Today: July 5, 2017

by: Zach Hartman

Amgen widens its biologic's applicability in colorectal cancer.

BioMarin and Merck advance to the last leg of the race in phenylketonuria.

SteadyMed also sees a finish on the horizon in pulmonary arterial hypertension.

Welcome to another edition of "3 Things in Biotech You Should Learn Today," a daily digest dedicated to helping you keep track of all the recent findings in pharmaceutical and biotech research.

And today we're considering all kinds of optimistic news, all you need to beat a post-holiday back-to-work funk!

Amgen widens its biologic's applicability in colorectal cancer

The treatment of metastatic colorectal cancer has continued to evolve in recent years with the approval of various agents and a shift in clinical mindset toward molecular testing for a wider array of mutations, especially in a bigger subset of RAS genes.

Amgen's (NASDAQ:AMGN) anti-EGFR antibody panitumumab (branded Vectibix) was originally approved by the FDA for patients with recurrent, KRAS wildtype (not mutated) colorectal cancer following progression on chemotherapy.

Now, panitumumab has been given the nod by the FDA for first-line treatment in combination with doublet chemotherapy (the FOLFOX regimen). This approval also includes the first multigene, next generation-based test to identify the mutational status of RAS in the tumor, as patients need to have normal KRAS and NRAS to have maximal chances of responding to panitumumab.

Looking forward: Combining chemotherapy and panitumumab in the first line for RAS wildtype patients yielded impressive progression-free and overall survival benefits, offering a new standard of care for patients with metastatic colorectal cancer. It remains to be seen exactly what should be done for patients with mutant RAS, but it is clear the EGFR antibody therapy is not part of that picture.

BioMarin and Merck advance to the last leg of the race in phenylketonuria

Phenylketonuria is an inherited disorder whereby patients are unable to metabolize the amino acid phenylalanine effectively, which can have serious consequences for development.

Typically, the condition is managed by strict dietary regulation to limit the amount of phenylalanine patients consume. However, there is currently only one approved therapy for this condition: BioMarin's (NASDAQ:BMRN) Kuvan, a tetrahydrobiopterin analog that stimulates residual activity of the enzyme responsible for metabolizing phenylalanine.

BioMarin, in partnership with Merck (NYSE:MRK), has also been developing an alternative therapeutic approach to phenylketonuria: modified recombinant phenylalanine ammonia lyase, also called PEG-PAL. PEG-PAL is meant to replace the metabolic function of phenylalanine hydroxylase, assisting in the metabolism of phenylalanine.

Based on favorable clinical trial findings, BMRN announced that it has filed an NDA with the FDA to seek approval for PEG-PAL. This is a key step in getting through the last regulatory hurdle before approval.

Looking forward: PEG-PAL offers a fresh take on the management of phenylketonuria, by directly supplementing the body's ability to metabolize phenylalanine. The next step for BMRN will be for the FDA to provide a response, which will set up the review window. The prospects looks promising for PEG-PAL, as clinical trial results have shown that it can help control developmental and symptomatic features of the disease.

SteadyMed also sees a finish on the horizon in pulmonary arterial hypertension

Pulmonary arterial hypertension is a serious disorder characterized by heightened blood pressure in the pulmonary arteries (hence the name of the disease). While agents like vasodilators and anticoagulants can help control the symptoms of this disorder, treatment is typically limited in efficacy.

One of the more recent developments for treating pulmonary arterial hypertension has been the introduction of prostacyclin analogs, which promote potent vasodilation.

SteadyMed (NASDAQ:STDY) is looking to be the latest in a large crowd of prostacyclin analogs, as the company announced submission of its NDA for Treyvent, which offers potential advantages in terms of drug delivery for patients.

Looking forward: Though this is an increasingly crowded field, Treyvent offers a few distinguishing features relating to drug delivery and convenience for patients. Pulmonary arterial hypertension continues to be an unmet need in the United States, so every option is welcome. We'll have to wait and see if STDY can capitalize on potential approval!


So, today is all about drug approvals and submissions of promising therapies for serious conditions. It's always fun to cover nothing but optimistic findings.

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Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.