Your Daily Pharma Scoop: Loxo Outstanding Results, Cimzia Trial Success, Audentes Positive Data

Analysis focus: LOXO

Loxo Oncology (NASDAQ:LOXO), which was recently covered in a series of excellent articles by our very own Dr. Zach Hartman, PhD, who collaborates with the Total Pharma Tracker and whose members saw an early view of the series (…and so on), is up strongly following preliminary results which showed that its new LOXO-292 experimental cancer drug led to tumor shrinkage in nearly 70% of patients regardless of where their cancer originated.

These results are surprisingly similar to earlier data from Loxos TRK inhibitor larotrectinib that was published in the New England Journal of Medicine, which, as Dr Hartman says, is a journal typically reserved for the highest-profile, practice-changing results. They only rarely publish phase 1 findings, since these studies generally do not produce results that provide definitive guidance for doctors.

That phase 1 data publication led to Bayer buying up this drug candidate for $400 million in upfront payments to LOXO, with another $650 million in potential milestones relating to development of their main drug larotrectinib and their other Trk inhibitor, LOXO-195.

This new drug, LOXO-292, also led to tumor shrinkage in nearly 70% of patients – and the most important thing? – this was regardless of where their cancer originated, as long as they had a certain genetic mutation in their RET genes.

RET fusions, an acquired rather than inherited gene defect, occur in about 2 percent of lung cancers, 10 to 20 percent of papillary thyroid cancers, and a small number of other cancers. Other mutations known as activating RET point mutations account for about 60 percent of medullary thyroid cancers, which comprise 3 percent of all thyroid cancers.

Those are outstanding numbers even compared to competitor Blueprint, whose competing RET drug saw an overall response rate of 37 percent, including 45 percent for NSCLC and 32 percent for medullary thyroid cancers. Compared to that, for LOXO-292, ORR was 65 percent in NSCLC, including three with brain metastasis, and 83 percent for patients with papillary thyroid cancer. For medullary thyroid cancers, ORR was 79%, with tumor shrinkage ranging from 9 to 45 percent.

Clearly, Dr. Hartman had it right when he said Loxo is a buy, even if he didnt even consider 292 at that time. This, as I see it, is an added bonus to his already strong Loxo thesis.

Stocks in News: Analysis of UCBJF, BOLD

UCB’s Cimzia successful in late-stage study in subtype of ankylosing spondylitis

Discussion: Cimzia, UCBs (OTCPK:UCBJF) drug candidate targeting patients with non-radiographic axial spondyloarthritis (patients with ankylosing spondylitis without x-ray evidence of an inflamed sacroiliac joint), demonstrated successful results in a phase 3 trial. Cimzia met its primary efficacy endpoint and showed that 47.2% of patients undergoing Cimzia treatment saw a significant improvement in symptoms at week 52 compared to 7.0% for placebo.Those are outstanding numbers, and looks likely that Cimzia, already currently approved in the U.S. for active ankylosing spondylitis, a type of arthritis primarily affecting the spine, as well as psoriatic arthritis and Crohn’s disease, will see a label expansion in this indication. In a study, 29 % of patients with IBP or inflammatory back pain met the criteria for nr-axSpA and 39 % of patients with CLBP or chronic low back pain had IBP.

Audentes reports positive results from study of AT132 in XLMTM

Discussion: Audentes Therapeutics (BOLD) announced that its phase 陆 trial of gene therapy AT132 in patients with X-Linked Myotubular Myopathy (XLMTM) met with continued positive results. XLMTM is an inherited disorder characterized by extreme muscle weakness, respiratory failure and early death. A total of 6 patients in the trial showed stable or increased neuromuscular function, and 5/6 saw improved respiratory function. However, adverse events may be a concern in future.

In other news

ASCO 2018, to be held in Chicago between June 1 and 5, has abstracts available here. We usually look for positive phase 2 data for taking new positions, and positive phase 3 data for signs of exit.

IQVIA (NYSE:IQV) is down on data integrity issues related to certain opioids. The FDA made an announcement that is has identified errors in the former’s National Sales Perspectives (NYSE:NSP) database. In a statement, IQVIA says it had previously identified the weight conversion based error and informed customers last month.

Amgens (NASDAQ:AMGN) Repatha has been approved with an expanded label in Europe for adults with established atherosclerotic cardiovascular disease (myocardial infarction, stroke or peripheral arterial disease) to reduce cardiovascular risk by lowering low-density lipoprotein cholesterol (LDL-C) levels. The CV outcome study that generated this data will help improve its market potential considerably.

Eiger (NASDAQ:EIGR) and Merck (NYSE:MRK) have expanded their license agreement to include rights to develop farnesyltransferase inhibitor lonafarnib for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria), a rare and fatal genetic condition characterized by accelerated aging in children. Eiger will be responsible for regulatory execution, commercialization and distribution activities of lonafarnib for Progeria.

Insmeds (NASDAQ:INSM) NDA for ALIS (Amikacin Liposome Inhalation Suspension) for the treatment of adult patients with nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex has been accepted by the FDA. The drug has both Breakthrough Therapy and Qualified Infectious Disease Product (QIDP) designations. PDUFA is on September 28.

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.

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