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Topline Abstract
Kura Oncology (NASDAQ:KURA) is an oncology-focused biotech creating a number of completely different sorts of focused remedy for various types of most cancers with excessive unmet want. On this article, I present a synopsis of their pipeline, in addition to the justification for my tentative optimism within the firm. General, I feel they’re value , arduous look, and a consideration of a small place in your portfolio, if you do not have one already.
Pipeline Overview
Ziftomenib
The present principal declare to fame for KURA is ziftomenib, an inhibitor of an rising goal referred to as menin, which has demonstrated key potential within the setting of acute myeloid leukemia. Particularly, Syndax’s revumenib has generated a whole lot of curiosity within the house of AML for sufferers who harbor mutations within the KMT2A gene.
KURA is focusing growth of ziftomenib on the subset of AML harboring NPM1 mutations, and the final we heard about this method was at EHA 2023 again in June, the place the corporate introduced a late-breaking abstract demonstrating a 35% fee of full remission in sufferers who acquired the advisable part 2 dose of ziftomenib. The length of response was reportedly 8.2 months, and ziftomenib confirmed early exercise in sufferers with different actionable co-mutations like FLT3 and IDH1. Though it ought to be famous that these have been very small affected person subsets.
The registrational portion of this trial (KOMET-001) began back in February 2023, and within the newest quarterly submitting they indicated that enrollment is continuing effectively and is anticipated to be accomplished within the first half of 2024. Provided that median follow-up within the part 1 examine was 8.8 months, I imagine it’s affordable to count on a knowledge readout both at EHA 2024 or ASH 2024, and KURA believes this part 2 examine can have “registrational potential.”
KURA can also be conducting a number of mixture trials of ziftomenib and different brokers like venetoclax, azacitidine, and chemotherapy within the KOMET-007 and KOMET-008 research, from which the corporate expects to have information within the first half of 2024. No outcomes have been posted up to now.
Tipifarnib
This farnesyl transferase inhibitor has been in growth for a very long time, having first been licensed from Janssen again in 2015. Farnesyl transferase is chargeable for shuttling Ras protein to its correct location within the cell throughout sign transduction, and it is thought that inhibiting this enzyme might be one backdoor to inhibiting Ras perform as a most cancers goal.
Thus far, not one of the tales for tipifarnib have panned out, however KURA is continuous on with one thing promising. They at the moment have Breakthrough Remedy Designation for tipifarnib within the setting of HRAS-mutated head and neck squamous cell carcinoma (HNSCC), a type of most cancers that is still a critical unmet want.
Certainly, they appear to have damaged by means of with tipifarnib. In a late-breaking abstract at ESMO 2023, KURA highlighted findings from 59 sufferers with HRAS-mutated HNSCC. Of the 38 sufferers who had evaluable illness, independently assessed responses have been proven in 10 sufferers (1 with full response). The median length of response by unbiased evaluation was 6.5 months.
This response fee is normally settlement with findings of other second-line studies, which present responses in simply over 10% of sufferers. Toxicity led to discontinuation in 7% of the sufferers enrolled within the examine. KURA has not guided on a timeline for initiating a pivotal trial or a attainable path to approval, however these findings do present a sign of efficacy for farnesyl transferase inhibitors within the setting of chosen subgroups of HNSCC. They’re additionally conducting a notable part 1/2 trial of tipifarnib together with Novartis’s PI3K inhibitor alpelisib in sufferers with PIK3CA-amplified or -mutated HNSCC.
KO-2806
Constructing on tipifarnib, KURA can also be creating a next-generation farnesyl transferase inhibitor, with the intention of enhancing varied pharmacokinetic and pharmacodynamic properties. The IND for KO-2806 was approved in January 2023, and the monotherapy trial had its first affected person handled back in October.
Additionally of observe, KURA introduced a collaboration with Mirati Therapeutics to mix KO-2806 and the KRAS G12C inhibitor adagrasib in sufferers with KRAS G12C-mutated non-small cell lung most cancers, signaling a possible future pathway for this agent.
Monetary Overview
Per their Q3 2023 submitting, KURA held $28.7 million in money and equivalents, with one other $423.9 million in short-term investments. In the meantime, working bills reached $42.5 million for the quarter, up from $36.5 in the identical interval of 2022. After curiosity and different revenue, the corporate realized a internet lack of $38.6 million.
With these reserves, KURA has an operational runway of 10-11 quarters.
Strengths and Dangers
KURA presents an fascinating funding case, with favorable early trial ends in two very completely different focused remedy areas. With tipifarnib, there aren’t actually any significant rivals. For ziftomenib, nevertheless, there are fairly a number of firms vying for the crown of menin inhibition. However the outcomes of menin inhibition are wanting adequate on the whole to assist this as a therapeutic method in AML, and a possible drug approval can be a internet optimistic, even within the face of competitors.
The money reserves of KURA additionally signify a power, in my view. With effectively over a yr of runway, this ought to be loads of time to see some crucial scientific trial outcomes and a attainable path to accelerated approval demystified.
Backside-Line Abstract
KURA is coming into a interval of crucial mass so far as scientific trial information go, they usually’ve received the money to assist their efforts for the foreseeable future. This makes me optimistic general about their prospects, and I’d give it a “purchase” advice. As all the time, take into accounts that there’s a excessive danger of drug growth failure, even when issues look promising in these early levels. Moreover, there isn’t a assure that the research they’re conducting will find yourself being registrational, at which level we’ll want to attend a for much longer time for confirmatory, randomized trials to be accomplished. Nonetheless, there may be a lot room for additional development for KURA.