First up was an announcement that the company had successfully completed a Phase 1 clinical trial for Kevetrin in advanced solid tumors. "The open-label, dose escalation study met Cellceutix's objectives by demonstrating the safety and tolerability of Kevetrin, while providing key information on the pharmacokinetics (method of action)," the company's Feb. 16 press release states.
In a follow-up release days later, the company disclosed additional data from the trial showing that Kevetrin demonstrated the ability to activate p53, a "tumor suppressor protein [also known as] the 'Guardian Angel of the Human Genome.'" Scientists have long recognized the potential benefits a drug acting upon p53 could have in treating cancer, according to Cellceutix, but, as of yet, no company has been able to accomplish the feat without excessive toxicity. Heading into Phase 2 studies, Cellceutix has become a global leader in the space, with the Phase 1 data confirming a strong safety profile for Kevetrin, according to Ehrlich.
In conversation with The Life Sciences Report, Cellceutix CEO Leo Ehrlich elaborated on the promise Kevetrin holds for cancer treatment. p53, he explained, has the ability to repair damaged cells or, if the damage cannot be repaired, cause apoptosis (cell death), thus eliminating the uncontrolled cell proliferation that defines cancer. In healthy people, p53 does the job well; in cancer patients, it does not, Ehrlich said. That Kevetrin has demonstrated an ability to increase p21, a downstream marker indicating activation of p53, raises the potential that the compound could not only "stabilize, but be an important component to help defeat the disease." Data from the study showed increased p21 expression compared to baseline in 67.5% (27 out of 40) of evaluable patients, regardless of tumor type, which the company notes is encouraging data that sets the scene for advanced studies.
Initially, the market reacted to this news as though it were a "whisper," when it could represent an "earthquake," Ehrlich said.
Kevetrin, which has been granted orphan drug designation by the FDA for ovarian cancer and retinoblastoma, was assessed in patients with advanced solid tumors at the Dana-Farber Cancer Institute and the Beth Israel Deaconess Medical Center. Cellceutix plans to file for a Phase 2 trial in ovarian cancer next month. Because Kevetrin's safety was demonstrated in the Phase 1 study, and pharmacokinetic data shows a very short half-life, the company expects that patients in the Phase 2 study will receive multiple doses of Kevetrin weekly, which it is believed will enhance efficacy.
"Kevetrin could not only stabilize, but be an important component to help defeat the disease."
"Kevetrin hits the tumors hard and fast and then exits the body; that's one of the beautiful attributes of this novel compound and what helps minimize toxicity," explained Ehrlich. "We saw some signs of tumor reduction with only one dose of Kevetrin per week in the Phase 1 study, while we better defined the safety profile. Armed with this data, we are going to get more aggressive at targeting tumors multiple times per week in Phase 2 research."
Ovarian cancer is a natural fit for a p53-targeting drug, according to Cellceutix. Data shows that damage to p53 is most frequently observed in ovarian cancer, suspected in playing a role in disease progression in about 95% of cases.
Elsewhere in the pipeline, on Feb. 26, the company submitted a special protocol assessment (SPA) request to the FDA for a Phase 3 trial of its single-dose antibiotic compound, brilacidin, in acute bacterial skin and skin structure infection (ABSSSI) caused by gram-positive bacteria, including methicillin-resistant Staphylococcus aureus (MRSA).
While Ehrlich acknowledged that the market being targeted with brilacidin is seemingly crowded, his "confidence is high" that Cellceutix's compound will gain a foothold, and is optimistic that it could actually become the drug of choice in treating ABSSSI should it gain FDA approval. Why? "Because brilacidin is a one-dose therapy and a completely new class of drug," Ehrlich explained. "Being a single-dose therapy already narrows the competition to only a couple peers [The Medicines Company's (MDCO:NASDAQ) Orbactiv and Allergan Inc.'s (AGN:NYSE) Dalvance]. Moreover, brilacidin has a much shorter half-life and can make the claim that it is the first new class of antibiotic to enter a late-stage trial in more than two decades, something no competitor can say."
Recently approved drugs and others in development, Ehrlich explained, are simply reformulations of existing antibiotics, and thus face the problem of bacterial resistance, which is a major issue globally."Bacteria doesn't just start out resistant to an effective drug that it has never seen before and that's what brilacidin brings to the table," the Cellceutix chief executive said. "When you consider these factors, the space certainly doesn’t look as congested."
Brilacidin has been designated a qualified infectious disease product (QIDP) under the GAIN (Generating Antibiotic Incentives Now) Act, signed into law by President Obama in 2012.
Finally, the company announced on March 17 that the last patient visit was concluded in its Phase 2 trial of the oral drug Prurisol for plaque psoriasis. Dosing in the 115-patient trial was completed earlier in the month. Top-line data from the trial is expected in May.
Ehrlich noted that for many, Prurisol, an ester of abacavir, has been the dark horse for Cellceutix, but excitement seems to be brewing as the data release approaches. This is likely due to investors seeing the value in a new psoriasis drug after AbbVie Inc. (ABBV:NYSE) agreed to pay $595 million upfront to Germany’s Boehringer Ingelheim for the experimental psoriasis drug BI655066 earlier this month.
"Cellceutix can make the claim that brilacidin is the first new class of antibiotic to enter a late-stage trial in more than two decades."
Nearly all biotechs developing a psoriasis drug target the moderate-to-severe patient class, but not Cellceutix in its Phase 2 study. Ehrlich explained that the company's confidence in Prurisol led to the decision to evaluate the drug under some of the most difficult situations, and that meant a short-duration study evaluating only one particular lesion in patients being treated with an oral drug (Prurisol). "It doesn't get much more difficult than that," Ehrlich said. "But, by going with a short study, we saved money, challenged our drug, and will have plenty of data for a late-stage trial."
Ehrlich further explained that by taking this approach, Cellceutix is assessing Prurisol's potential for two "huge markets," mild-to-moderate psoriasis and moderate-to-severe psoriasis. "It's a little more savvy approach than one might think at first blush," and one Ehrlich thinks the market may be overlooking.
As all three compounds progress through clinical trials, Ehrlich anticipates a number of "catalytic moments" in Cellceutix's near future. "We have many shots on goal," the CEO said, "and huge opportunities to address unmet medical needs."
Leo Ehrlich is a cofounder of Cellceutix and has served as CEO since November 2010. Ehrlich has more than 25 years of executive leadership experience in building and managing emerging growth companies. Ehrlich is a certified public accountant and received his BBA from Bernard Baruch College of the City University of New York.
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1) Tracy Salcedo compiled this article for Streetwise Reports LLC, publisher of The Gold Report, The Energy Report and The Life Sciences Report, and provides services to Streetwise Reports as an employee. She owns, or her family owns, shares of the company mentioned in this interview: None.
2) Cellceutix Corp. is a sponsor of Streetwise Reports.
3) Leo Ehrlich had final approval of the content and is wholly responsible for the validity of the statements. Opinions expressed are the opinions of Leo Ehrlich and not of Streetwise Reports or its officers.
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