SEATTLE, Sept. 29, 2020 /PRNewswire/ — CTI BioPharma Corp. (Nasdaq: CTIC) at present introduced that following a latest Pre-NDA assembly with the U.S. Meals and Drug Administration (“FDA” or “the Company”), the Firm has reached an settlement to submit an NDA for the potential accelerated approval of pacritinib as a remedy for myelofibrosis sufferers with extreme thrombocytopenia (platelet rely lower than 50 x 109/L). The NDA might be primarily based on the obtainable knowledge from the Firm’s accomplished Section Three PERSIST-1 and PERSIST-2 trials and the Section 2 PAC203 dose-ranging trial. The FDA has agreed to a rolling NDA submission which is predicted to begin inside a couple of weeks, with completion of the NDA submission anticipated within the first quarter of 2021. The continuing Section 3 PACIFICA trial is predicted to be accomplished as a post-marketing dedication.
“Because the completion of the PAC203 Section 2 dose-ranging trial, we have now been working collaboratively with the FDA to establish an expeditious approval pathway for pacritinib in myelofibrosis sufferers with extreme thrombocytopenia, a affected person inhabitants with an essential unmet medical want as a consequence of decreased survival and restricted therapeutic choices. Throughout a latest Pre-NDA assembly, we recognized a knowledge bundle from the PERSIST-1, PERSIST-2 and PAC203 Section 2 trials that may function the premise for an accelerated approval software. Particularly, we mentioned threat mitigation measures to handle the FDA’s prior considerations relating to security,” mentioned Adam R. Craig, M.D., Ph.D., President and Chief Government Officer of CTI Biopharma. “In myelofibrosis sufferers, extreme thrombocytopenia happens because of illness or drug-related toxicity from present therapies. There is no such thing as a accepted drug that particularly addresses the unmet want of the myelofibrosis sufferers who’ve extreme thrombocytopenia. Pacritinib has demonstrated scientific profit in treating these sufferers in a number of trials and now has the potential to change into a brand new remedy choice for treatment-naïve and second-line myelofibrosis sufferers in 2021.”
About Myelofibrosis and Extreme Thrombocytopenia
Myelofibrosis is a kind of bone marrow most cancers that ends in formation of fibrous scar tissue and may result in extreme anemia, weak spot, fatigue and an enlarged spleen and liver. Sufferers with extreme thrombocytopenia are estimated to make up greater than one-third of sufferers handled for myelofibrosis, or roughly 17,000 individuals. Extreme thrombocytopenia, outlined as blood platelet counts of lower than 50,000 per microliter, has been proven to lead to total survival charges of simply 15 months. Thrombocytopenia in sufferers with myelofibrosis is related to the underlying illness however has additionally been proven to correlate with remedy with ruxolitinib, which may result in dose reductions, and because of this, might doubtlessly scale back scientific profit. Survival in sufferers who’ve discontinued ruxolitinib remedy is additional compromised, with a mean total survival of seven to 14 months. Myelofibrosis sufferers with extreme thrombocytopenia have restricted remedy choices, creating a major space of unmet medical want.
Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. The JAK household of enzymes is a central part in sign transduction pathways, that are important to regular blood cell progress and growth, in addition to inflammatory cytokine expression and immune responses. Mutations in these kinases have been proven to be immediately associated to the event of a wide range of blood-related cancers, together with myeloproliferative neoplasms, leukemia and lymphoma. Along with myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in situations akin to acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), power myelomonocytic leukemia (CMML), and power lymphocytic leukemia (CLL), as a consequence of its inhibition of c-fms, IRAK1, JAK2 and FLT3.
In March 2008, pacritinib acquired orphan drug designation for the remedy of major myelofibrosis (MF), post-polycythemia vera MF, and post-essential thrombocythemia MF.
In August 2014, pacritinib was granted Quick Monitor designation by the FDA for the remedy of intermediate and excessive threat myelofibrosis, together with, however not restricted to, sufferers with disease-related thrombocytopenia (low platelet counts); sufferers experiencing treatment-emergent thrombocytopenia on different JAK2 inhibitor remedy; or sufferers who’re illiberal of or whose signs usually are not properly managed (sub-optimally managed) on different JAK2 remedy.
About CTI BioPharma Corp.
We’re a biopharmaceutical firm targeted on the acquisition, growth and commercialization of novel focused therapies for blood-related cancers that supply a novel profit to sufferers and their healthcare suppliers. We focus our efforts on therapies that concentrate on blood-related cancers the place there may be an unmet medical want. Particularly, we’re targeted on evaluating pacritinib, our sole product candidate at present in lively growth, for the remedy of grownup sufferers with myelofibrosis. As well as, we have now just lately began growing pacritinib to be used in hospitalized sufferers with extreme COVID-19, in response to the COVID-19 pandemic. We’re headquartered in Seattle, Washington.
Statements included on this press launch that aren’t historic in nature are forward-looking statements inside the that means of Part 27A of the Securities Act of 1933 and Part 21E of the Securities Alternate Act of 1934 and the Personal Securities Litigation Reform Act of 1995. These forward-looking statements are primarily based on present assumptions that contain dangers, uncertainties and different elements that will trigger the precise outcomes, occasions or developments to be materially totally different from these expressed or implied by such forward-looking statements. These dangers and uncertainties embody, however usually are not restricted to: our potential to conduct and full scientific trials in our at present anticipated timeframes; our potential to efficiently exhibit the protection and efficacy of pacritinib; our expectations relating to the completion and end result of our PACIFICA Section Three trial and our PRE-VENT Section Three trial; the danger that the FDA might decide that the profit/threat profile of pacritinib on the dose chosen for the PACIFICA Section Three trial doesn’t help approval; the danger that the FDA might decide that the profit/threat profile of pacritinib within the PRE-VENT Section Three trial doesn’t help approval or requires further scientific knowledge for approval; the danger that pacritinib might fail in its growth by our PACIFICA and PRE-VENT trial; our potential to submit a rolling NDA for pacritinib within the timeline at present anticipated; our potential to obtain regulatory approval for pacritinib pursuant to the accelerated approval pathway or in any respect; the danger that pacritinib could also be delayed to some extent the place it isn’t commercially viable; and people dangers extra totally mentioned within the part entitled “Threat Elements” in our Annual Report on Kind 10-Ok for the yr ended December 31, 2019 and subsequent quarterly reviews on Kind 10-Q. These forward-looking statements communicate solely as of the date hereof and we assume no obligation to replace these forward-looking statements, and readers are cautioned to not place undue reliance on such forward-looking statements. “CTI BioPharma” and the CTI BioPharma brand are registered logos or logos of CTI BioPharma Corp. in varied jurisdictions. All different logos belong to their respective proprietor.
SOURCE CTI BioPharma Corp.
Posted: September 2020