Appili Therapeutics Acquires Clinical-Stage Program From FUJIFILM Toyama Chemical

Appili Therapeutics Inc. recently announced it has signed an agreement with FUJIFILM Toyama Chemical Co., Ltd. to acquire and develop the novel antifungal drug T-2307, which will now be called ATI-2307. This drug candidate is a novel broad-spectrum antifungal agent that has been evaluated in multiple preclinical studies and three human Phase I clinical trials. Today’s agreement with FUJIFILM Toyama Chemical assigns Appili exclusive worldwide rights (ex-Japan) to develop and commercialize this antifungal candidate. With the execution of the agreement, FUJIFILM Toyama Chemical is eligible to receive from Appili future regulatory and commercial milestones payments, as well as a royalty on future net sales.

“Appili was founded with the purpose of identifying, acquiring, and rapidly advancing the most promising anti-infective technologies,” said Appili CEO Kevin Sullivan. “The acquisition of this highly differentiated, clinical-stage program added immediate incremental value to the Company and is an exciting step forward as we continue to build our portfolio and deliver on our commitment of growing shareholder value and meeting patient needs by addressing urgent and underserved public health threats.”

ATI-2307 is a novel broad-spectrum antifungal with a highly differentiated mechanism of action that is not currently susceptible to existing resistance mechanisms. FUJIFILM Toyama Chemical researchers developed the compound internally. It exhibits broad-spectrum antifungal activity both in vitro and in vivo against multiple high priority and clinically important fungi, including Cryptococcusand the multi-drug resistant Candida species. FUJIFILM Toyama Chemical advanced ATI-2307 into clinical development and completed three Phase I clinical studies that demonstrated the drug candidate to be well tolerated in humans at anticipated therapeutic dose levels.

“FUJIFILM Toyama Chemical is excited to announce this agreement with Appili Therapeutics,” said Junji Okada, President and Chief Operating Officer of FUJIFILM Toyama Chemical. “Appili has demonstrated deep expertise in accelerating anti-infective drug development that is well aligned with FUJIFILM Toyama Chemical’s focus on innovative R&D in the area of infectious disease. FUJIFILM Toyama Chemical is committed to solving public health issues through innovation, high-value drug development, and accretive partnership. We are confident that Appili is the right partner to help maximize the potential of ATI-2307 and address the urgent threat of invasive and difficult-to-treat fungal infections.”

ATI-2307 has demonstrated activity against multiple difficult-to-treat fungi, including Cryptococcus and Candida species. Appili will initially focus on developing ATI-2307 for the treatment of cryptococcal meningitis. Cryptococcal meningitis is an opportunistic fungal infection that is a major cause of mortality worldwide. The largest burden of the disease is found in immunocompromised patients in sub-Saharan Africa, South, and Southeast Asia, and is a growing problem in the North America and Europe with increasing numbers of solid organ transplant and cancer therapy treatments. In-hospital acute mortality from cryptococcal meningitis continues to remain high despite current therapies. The current standard of care for cryptococcal meningitis, which is amphotericin B in combination with flucytosine, is associated with significant toxicity, including the potential for kidney failure.

“For many patients, standard-of-care antifungal agents are toxic and outcomes are poor,” said Dr. Armand Balboni, Appili’s Chief Development Officer. “Rising rates of antifungal resistance and the emergence of intrinsically resistant pathogens threaten to further erode an already inadequate antifungal armamentarium. Additionally, population aging and growing utilization of immunosuppressive therapies increasingly put patients at increased risk of fungal infections that are difficult to treat and cause severe strain on hospital resources and increase patient morbidity and mortality.

“Cryptococcal meningitis is a devastating disease with great unmet medical need. We think that ATI-2307 has great potential to provide a more effective and safer therapeutic option for patients with these invasive fungal infections,” continued Dr. Balboni.

In addition to cryptococcal meningitis, Appili is exploring development options for the treatment of refractory, resistant, or otherwise difficult to treat Candida infections. Despite the widespread availability of front-line azole and candin class antifungals, important segments of patients are infected with highly resistant or multi-drug resistant Candida species in which outcomes are poor.vi

Appili Therapeutics Inc. was founded to advance the global fight against infectious disease by matching clearly defined patient needs with drug development programs that provide solutions to existing challenges patients, doctors, and society face in this challenging disease space. Appili has built a pipeline of assets designed to address a broad range of significant unmet medical needs in the infectious disease landscape. This diverse pipeline aims to address some of the most urgent threats in global public health. Via an in-licensing program, Appili acquired the rights to ATI-1701, a vaccine for tularemia, that it is developing to mitigate the risks of a very serious biological weapons threat. ATI-1503 is a drug discovery program aimed at generating negamycin analogue candidates, which are a novel class of antibiotics with broad-spectrum activity against Gram-negative superbugs. ATI-1501 employs Appili’s proprietary, taste-masked, oral-suspension technology with metronidazole for the growing number of patients with difficulty swallowing. Headquartered in Halifax, Nova Scotia, with offices in Mississauga, Ontario, Appili is pursuing worldwide opportunities in collaboration with science and industry commercial partners, governments and government agencies. For more information, visit www.AppiliTherapeutics.com.

FUJIFILM Toyama Chemical was launched by merging FUJIFILM RI Pharma Co., Ltd., a company that conducts research, development, manufacture, and sales of radiopharmaceuticals, and TOYAMA CHEMICAL CO., LTD., a company that conducts research, development, manufacture, and sales of small molecule pharmaceutical products. FUJIFILM Toyama Chemical dedicates efforts to developing innovative diagnostic and therapeutic radiopharmaceuticals and therapeutic drugs with unique action mechanisms in the fields of “oncology,” “central nervous system diseases,” and “infectious diseases” where significant unmet medical needs still exist, with close collaboration with FUJIFILM Corporation, which focuses on research of new medicines. It will also advance the development of new medicines utilizing drug delivery system (DDS) technologies that deliver the required amount of a drug in a timely manner to a specific body area. Also, by exploring synergy with in vitro diagnostic (IVD) devices and reagents owned by Fujifilm group companies, the company will expand its offering of comprehensive solutions from “diagnosis” to “treatment.”