Sichuan Biokin Pharmaceutical Co., Ltd.

DOVER, Del., April 4, 2024 /PRNewswire/ -- Eilean Therapeutics LLC, a biopharmaceutical company dedicated to discovering and developing best-in-class and first-in-class small molecule inhibitors to target escape mutations in hematologic and solid malignancies, is pleased to announce the clearance from the Human Research Ethics Committee in Australia for lomonitinib (ZE46-0134) allowing the company to proceed with a Phase I clinical trial in relapsed/refractory (R/R) AML. Lomonitinib is a highly potent and selective plan-FLT3/IRAK4 inhibitor that targets clinically relevant FLT3 mutations and putative escape pathways. "This is a major milestone for the Eilean team, as we continue to progress an accelerated development of lomonitinib," stated Iain Dukes, Chief Executive Officer of Eilean Therapeutics. "Given the safety profile, extended half-life and target engagement demonstrated in a healthy volunteer study, we look forward to initiating our clinical trial and testing our approach in R/R AML patients." About Lomonitinib Lomonitinib is a highly potent and selective inhibitor of FLT3 ITD, TKD and other clinically relevant FLT3 mutations, as well as IRAK4. FLT3 mutations are the most frequently identified mutations in AML. There are two main mechanisms of resistance to FLT3 inhibitors: the FLT3-ITD-F691L mutation deemed the "gatekeeper" mutation that confers resistance to all currently approved FLT3 inhibitors and the activation of the IRAK4 escape pathway. Lomonitinib inhibits both resistance mechanisms. About the Phase 1 Clinical Trial The Phase I study will evaluate the safety, tolerability, pharmacokinetics, and anti-tumor activity of lomonitinib as monotherapy in R/R AML patients with FLT3 mutations (ITD, TKD or gatekeeper). The study will include a dose escalation and an expansion phase with up to 60 total participants. Eilean Therapeutics is planning to initiate the study in Q2 2024 across multiple investigative sites in Australia. About Eilean Therapeutics Eilean Therapeutics LLC is a biopharmaceutical company co-founded by OrbiMed, Torrey Pines Investment and Dr John C. Byrd, focused on the discovery and development of best-in-class and first-in-class small molecule inhibitors that target escape mutations in hematologic and solid cancers. The company is utilizing a proprietary hybrid AI (Expert Systems Inc.), leveraging its key partners proprietary data, chem/bio tools, knowledge and expertise to choose highly valuable molecular mechanism of pathology, to rationally design, accelerate discovery and optimize development of best-in-class and first-in-class therapies. Eilean Therapeutics' goal is to utilize its capabilities and platform to become a leader in developing novel breakthrough medicines to maximize the clinical benefit when treating hematologic and solid malignancies. For more information visit Media Contact: Amy Burd CSO [email protected] SOURCE Eilean Therapeutics

- Funding secured from CF Foundation and current investors for SPL84 Phase 2 study - - Two additional early-stage candidates expected to commence Phase 1-2a studies over the next 12 months – JERUSALEM, April 3, 2024 /PRNewswire/ -- SpliSense, a clinical-stage biotechnology company focused on the development of transformative RNA-based therapies for pulmonary diseases including cystic fibrosis (CF), muco-obstructive diseases like COPD, asthma, non-CF bronchiectasis (NCFB) and idiopathic pulmonary fibrosis (IPF), today announced that the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for the initiation of a Phase 2 study for SPL84 in CF. SPL84 is the Company's lead antisense oligonucleotide (ASO) product for the treatment of people with CF carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The IND Clearance for the Phase 2 study initiation was granted following SPL84's favorable safety profile, supported by Phase 1 and preclinical toxicological and pharmacological data demonstrating the potential for full restoration of CFTR protein and activity, based on clinically predictive CF models. The Company has also announced today that it has secured funding from the CF Foundation and other existing investors to support the SPL84 Phase 2 study and additional pulmonary programs. This represents the CF Foundation's third investment in SpliSense's SPL84 program. "We are excited to initiate the Phase 2 study for SPL84 in CF with the support of the CF Foundation and other existing investors," said Gili Hart, PhD, Chief Executive Officer of SpliSense. " Although currently available CF therapies have managed to significantly increase life expectancy over the past few decades, there is still a significant unmet medical need for disease modifying treatments. Our lead product, SPL84, has been shown to fully restore CFTR activity in the CF gold standard pharmacological model. With the recent FDA IND clearance, we hope to be able to deliver, a life-changing treatment for people with CF carrying the 3849+10 Kb C->T mutation and expand our unique technology to additional CF mutations where there is a significant unmet need." "In addition, SPL84 serves as proof-of-concept for our platform for the treatment of additional pulmonary diseases, including muco-obstructive diseases and IPF, which are expected to enter the clinic over the next 12 months," added Dr. Hart. About Cystic Fibrosis (CF) CF is a genetic multisystem disorder that originates from various mutations in the CFTR gene, which is responsible for the production of the CFTR protein, a chloride channel expressed in the lungs as well as in other tissues. The past decade has seen a dramatic change in CF care with the approval of new CFTR modulators. However, approved CFTR modulators do not support all people with CF and do not offer a cure for the disease. Thus, new strategies of therapeutic development are essential to address partially responsive and non-responsive people with CF, specifically those carrying the 3849+10 Kb C->T splicing mutation. About SPL84 SpliSense utilizes short, precisely targeted proprietary RNA stretches called Antisense Oligonucleotides (ASOs) to modulate specific mRNA sequences or correct various mutations in the target mRNA. SPL84 binds specifically to the mutated CFTR RNA in the targeted sequence, leading to the modulation of the mutated region in the mRNA, potentially allowing the cell to produce fully functional CFTR proteins. SPL84 is administered directly and preferentially to the lungs via inhalation where it is designed to be taken up by the lung cells and to drive the production of corrected CFTR mRNA and eventually fully functional CFTR proteins. About SPL5AC (Muco-Obstructive Diseases) and SPL5B (IPF) SpliSense is advancing two preclinical ASO assets for the treatment of additional pulmonary diseases, including muco-obstructive diseases and IPF. Patients with muco-obstructive lung disease are susceptible to severe mortality and morbidity, creating an unmet need for the development of an effective and potent agent in the treatment of mucin induced lung disease. SPL5AC is an ASO that specifically lowers the level of MUC5AC expression and production in mucus, decreases mucus viscosity, eliminates accumulation in the airways, and reduces infection and inflammation as demonstrated in three proof of-concept disease animal models. Chronic overexpression of MUC5B is suggested to be the single greatest risk factor for the development of IPF. SPL5B is an ASO that lowers MUC5B expression and production avoiding accumulation of MUC5B mucin and plugs in the distil regions of the lungs. By lowering MUC5B levels and plugs formation, SpliSense aims to slow or delay IPF progression and improve patient's clinical condition and quality of life. About SpliSense SpliSense is a biotechnology company focused on the development of RNA-based treatments for pulmonary diseases. The Company's pioneering platform harnesses ASOs for treatment of unmet cystic fibrosis mutations and large pulmonary diseases including muco-obstructive diseases and IPF. Through its novel ASO pulmonary approach, SpliSense aims to address specific genetic mutations or deficiencies, by restoring or reducing protein function, thus targeting the root cause of the disease. The Company's investors include Cystic Fibrosis Foundation, OrbiMed, Israel Biotech Fund, Integra Holdings and Biotel Limited. For additional information, please visit our website at: Company contact: Tsipi Haitovsky Global Media Liaison +972-52-5989-892 [email protected] SOURCE SpliSense

Driven to make targeted medicines even more precise, Alterome Therapeutics pocketed a $132-million Series B to move a pair of oncogenic driver-targeting small molecules into the clinic within the next year.Led by Goldman Sachs Alternatives, Wednesday’s financing also saw participation from Canaan Partners, Driehaus Capital Management, Invus, Digitalis Ventures, Blue Owl Capital, as well as existing investors Orbimed, Nextech Invest, Vida Ventures, Boxer Capital, and Colt Ventures. The round adds to the $99 million Alterome raised in 2022 between a Series A and extension.The biotech takes its name from the biological alterome, which encompasses all the disease-driving alterations in genes – and largely remain undrugged. According to the company, while several medicines targeting oncogenic mutations have been approved, they can have off-target effects that limit their therapeutic window, diminishing their benefit for patients.Alterome is deploying its Kraken platform to design mutation- and isoform-selective small molecules that preferentially target cancer cells and avoid healthy cells. The goal of the structure-guided, machine learning approach is to improve the inhibitory power and safety profile of precision medicines.The Kraken provides atomic-level insights into molecular interactions to predict ligand and binding activity so Alterome’s team of medicinal chemists can discover selective, alteration-specific compounds. “The precision oncology field has arrived at a special moment with the exciting evolution of both drug discovery and precision medicine,” CEO Eric Murphy said. “A unique integration of structure-guided drug discovery with deep translational biology is facilitating the development of novel therapies that address previously inaccessible proteins.”Precision pipelineAlterome’s lead programme targets AKT1 E17K, a clinically validated oncogene that drives about 2% of all tumours, including breast, endometrial and prostate cancers. The oral small molecule, dubbed ALTA-2618, was designed to be a mutant-selective, covalent allosteric inhibitor of AKT1 E17K.The biotech’s second candidate, ALTA-3263, is directed against KRAS, a well-known regulator of cellular signalling that’s mutated in more than 20% of all cancers. However, according to Alterome, the first-generation of KRAS inhibitors had a narrow scope of mutational coverage leading to limited durability and poor tolerability. In contrast, Alterome’s compound is isoform-selective and targets 90% of KRAS mutations, including the most common, G12V and G12D.