Paroxysmal Nonkinesigenic Dyskinesia

MediMabBio announce a new memorandum of understanding (MOU) with PDCline Pharma. This collaboration is aimed to explore the synergy between our MMB101/MFA011 and PDC*line Pharma’s plasmacytoid dendritic cell (PDC) cancer vaccines to revolutionize cancer treatment. Together, our promising drug candidates, targeting TIGIT and other immuno-oncology pathways, would enhance the efficacy of PDC cancer vaccines. By leveraging PDC*line Pharma’s expertise, we aim to boost the efficacy of our therapies and explore innovative treatment strategies. Link to Korean news article

Unlike antibody-drug conjugates (ADCs) that focus on antigens, PDCs are able to target the tumor microenvironment that has universal features across solid tumors. Despite all of the advances made in treating different cancers over the past several years, there is still significant room for improvement in oncology. That’s the thinking of the leadership team at Connecticut-based Cybrexa Therapeutics who touted the potential of their novel class of tumor-targeting peptide drug conjugate (PDC) therapeutics at the annual J.P. Morgan Health Care Conference. Cybrexa Chief Executive Officer Per Hellsund told BioSpace that the company’s alphalex technology platform is designed to boost the target opportunity within cancer treatment programs. Hellsund described the company’s approach as a way to “improve the therapeutic index.” Unlike antibody-drug conjugates (ADCs) that focus on antigens, PDCs are able to target the tumor microenvironment that has universal features across solid tumors. “It provides opportunities for broad targets,” Hellsund said. “We plan to disrupt the ADC market by expanding the reach of the molecules being targeted. We can take payloads that have been proven by ADCs and expand them into new patient populations.” The company’s lead asset is CBX-12 (alphalex-exatecan), which is in Phase I development as a potential treatment for solid tumors. CBX-12 is an alphalex conjugate that includes the highly potent topoisomerase I inhibitor exatecan. The inhibitor is in the same class as payloads used by two well-known and approved ADCs, AstraZeneca ’s Enhertu and Gilead Sciences ’ Trodelvy. Hellsund explained that unlike the ADCs, CBX-12 is able to target cancer cells independently of the overexpression of antigens that the ADCs target. The company believes CBX-12 has potential synergy with PD-1 inhibitors and other immuno-oncology drugs. In October, the company released interim data for the study that showed “robust efficacy signals and tolerability” in patients who have been diagnosed with metastatic cancer. So far, the data seen from the early study is serving as a key demonstration of the company’s alphalex technology, an antigen-independent PDC tumor-targeting platform, Hellsund said. In data released so far, one patient with ovarian cancer demonstrated a complete response to CBX-12. There has also been a partial response in one patient, and another heavily pretreated patient has achieved stable disease. “There’s a real belief that this technology can help patients,” Hellsund said. “The clinical benefits we’ve seen, including complete response and low incidence of serious adverse effects related to CBX-12, reinforce our belief that CBX-12 could dramatically change the lives of patients who have few or no therapeutic options.” Cybrexa has plans to investigate CBX-12 in different solid tumors, including breast, ovarian and non-small cell lung cancer. The company is on track to accelerate the development of CBX-12 and is eying multiple Phase II studies that are expected to be initiated in the second half of 2022. “So far the data looks very promising,” Hellsund said of CBX12. “We’re very happy with what we’re seeing with proof of mechanism to date. We’re really excited about this platform and the potential to help patients.” The Phase I study is expected to be completed later this year. Data garnered from the study will ultimately guide the future plans at Cybrexa, Hellsund noted. He added that the data generated in the study will also go a long way to validate the targeting mechanism of action of the PDC platform. “If we can get the drug into the tumor and spare the tissue, we know it will work,” he said. Unlike several large trials over the past two years, Hellsund said their clinical program has not been negatively impacted by COVID-19. He admits the company has been fortunate that its program has not been delayed and said he hopes that good fortune will continue as the company begins to position its two other assets, CBX15 and CBX13, for the clinic. Coinciding with the advancement of Cybrexa’s platform is a need for additional fundraising. Chief Financial Officer Stephen Basso said the $25 million raised last year in a Series B financing round provided the company with enough funding to support operations through the end of this year. Basso said they anticipate securing additional funding that will support the launch of expansion cohorts for CBX-12 and begin clinical trials for the company’s other top candidates. Basso said they are looking at a crossover financing round of $75 million that will finance the company for another two years. He hinted that this will allow the company to broaden its investor based and eventually position it for a possible initial public offering. The J.P. Morgan conference typically brings companies face-to-face with multiple investors. While that mode is usually preferable for both investor meetings and potential partnering opportunities, Basso and Hellsund said they are being proactive with their time setting up the necessary meetings to share the Cybrexa story. “Our objectives are to meet new investors and we are always looking to maintain relationships with bankers and strategic partners,” Hellsund said. He added that the company is excited to share information about its technology and the possible disruption of the ADC market the company envisions.

- No Unexpected Safety Findings During the Course of the Study - - On Track to File an IND in the First Quarter 2021 and Commence Phase I Trial by Mid Year 2021 - NEW HAVEN, Conn., Jan. 28, 2021 (GLOBE NEWSWIRE) -- Cybrexa Therapeutics, an oncology-focused biotechnology company developing a new class of therapeutics through its alphalex™ Peptide Drug Conjugate (PDC) tumor targeting platform, today announced that it has completed the GLP toxicology study of its lead candidate, CBX-12. The positive results for this study serve as a guide for dosing regimen for the planned Phase I trial of CBX-12 as Cybrexa intends to file the IND during the first quarter 2021. “The successful completion of the GLP toxicology studies is an early, yet significant milestone in the development of our lead candidate CBX-12, a highly potent alphalex PDC conjugate that we believe has the potential to impact several types of solid tumors, both as a monotherapy and in combination with PD-1 inhibitors and other immuno-oncology drugs,” said Per Hellsund, President and CEO of Cybrexa. “Additionally, the study results are relevant to the development of our other pipeline candidates, as they suggest the utility of our alphalex PDC tumor targeting platform for targeted intracellular drug delivery, independent of surface antigens.” In the GLP toxicology studies CBX-12 was delivered via IV infusion, and based on multiple parameters, including mortality, clinical observations, body weights and food consumption, the results established a significant therapeutic index of ~20X over exatecan for CBX-12. Additionally, in accordance with the FDA feedback from the pre-IND meeting, Cybrexa completed a rodent respiratory safety pharmacology study with no notable clinical observations, as well as a hemolytic potential and plasma compatibility study with no hemolysis or plasma incompatibility observed. CBX-12 is a novel treatment for solid tumors that includes a highly potent topoisomerase I inhibitor payload that is in the same class as the payloads used by antibody-drug conjugates (ADCs) ENHERTU® and TRODELVY™. In contrast to these ADCs, CBX-12 is able to target cancer cells independent of antigen overexpression, which should greatly expand the addressable patient populations. About the alphalex™ Technology Platform The Cybrexa alphalex™ technology platform – which consists of a pHLIP® peptide, linker, and small molecule anti-cancer agent (payload) – enables antigen-independent targeting of tumors and intracellular delivery of highly potent anticancer therapies, creating therapeutics that can revolutionize the standard of care. pHLIP® peptides are a family of pH-Low Insertion Peptides that target acidic cell surfaces. pHLIP® was developed at Yale University and the University of Rhode Island, and is exclusively licensed to pHLIP, Inc. alphalex™ represents the disruptive next generation in tumor targeting. View a video of the mechanism of action of the technology at . About Cybrexa Cybrexa is a privately-held biotechnology company dedicated to developing next-generation tumor-targeted cancer therapies using its alphalex™ platform. The Company’s lead candidate, CBX-12, an alphalex™-exatecan conjugate, is expected to enter Phase I/II in 2021 in advanced solid tumors. Cybrexa also has other preclinical toxin conjugate programs as well as synthetic lethality programs. Cybrexa was founded by physician-scientists and has an experienced management team that has built numerous successful life sciences companies. For more information about Cybrexa, please visit . Contacts Cybrexa TherapeuticsLisa RehmEmail: lisa.rehm@cybrexa.com Investor RelationsStephanie CarringtonTel: 646-277-1282Email: Stephanie.Carrington@westwicke.com Media RelationsMark CorbaeTel: 203-682-8288Email: Mark.Corbae@westwicke.com