PROS1

Star Therapeutics has formed two publicly disclosed companies to date—Electra and Vega, both of which are named after stars. Investors have showered Star Therapeutics with $90 million to grow its portfolio companies and develop new antibody drugs for diseases with high unmet need.  Sofinnova Investments led the oversubscribed series C round and was joined by new investors Qatar Investment Authority, Catalio Capital Management, Agent Capital, Soleus Capital and NYBC Ventures alongside all seven existing investors. The new funds nearly double the $100 million Star had already raised since its founding in late 2018.  “Each of our programs goes after multiple diseases with each antibody therapy—so it's a constellation of diseases for every one of our programs,” Star's CEO and founder Adam Rosenthal, Ph.D., told Fierce Biotech.   Star is a biotech portfolio company that has formed two publicly disclosed offshoots to date—Electra Therapeutics and Vega Therapeutics, both of which are named after stars. While Electra is now an independently financed company, blood-disorder-focused Vega remains under Star’s wing.   The newest financing will in part go toward Vega’s VGA039, a monoclonal antibody currently being assessed in a phase 1a/1b study that launched earlier this year. The asset is designed to treat von Willebrand disease (VWD), a bleeding disorder in which blood can’t clot properly. Available treatments, such as factor replacement therapies, are limited and require frequent intravenous infusions.  Vega hopes VGA039 can address these limitations as a subcutaneously self-administered antibody therapy that targets a fundamental mechanism of clot formation via protein S, Rosenthal explained, adding that the company thinks the asset could transform the VWD treatment landscape. “This is very similar actually to what Hemlibra has done in the hemophilia A space,” Rosenthal said, likening the asset’s potential to Roche’s and Chugai’s bispecific monoclonal antibody blockbuster. “Prior to that, really there was only factor replacement therapies, which often are quite burdensome on patients.” The new funds are expected to move VGA039—which received FDA orphan-drug designation this spring—through the entirety of the phase 1a/1b trial, Rosenthal said. “I think we're one of the first and probably the furthest along,” Rosenthal said, referring to antibody therapy developers for VWD. “So, it's an area that probably will get more competitive as people are acknowledging and realizing the unmet need and VWD.” VGA039 also holds potential among other bleeding disorders such as other factor deficiencies and platelet conditions. Star’s series C cash will also be channeled into additional portfolio companies that have yet to be publicly revealed. The business's design allows flexibility when building out teams that have expertise in specific therapeutic areas and to pursue different financing strategies and opportunities for each company, Rosenthal explained.  Star’s constellation of companies boldly go into “areas of white space,” the CEO said—or rare diseases with unmet medical need—a strategy he dubs the company’s “special sauce.”  It also helps explain the emphasis on stars, which Rosenthal said represent the plethora of opportunities available to treat the multitude of diseases that exist today.

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Vega Therapeutics, Inc., a clinical stage biotechnology company developing novel therapies for rare blood disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for VGA039 for the treatment of the rare bleeding disorder, von Willebrand disease (VWD). VGA039 is a first-in-class antibody therapy with a novel mechanism of action that modulates Protein S – a key co-factor involved in thrombin generation during both the initiation and propagation phases of coagulation. By promoting thrombin generation through targeting Protein S, VGA039 addresses a fundamental mechanism of clot formation in VWD and, as a subcutaneously self-administered antibody therapy, has potential to transform VWD treatment. “Receiving orphan drug designation status is an important step for VGA039,” said Gary Patou, MD, Chief Medical Officer of Vega Therapeutics. “VGA039 has the potential to be a new treatment for VWD with a pro reduce the treatment burden for people living with this disease.” An ongoing Phase 1 clinical study (NCT05776069) is designed to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic pro VGA039 in healthy volunteers and VWD patients across sites in the EU and US. In preclinical studies, VGA039 has demonstrated efficacy in VWD, as well as in numerous congenital bleeding disorders. About von Willebrand disease Von Willebrand disease (VWD) is a bleeding disorder in which the blood does not clot properly. In VWD, defective or low amounts of von Willebrand factor (VWF) lead to insufficient platelet adhesion and unstable clot formation. VWD causes severe bleeding that can damage organs and lead to significant impact on patients’ daily lives. Current treatments are limited and include factor replacement therapies that require frequent intravenous (IV) infusions. About Vega Therapeutics Vega Therapeutics is a clinical stage biotechnology company developing novel, first-in-class therapies for rare blood disorders with overlooked patient needs, starting with von Willebrand disease (VWD). The company’s lead product candidate, VGA039, is the first purpose-built antibody therapy for VWD with a novel mechanism of action targeting Protein S. By attenuating Protein S cofactor function for tissue factor pathway inhibitor α (TFPIα) and activated protein C (aPC), VGA039 augments and restores thrombin generation during both the initiation and propagation phases of coagulation, addressing fundamental deficiencies of clot formation in VWD. VGA039 has potential to be a universal hemostatic therapy that can treat various bleeding disorders. For more information, please visit ‑therapeutics.com and follow us on LinkedIn.

Phase 1 clinical trial advances with first subject dosed in the E.U. and IND accepted in the U.S. SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Vega Therapeutics, Inc., a clinical stage biotechnology company developing novel therapies for rare blood disorders, today announced that its clinical trial program for VGA039 has been initiated. The first subject has been dosed in the Phase 1 study of VGA039 (NCT05776069), a multinational clinical trial being conducted following approvals of the Investigational New Drug Application (IND) by the U.S. Food and Drug Administration (FDA) and Clinical Trial Application (CTA) in Europe. The clinical trial program will begin with enrollment of healthy volunteers and expand to include patients with von Willebrand disease (VWD). VGA039 is a first-in-class antibody therapy with a novel mechanism of action that modulates Protein S – a key co-factor involved in thrombin generation during both initiation and propagation of coagulation – with preclinical data demonstrating efficacy in numerous congenital bleeding disorders, including VWD. By promoting thrombin generation through targeting Protein S, VGA039 addresses a fundamental mechanism of clot formation in VWD and, as a subcutaneously self-administered antibody therapy, has potential to transform VWD treatment. VGA039 also has potential to treat additional bleeding disorders, including other factor deficiencies and platelet disorders. “We are excited to advance VGA039 into the clinic as the first purpose-built antibody therapy for VWD, representing a new approach with the potential to reduce the treatment burden for these patients,” said Gary Patou, MD, Chief Medical Officer of Vega Therapeutics. “It is our goal at Vega to enable better therapies for patients with VWD, and we are excited to have taken the first step by dosing our first subject.” “I appreciate the dedication of the Vega team in pursuing a novel mechanism of action focusing on VWD. VWD is a bleeding disorder with high treatment burden and unmet medical need for many patients that impacts their health and daily lives,” said Ayesha Zia, MD, Director of Hemostasis Thrombosis Program, University of Texas Southwestern Medical Center. The single ascending dose portion of the Phase 1 clinical study is designed to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic pro VGA039 in healthy volunteers and VWD patients. Vega plans to enroll the Phase 1 clinical trial for VGA039 at clinical trial sites across the U.S. and other countries. About von Willebrand disease Von Willebrand disease (VWD) is a bleeding disorder in which the blood does not clot properly. In VWD, defective or low amounts of von Willebrand factor (VWF) lead to insufficient platelet adhesion and unstable clot formation. VWD causes severe bleeding that can damage organs and lead to significant impact on patients’ daily lives. Current treatments are limited and include factor replacement therapies that require frequent intravenous (IV) infusions. About Vega Therapeutics Vega Therapeutics is a clinical stage biotechnology company developing novel, first-in-class therapies for rare blood disorders with overlooked patient needs, starting with VWD. The company’s lead product candidate, VGA039, is the first purpose-built antibody therapy for VWD with a novel mechanism of action targeting Protein S. By attenuating Protein S co-factor function for tissue factor pathway inhibitor α (TFPIα) and activated protein C (aPC), VGA039 augments and restores thrombin generation during both initiation and propagation of coagulation, addressing a fundamental mechanism of clot formation in VWD. VGA039 has potential to be a universal hemostatic therapy that can treat various bleeding disorders. For more information, please visit ‑therapeutics.com and follow us on LinkedIn.