BlueSphere Bio, Inc.

BlueSphere accepted as a virtual member of Johnson & Johnson Innovation – JLABS, a premier global network of health incubatorsPITTSBURGH, Aug. 14, 2024 (GLOBE NEWSWIRE) -- BlueSphere Bio, a drug development company focused on the discovery of novel T-cell receptor (TCR) based therapies, announced it has been accepted as a virtual member of Johnson & Johnson Innovation - JLABS. JLABS is a global network of open innovation ecosystems, enabling and empowering innovators to create and accelerate the delivery of life-enhancing healthcare solutions to patients around the world. “We are very excited to join JLABS,” said Chief Executive Officer Keir Loiacono, Esq. “At BlueSphere, we have a staged development strategy to bring the next generation of TCR-based therapies to the clinic as quickly and efficiently as possible. Being part of JLABS will help us achieve that goal.” BlueSphere uses their proprietary TCXpressTM platform to identify novel TCRs for known and unknown antigens relevant to hematologic malignancies, solid tumors, and rare disease. Using TCXpress, BlueSphere has built a differentiated and stepwise clinical development strategy for TCR-based therapeutics, starting in hematologic malignancies. For their first-in-human study, the company has recently received a letter to proceed from the FDA to treat high-risk AML, ALL, and MDS patients in conjunction with CD34+ stem cell transplant. This TCR T-cell therapy candidate (BSB-1001) targets the minor histocompatibility antigen HA-1. Expanding upon this lead program, the company has three additional TCR candidates ready for product development that can be used to expand HLA population coverage and the addressable market size in this high-risk leukemia space. In addition, BlueSphere recently announced their TCX-102 program, an autologous TCR-T cell therapy that will treat AML patients with mutated NPM-1, a foundational mutation commonly found in this indication. An IND is expected in Q2 of 2025 for the TCX-102 program. Together, these five TCR-T candidates give BlueSphere a uniquely broad portfolio of cell therapies to address significant unmet medical needs in hematologic oncology. Looking beyond these hematologic programs, BlueSphere has recently announced a strategic collaboration with the NCI in recurrent respiratory papillomatosis (RRP), a rare orphan disease. BlueSphere will provide validated HPV-specific TCRs to the experts at NCI, who will then manufacture the TCR-T cell product and administer it under an NCI-sponsored clinical trial. About BlueSphere BlueSphere Bio is the first translational sciences stand-alone company formed by UPMC Enterprises, the innovation, commercialization, and venture capital arm of the Pittsburgh-based health system. BlueSphere Bio was founded upon the proprietary TCR discovery platform – TCXpress™, designed to isolate and functionally characterize TCRs with speed and efficiency. This platform can be used in various clinical settings to discover TCRs for use in either TCR-based cell therapy or in antibody-like molecules, such as bi-specific T cell engagers. While the Company’s initial focus is oncology, the platform could be deployed in other therapeutic areas. BlueSphere is leveraging the breadth and depth of TCXpress™ to develop a portfolio of TCR-based assets with an initial clinical focus on acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), and myeloid dysplastic syndrome (MDS). BSB-1001 is a TCR T cell therapy targeting the minor histocompatibility antigen-1 (HA-1) and is the first clinical candidate in the Company’s TCX-101 clinical program. Under the TCX-101 clinical program, BSB-1001 will be dosed simultaneously with allogeneic hematopoietic stem cell transplant. BlueSphere anticipates enrolling its first patient in the TCX-101 program in 4Q2024. Using TCXpress™, the company has also discovered and nominated three additional miHA targeting TCRs for clinical development, all for use in the same clinical setting as BSB-1001. These additional miHA candidates will become part of the TCX-101 Program as a panel that positions the Company with best-in-class HLA coverage in these hematologic indications. In addition to TCX-101, BlueSphere has further broadened its AML therapy pipeline to address an additional subset of patients by discovering and nominating a lead TCR reactive against mutant NPM-1 for the TCX-102 program. The TCX-102 program will be autologous and not given in combination with stem cell transplant. An IND is expected in the second quarter of 2025. Finally, in addition to a strategic collaboration with the National Cancer Institute to advance a novel TCR T-cell therapy targeting the rare orphan disease, recurrent respiratory papillomatosis (RRP), BlueSphere is identifying a broad panel of TCRs to deploy in its own first solid tumor program, which will be announced later this year. Company ContactKim JaffeSenior Vice President, Head of Business Development & Strategy+1- 609-306-7042kjaffe@bluespherebio.com Media ContactAndrew MielachLifeSci Communications+1-646-876-5868amielach@lifescicomms.com

PITTSBURGH, June 04, 2024 (GLOBE NEWSWIRE) -- BlueSphere Bio, a T-cell receptor (TCR) T-cell therapy company utilizing a powerful TCR discovery platform to identify novel therapeutic candidates for patients with hematologic malignancies and solid tumors, today announced a strategic collaboration with the National Cancer Institute (NCI), part of the National Institutes of Health. The collaboration will seek to advance a novel TCR T-cell therapy targeting recurrent respiratory papillomatosis (RRP), a rare orphan disease. Under the terms of a Cooperative Research and Development Agreement (CRADA), BlueSphere will collaborate with the Center for Cancer Research (CCR) at the NCI. The clinical studies contemplated under the CRADA will be conducted under the leadership of Clint Allen, M.D. and Scott Norberg, D.O. at the CCR, NCI. An earlier Material Transfer Agreement had granted BlueSphere access to HPV+ tumor samples collected by the NCI, with which BlueSphere leveraged its proprietary high-throughput TCR discovery platform, TCXpress™, to identify multiple TCRs with a high affinity for human papilloma virus (HPV) 6 and 11, strains of the virus intricately linked to RRP. "This strategic collaboration with the National Cancer Institute represents a significant milestone for BlueSphere that allows us to expand the potential of the TCRs identified with our TCXpress™ platform beyond our initial oncology programs in leukemia," said Keir Loiacono, Esq., Chief Executive Officer of BlueSphere. "With the combined expertise of Drs. Allen and Norberg and the extensive resources of the NCI, we are well-positioned to explore the vast potential of the TCRs identified with our TCXpress™ platform as an innovative treatment pathway for RRP. This collaboration provides us with a unique opportunity to demonstrate proof-of-concept efficacy of the TCRs identified with our TCXpress™ platform in a novel therapeutic domain. Notably, unlike solid tumors, RRP presents distinctive challenges without the constraints of the tumor microenvironment, presenting a promising avenue for therapeutic intervention." Dr. Norberg added, "This collaboration aligns with our mission of developing novel therapies for patients suffering with RRP. This collaboration seeks to determine whether TCR-T cell therapy has the potential to eliminate the underlying cause of the disease, rather than simply treating symptoms that result from this condition. This novel approach requires a shared commitment to innovation, underscoring the potential transformative impact this collaboration aims to achieve." RRP is a disease most commonly caused by two strains of HPV: HPV 6 and HPV 11, whereby benign papillomas grow in a patient’s air passages leading from the nose and mouth into the lungs1. In certain patients, these papillomas can be debilitating and difficult to treat, particularly when found within the lungs. The focus of the collaboration will be to prepare the identified TCRs for clinical studies and to investigate the efficacy of the resulting clinical-grade TCRs in a future early-stage clinical trial targeting RRP. Per the terms of the CRADA, the contemplated clinical trial may be sponsored by the NCI or BlueSphere. BlueSphere will provide support and transfer the TCR sequences to the NCI, while the NCI will conduct all testing, sequence modifications, process development, manufacturing, and clinical trial activities. About BlueSphere Bio BlueSphere Bio is the first translational sciences stand-alone company formed by UPMC Enterprises, the innovation, commercialization, and venture capital arm of the Pittsburgh-based health system. BlueSphere Bio was founded upon the proprietary TCR discovery platform – TCXpress™, designed to isolate and functionally characterize TCRs with speed and efficiency. This platform can be used in various clinical settings to discover TCRs for use in either TCR-based cell therapy or in antibody-like molecules, such as bi-specific T cell engagers. While the Company’s initial focus is oncology, the platform could be deployed in other therapeutic areas. BlueSphere is leveraging the breadth and depth of TCXpress™ to develop a portfolio of TCR-based assets with an initial clinical focus on acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and myeloid dysplastic syndrome (MDS). Under its TCX-101 program, BlueSphere anticipates enrolling its first patient in 2024 for treatment with its first-in-human candidate, BSB-1001. BSB-1001 is a TCR T cell therapy targeting the minor histocompatibility antigen-1 (HA-1) to be used in conjunction with allogeneic hematopoietic stem cell transplantation (alloSCT). Using TCXpress™, the company has also discovered and nominated three additional miHA targeting TCRs for clinical development, all for use in the same clinical setting as BSB-1001. These additional miHA candidates will become part of the TCX-101 Program as a panel that positions the Company with best-in-class HLA coverage in these hematologic indications. In addition to TCX-101, BlueSphere has further broadened its AML therapy pipeline to address an additional subset of patients by discovering and nominating a lead TCR reactive against mutant NPM-1 for the TCX-102 program. The TCX-102 program will be autologous and not given in combination with stem cell transplant. An IND is expected in the second quarter of 2025. Finally, in addition to a strategic collaboration with the National Cancer Institute to advance a novel TCR T-cell therapy targeting the rare orphan disease, recurrent respiratory papillomatosis (RRP), BlueSphere is identifying a broad panel of TCRs to deploy in its own first solid tumor program, which will be announced later this year. 1 https://www.nidcd.nih.gov/health/recurrent-respiratory-papillomatosis BlueSphere ContactKim JaffeSenior Vice President, Head of Business Development & Strategy+1-609-306-7042kjaffe@bluespherebio.com BlueSphere Media ContactAndrew MielachLifeSci Communications+1-646-876-5868amielach@lifescicomms.com

Prioritizing resources to advance avexitide in hyperinsulinemic hypoglycemia indications Active discussions underway with potential partners to advance late-stage virology programs Reduction in workforce executed to align with focus on avexitide; extends cash runway to Q4 2024 David Apelian, MD, PhD, MBA, appointed CEO Live conference call and webcast at 8:30 am ET today PALO ALTO, Calif., June 29, 2023 /PRNewswire/ -- Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), a commercial-stage biopharmaceutical company focused on the development of innovative therapies for rare diseases, today announced that following an extensive portfolio prioritization review, the Company will focus its development efforts on advancing avexitide in hyperinsulinemic hypoglycemia indications. The Company will continue to commercialize Zokinvy ® (lonafarnib) for the treatment of Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies. In addition, Eiger is evaluating strategic partnering options for its virology assets, lonafarnib and peginterferon lambda. A 25% reduction in workforce, a reduction in out-of-pocket spending related to its hepatitis delta (HDV) development program and its existing term loan are expected to extend the Company's cash runway into the fourth quarter of 2024. The Company also announced that it has appointed David Apelian, MD, PhD, MBA, who has served as interim Chief Executive Officer since December 2022, as the Company's next CEO. "Today's environment necessitates prudent and strategic evaluation of how to advance our promising and diverse pipeline in order to develop potential breakthrough medicines for patients and to drive stockholder value," said David Apelian, MD, PhD, MBA, CEO of Eiger. "After a thorough examination of our programs, we are deploying our resources toward recognizing the compelling potential of avexitide in metabolic diseases. Our initial focus will be on post-bariatric hypoglycemia, or PBH , where we see the highest revenue potential, have demonstrated proof-of-concept in Phase 2 clinical trials, and have FDA alignment on Phase 3 endpoints, sample size, and study design. In the future, we also intend to develop avexitide for congenital hyperinsulinism as a second indication. We plan to provide further guidance next quarter on the progress of our business development efforts for our virology assets, as well as definitive plans for the PBH Phase 3 study initiation and key study milestones." Dr. Apelian continued, "For our other late-stage assets, lonafarnib and peginterferon lambda, we believe the strength of our data and alignment on a regulatory path with FDA make them attractive to potential collaborators. We are actively engaged in discussions with potential partners." "David has embraced his role as CEO, which was a natural transition given his proven tenure with Eiger as a board member and previous Chief Operating Officer," said Thomas Dietz, PhD, non-executive Chairman of the Board. "David's extensive clinical development and regulatory experience gained from working at large pharmaceutical and biotechnology companies, combined with his strategic vision and previous leadership roles at Eiger, make him the right choice to lead Eiger." Current Pipeline Avexitide for Post-Bariatric Hypoglycemia (PBH) A large orphan disease with a growing population; caused by complications in bariatric surgery Prevalence of approximately 180,000 in the US and approximately half that in the EU Avexitide is the only drug in development for PBH with Breakthrough Therapy designation FDA alignment on pivotal Phase 3 study endpoints, sample size, and design Avexitide for Congenital Hyperinsulinism An ultra-rare, life-threatening, pediatric disorder of persistent hypoglycemia that results in irreversible brain damage in up to 50% of children Breakthrough Therapy designation from FDA Rare Pediatric Disease designation Zokinvy® (lonafarnib) for Progeria and Processing-Deficient Progeroid Laminopathies First quarter 2023 net product revenue of $4.1 million; full year 2022 net product revenue of $12.7 million Partnering Opportunities Lonafarnib-Based Regimens for HDV First-in-class, oral prenylation inhibitor Phase 3 D-LIVR study complete; presented at EASL 2023 in Vienna, Austria Week 48 primary endpoint achieved in both lonafarnib arms vs PBO (1.9%) (N=407) Composite response rate in oral arm = 10.1% (p=0.0044) Composite response rate in combination arm = 19.2% (p<0.0001) 24-week post-treatment data demonstrate that both lonafarnib arms showed a statistically significant difference in composite response rate compared to placebo (N=338) Composite response rate in oral arm = 14.2% (p<0.0001) Composite response rate in combination arm = 26.4% (p<0.0001) HDV RNA BLQ response rate in oral arm = 6.1% (p=0.168) HDV RNA BLQ response rate in combination arm = 22.7% (p<0.0001) Pre-NDA meeting with FDA supportive of potential path to approval for oral and combination therapy Peginterferon Lambda First-in-class, well-tolerated interferon Potential to be interferon of choice in antiviral combination therapies Phase 3 LIMT-2 study of peginterferon lambda monotherapy fully enrolled next month Potential for edevelopment in HDV, HBV, respiratory diseases such as COVID and influenza, as well as other virology indications. Conference Call Details The live and replayed webcast of the call will be available through the company's website at . To participate in the live call by phone, dial (866) 524-3160 (U.S.) or (412) 317-6760 (International). The webcast will be archived and available for replay for at least 90 days after the event. About Dr. David Apelian Dr. Apelian has 23 years of clinical development and regulatory experience with large pharmaceutical and biotechnology companies, including nearly six years in senior management, advisory and Board roles at Eiger. He has served as the Company's interim CEO since December 2022. He had previously served as the Company's Chief Operating Officer and Executive Medical Officer starting in 2018, responsible for heading the company's R&D, Medical and Regulatory Affairs operations. Following his departure from that position in 2019, he has served as a Senior Clinical Advisor and board member at Eiger. Dr. Apelian was also the founding CEO of BlueSphere Bio. In addition, Dr. Apelian was Executive Vice President and Chief Medical Officer of Achillion Pharmaceuticals, Inc. where he was responsible for creating portfolio strategy and managing the company's clinical development programs. Earlier in his career, he held senior management positions at GlobeImmune, Bristol-Myers Squibb, and Schering Plough. Dr. Apelian has extensive experience in chronic HCV, chronic HBV, immune oncology and rare diseases. About Post-Bariatric Hypoglycemia Post-bariatric hypoglycemia (PBH) and other forms of hyperinsulinemic hypoglycemia (HH) after gastrointestinal surgeries are characterized by exaggerated secretion of glucagon-like peptide-1 (GLP-1) after meals, dysregulated secretion of insulin, and a rapid drop in blood sugar. Hypoglycemia typically occurs one to three hours after meals and is often accompanied by symptoms of brain glucose starvation (neuroglycopenia), such as blurred vision, confusion, speech difficulty and incoordination. Severe hypoglycemia events can result in serious outcomes such as loss of consciousness, falls, seizures, and motor vehicle accidents, putting patients at risk for death and disability. Recurrent hypoglycemia events can have serious implications on quality of life, including heightened fear of hypoglycemia and reduced functionality, such as ability to work, drive and independently perform activities of daily living. PBH is an orphan disease caused by complications in bariatric surgery with a prevalence of approximately 180,000 in the US and approximately half that in the EU. About Avexitide Avexitide is an investigational, first-in-class glucagon-like peptide-1 receptor (GLP-1r) antagonist in development for the treatment of post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI). Avexitide has been granted Breakthrough Therapy designation for both PBH and HI. Because avexitide binds to the GLP-1r on pancreatic beta cells and prevents GLP-1r signaling, it works upstream of beta cell insulin secretion to reduce dysregulated insulin secretion and the occurrence of hypoglycemia. By addressing underlying disease mechanisms, avexitide may offer a targeted approach to treating hypoglycemia in patients with hyperinsulinemic hypoglycemia, including PBH and HI. Eiger has developed a novel formulation of avexitide for subcutaneous injection. About Eiger Eiger is a commercial-stage biopharmaceutical company focused on the development of innovative therapies for are diseases. Eiger's lead product candidate, avexitide, is a well characterized, first-in-class GLP-1 antagonist for the treatment of post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI). Avexitide is the only drug in development for PBH with Breakthrough Therapy designation from the FDA. For additional information about Eiger and its clinical programs, please visit . Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts, including statements regarding our future financial condition, timing for and outcomes of clinical results, prospective products, preclinical and clinical pipelines, regulatory objectives, business strategy and plans and objectives for future operations, are forward-looking statements. Forward-looking statements are our current statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things, the timing of our ongoing and planned clinical development; our ability to identify, pursue and enter into partnering opportunities for our virology assets; the sufficiency of our cash, cash equivalents and investments to fund our operations into fourth quarter of 2024, including the scope and impact of any savings from our workforce reduction and cash conservation efforts; the revenue potential of avexitide in post-bariatric hypoglycemia and congenital hyperinsulinism; our ability to finance, independently or through collaborations, the continued advancement of our development pipeline; and the potential for success of any of our products or product candidates. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Eiger makes, including additional applicable risks and uncertainties described in the "Risk Factors" section in Eiger's Quarterly Report on Form 10-Q for the quarter ended March 31, 2023 and Eiger's subsequent filings with the SEC. The forward-looking statements contained in this press release are based on information currently available to Eiger and speak only as of the date on which they are made. Eiger does not undertake and specifically disclaims any obligation to update any forward-looking statements, whether as a result of any new information, future events, changed circumstances or otherwise. Investors: Sylvia Wheeler Wheelhouse Life Science Advisors [email protected] Media: Aljanae Reynolds Wheelhouse Life Science Advisors [email protected] SOURCE Eiger BioPharmaceuticals, Inc.