GlobeImmune, Inc.

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.

BlueSphere CEO Dr. David Apelian, M.D., Ph.D/Courtesy BlueSphere Bio One of the greatest recent innovations in cancer treatment, CAR T therapies have sent some patients into long-lasting remission, with speedier recoveries, due to the lack of aggressive chemotherapy involved. They are the darling of numerous biopharma companies, including Sorrento Therapeutics, Kite Pharma (Gilead), and Allogene Therapeutics. But they still have their limitations. They can only target a single tumor-associated antigen, recognize only cell surface antigens, and are not wholly tumor-specific, only targeting proteins expressed by normal and malignant cells. Enter BlueSphere Bio, a small biotech aiming to overcome these challenges with a precision approach to T cell therapy. Headquartered in Pittsburgh, Pa., and founded in 2018, BlueSphere has been operating under the radar. Not anymore. BioSpace sat down with Chief Executive Officer Dr. David Apelian, M.D., Ph.D., who shared why he believes the company’s TCXpress is the “premier T cell receptor platform” that can have a significant impact on cancer. When Apelian spoke with Co-Founder and Chief Scientific Officer Dr. Mark Shlomchik, M.D., Ph.D., about technology he was developing that could rapidly screen and capture thousands of T cell receptors from the sample more efficiently and cost-effectively than traditional methods, he was intrigued. “If you could identify those receptors that will direct the T cells to kill a tumor, amplify those cells in greater number outside the patient in a test tube, so to speak, and then reintroduce a healthy version of that T cell into the patient, you’ve essentially transcended what vaccines are hoping to do,” said Apelian, who was previously chief medical officer at GlobeImmune, one of the early leaders in cancer vaccines. “You’re getting right to the endpoint that you want.” The overall approach also consists of a downstream program that utilizes BlueSphere’s NEOXpress platform, which will enable the individualization of treatments for specific cell tumors. BlueSphere profiles tumor cells by DNA and RNA sequencing and identifies the neoantigen pro those tumors. “In parallel, we’re extracting the T cells from that same tumor, we’re identifying the repertoire of TCRs, and then we’re marrying the two datasets up. It’s a multiplex approach,” Apelian said. Next, they will identify the ones that “light up,” characterize those T cell receptors and their ability to kill the tumor, and specifically recognize the target antigen but not the native antigen. BlueSphere will put the platform to its first test late in 2022 with the planned launch of a clinical trial in bone marrow recipients with high-risk leukemia. Apelian shared that acute myeloid leukemia (ALL) is likely to be the lead indication in that program. Here, they will be targeting minor histocompatibility antigens. When a high-risk AML patient has not responded to first-line therapy or failed a first bone marrow transplant, Apelian said the chance of a successful transplant is probably 10%—if that. While essentially a match, there are still many minor variations between a donor and recipient. This program aims to raise those odds. “We can engineer a T cell into the donor cell, which the recipient is already getting anyway; we can eliminate all the other minor antigen variants, and then shift that to a leukemia-only response,” he explained. This enables complete engraftment, knocking out any residual leukemia cells along with the patient’s marrow and ultimately leading to improved long-term survival. This is an example of an allogeneic, off-the-shelf target. On the other end of the spectrum, BlueSphere’s technology enables it to take a precise approach to each patient’s tumor. Apelian explained that certain cancers called “hot tumors” possess a high number of different antigens. Melanoma probably has the most complex neoantigen pro these tumors are caused by exposure to the sun, which allows more mutations to occur. Non-small-cell lung cancers also have a fair number of neoantigens. “We can literally take a patient’s tumor and extract the tumor-specific antigens that patient has generated uniquely in their own tumor, and then identify the receptors to target the patient’s very own tumors,” Apelian said. The benefit of this individualized therapy, he continued, is that “the patient’s body is already making those T cell receptors, so it’s very unlikely we’re going to make a receptor that’s going to be toxic to the patient.” Then, BlueSphere can enrich these receptors, grow them in large quantities in a re-engineered healthy version of the T cell, and replace them in the patient’s body. “Those T cells are sick; they’re exhausted. So, we’re taking that receptor part and re-engineering it into a healthy version of their own T cell, and then reintroducing it into the patient,” Apelian said. “That’s going to be the game-changer for solid tumors.” Conversely, there are other cancers known as cold tumors. These include ovarian cancer, prostate cancer, pancreatic cancer, glioblastomas, and most breast cancers. Apelian believes his company’s platform has the throughput capability to tackle these as well. “We think we have enough throughput with our TCXpress to even identify TCRs for those neoantigens in so-called cold tumors, but that’s kind of a downstream project for us,” he said. In the middle of this range, BlueSphere will look at other exciting targets for solid tumors that can be re-engineered ahead of time to treat a myriad of different cancers. While some elements of the work will be off-the-shelf, and there is a movement in the industry toward finding a universal cell, Apelian explained many advantages to the autologous approach. “I understand the motivation for that [quest for a universal cell], but I think you’re going to lose a lot of the specificity and the elegance—the exquisite specificity of T cell,” he said. “We think it’s going to be more likely that autologous approaches and individualized therapies will be safer and more effective.” BlueSphere is conducting what it calls the “virtual patient program” to determine the next steps in its pipeline. Instead of waiting to complete manufacturing aspects necessary for individualized tumor therapies, the company is building a tissue repository to test various tumors. Here, they will identify the neoantigen profile, extract the T cell repertoire from the tumor, and characterize the potential to recognize and kill the tumor. “We’re collecting tissues, local tissues and some tissues from the National Cancer Institute, and we’re testing as if they were patients in a study,” Apelian explained. He also expressed an interest in working collaboratively to solve cancers outside of BlueSphere’s purview and other diseases. “I could see this [technology] being useful a huge array of diseases that we can treat by appropriately re-engineering T cells to have the right T cell receptors,” he said. With obvious ambition and belief in its platform, BlueSphere expects to double its workforce, which sits at 38 people, over the next 18 to 24 months. “It’s a super exciting time for the company,” Apelian said, “It feels like moonshot startup excitement, which is fun. Everyone kind of appreciates how game-changing this platform could be.”

NEW YORK, June 04, 2021 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced that current Chief Business Officer Jeffrey Rona has expanded his role to include Chief Financial Officer responsibilities. “Ovid is moving forward with a very focused corporate strategy. Combining the business development and financial functions and strategy is another step we have taken to simplify and empower the company to move nimbly and effectively,” said Jeremy M. Levin, DPhil, MB, BChir, Chairman and Chief Executive Officer of Ovid Therapeutics. “Jeff is a terrific professional and has accomplished much at Ovid. I welcome him into this expanded role and look forward to working with him more closely.” “Ovid’s plans for its future are exciting. I look forward to collaborating with the Executive team and Board to drive the Company’s business and financial growth strategy,” commented Mr. Rona. “Leading the corporate and business development efforts, financial reporting and planning, and building relationships with the investment community, are critical to advancing Ovid’s mission. I welcome the opportunity to take on these roles.” Jeffrey Rona has more than 25 years of experience as an investment banker, Chief Business Officer, and Chief Financial Officer in the life sciences. Prior to joining Ovid Therapeutics, he was the Western region Managing Director for Danforth Advisors, a life science financial strategy consultancy. Previously, he held the titles of Chief Business Officer at GlobeImmune, Chief Financial Officer at AlgoRx Pharmaceuticals, Chief Financial Officer at Great Basin Scientific, and Director, Investment Banking at UBS. He received his Bachelor of Science degree in accounting from Case Western Reserve University. About Ovid Therapeutics Ovid Therapeutics Inc. is a New York-based biopharmaceutical company using its BoldMedicine® approach to develop medicines that transform the lives of patients with rare neurological disorders. We believe these disorders represent an attractive area for drug development as the understanding of the underlying biology has grown meaningfully over the last few years and today represents a substantial opportunity medically and commercially. Based on recent scientific advances in genetics and the biological pathways of the brain, we aim to identify, discover and acquire novel compounds for the treatment of rare neurological disorders. We have built a deep knowledge of such disorders, how to treat them and how to develop the clinically meaningful endpoints required for development of a compound in these disorders. We continue to execute on our strategy to build this pipeline by discovering, in-licensing and collaborating with leading biopharmaceutical companies and academic institutions. Ovid’s emerging pipeline programs include OV329, a small molecule GABA aminotransferase inhibitor for seizures associated with Tuberous Sclerosis Complex and Infantile Spasms; OV882, a short hairpin RNA therapy approach for Angelman syndrome; OV815, a genetic therapy approach for KIF1A associated neurological disorder; and other non-disclosed research targets. Additionally, Ovid maintains a significant financial interest in a program directed to the exploitation of soticlestat, for which Takeda is responsible for the global development and commercialization, if soticlestat is successfully developed and commercialized. Two phase 3 trials for soticlestat in Dravet syndrome and Lennox-Gastaut syndrome are expected to begin in mid-2021. For more information on Ovid, please visit . Forward-Looking Statements This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding the development of Ovid’s pipeline and the clinical and regulatory development and commercialization of soticlestat. You can identify forward-looking statements because they contain words such as “will,” “appears,” “believes” and “expects.” Forward-looking statements are based on Ovid’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include, without limitation, uncertainties in the development and regulatory approval processes, and the fact that initial data from pre-clinical and clinical trials may not be indicative, and are not guarantees, of the final results of the trials and are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and/or more patient data become available. Additional risks that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovid’s filings with the Securities and Exchange Commission under the caption “Risk Factors.” Such risks may be amplified by the COVID-19 pandemic and its potential impact on Ovid’s business and the global economy. Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. Contacts Investors and Media:Ovid Therapeutics Inc.Investor Relations & Public Relationsirpr@ovidrx.com OR Investors:Argot PartnersDawn Schottlandt212-600-1902ovid@argotpartners.com Media:Dan Budwick1ABdan@1abmedia.com