Neurovance, Inc.

Two biotech CEOs shared how they remained afloat amid rocky market waves and why they think their companies are positioned well for the future. After a brutal year of biotechs slashing programs and culling staff, the hottest topic at last week’s J.P. Morgan Healthcare conference was whether the frosty market is likely to thaw in 2023. But while some companies are counting every dollar, Fierce Biotech caught up with two CEOs of well-financed companies to find out how they are riding the rocky market waves and why they think they are well-positioned for the next few years. As of May 2022, the prolonged bear market saw about 150 to 200 public biotechs holding onto less than a year’s worth of cash, according to a report from Hatteras Venture Partners. Of 670 small-cap biotechs, only about 200 had cash runways projected to extend through at least 2024, according to the same report. Two of those companies are Agios Pharmaceuticals and Cerevel Therapeutics.  When asked how it felt being a publicly traded biotech last year, Agios CEO Brian Goff said it felt great, citing the company’s “incredibly strong” balance sheet, which clocked in at about a billion dollars at the end of 2022’s third quarter. The money is expected to allow Agios—which is developing cellular metabolism therapies for rare diseases—to carry through all its late-stage programs, or into 2026, whichever comes first. The CEO noted that a strong bank balance also comes with great responsibility. “It's not like we're just walking around with a lot of cash,” Goff explained. “We take that capital allocation very seriously. But if what you're asking is: Does it feel good to not be at the mercy of raising equity constantly? Yeah, that feels great.”   Goff—who joined Agios in August 2022, succeeding former CEO Jacqualyn Fouse, Ph.D.—said he took time off to look for his next role after serving as Alexion’s chief commercial and global operations officer. The industry veteran—who has also held positions across Johnson & Johnson, Novartis, Shire’s Baxalta, Baxter and Neurovance—eventually decided on Agios. The biotech's rare disease focus drew Goff in, a feature the CEO believes makes the company nimble and able to look at other potential assets that might be off the radar for some competitors. Alongside five pivotal studies, Massachusetts-based Agios was also coming off a February 2022 FDA approval of Pyrukynd for treating hemolytic anemia in adults with pyruvate kinase deficiency—the first approved disease-modifying therapy for adults in the indication—and received European approval in November with Goff at the helm.   “The really great part about our story is the consistency of data we've seen now not only across different stages but across three different anemias,” Goff said. The company is currently testing Pyrukynd, also called mitapivat, in a phase 3 trial for adults with thalassemia and a phase 2/3 study for adults with sickle cell disease, while also assessing the drug’s efficacy in pediatric patients. “It allows us to treat successively larger patient populations,” Goff explained. “But it also adds to the de-risking element in the way people look at us as a company. As we build more data, each of the subsequent indications to come look more and more realized, so to speak.”        But even Agios wasn't immune from the wave of layoffs and pipeline restructurings that battered the industry last year. The biotech let go of 50 employees and shuffled its R&D leadership team in May, right before Goff entered the picture. The changes unlocked between $40 million and $50 million in cost savings. That spring also saw Agios “remove the non-validated portion” of its pipeline, Goff said, adding that the move was made to prioritize assets that “had a meaningful pathway forward.” So what sets Agios apart from its biotech peers who are struggling to stay afloat?  “I think companies that are successful stay dynamic,” Goff said, citing Agios’ historical flexibility. After developing cancer drug Tibsovo, the company sold off its oncology arm to Servier for $1.8 billion in 2021 and pivoted toward rare diseases.  “You have to be strategically bold to make the changes when they're needed,” Goff said, noting that it’s equally as important to make sure “you really take care of the way that people are treated through those transitions.”   Goff also keeps an ear tuned to what the emerging talent recruited at Agios think about the biotech's direction, arguing that “modern companies need to embrace the fact that the lines are blurred between the purpose of the company, the real experience of the individual and their own personal life.” The dynamism required to succeed also means that employees are the first to receive information regarding a company change. “We've spent a lot of time and effort involving our employees and the ‘why’ of where we are, where we're headed and what we need to prepare for,” said Goff. Keeping your employees onboard by actively communicating with them is also important at Pfizer spinout Cerevel. The neuroscience-focused biotech has grown its workforce from a team of 50 in 2019 to just over 300 at the start of this year.   “The company was barely six months old and we commissioned work on what our values would be, what kind of culture we want,” CEO Tony Coles, M.D., told Fierce. Coles believes that the early investment in the company’s ethos has helped guide the biotech’s hiring processes. “I think people look at the fact that we were intentional about the kind of culture we wanted to build, and that's been attractive,” the CEO explained. “While there has been a lot of fluctuation in the job market, I think companies that have a clear brand—either culture or the value proposition of the employment opportunity—will do better and have done better.” The biotech—which is focused on neurological diseases including Parkinson’s, epilepsy and schizophrenia—had a balance sheet with just over a billion dollars at the end of last year’s third quarter, money anticipated to carry the company into 2025. Cerevel went public two years ago and has raised $1.4 billion in total since its launch four years ago.   “We have, in a sense, an embarrassment of riches,” Coles said. “Those riches include largely the breadth of the portfolio. Given that we have a number of things to push forward in development—I think that's been of great interest to a lot of investors.” Coles suggests that the Massachusetts-based biotech’s wide-ranging portfolio offers “the opportunity to be the premier neuroscience company.” He cites the high prevalence of patients with Alzheimer’s disease who also suffer from neuropsychiatric symptoms (97%) and have psychosis associated with the disease (40%). The company believes this provides ideal market conditions for its drug emraclidine. The muscarinic M4 selective positive allosteric modulator is currently being assessed in a phase 2 trial in patients with schizophrenia who experience psychosis. Last month, safety data from a phase 1b study validated that the drug doesn’t raise blood pressure—a concern for patients who may require chronic dosing—and the company is now focused on an efficacy readout from the trial set for the first half of 2024. The biotech is hoping to position the therapy as a new standard of care that does not come with debilitating side effects due to blocking dopamine receptors. That being said, the company faces some stiff competition in the form of Karuna Therapeutics, which is also going after psychosis tied to schizophrenia or Alzheimer’s with its late-stage asset KarXT. The oral, investigational M1/M4-preferring muscarinic agonist is currently being tested in several phase 3 trials, placing Karuna a step closer to crossing the finish line to market than Cerevel if successful.  While opportunity appears abundant for Cerevel, the company still has much to prove. All of the biotech’s assets are currently in development, including several preclinical programs and four other clinical-stage therapeutic candidates beyond emraclidine. Ultimately, both CEOs believe 2023 will be a year full of important milestones further validating their pipelines and companies. “Mainly, 2023 is a big setup for some really great events coming in 2024,” Agios’ Goff said. “I think it's really just nose to the grindstone and just getting the work done,” Cerevel’s Cole explained. “If we do that, and if we're judicious and disciplined in our spending, I think 2023 will be a very successful year.”

– Current CEO, Jackie Fouse, Ph.D., to Transition to Chair of Board of Directors Effective August 8, 2022 – – Agios to Host Investor Webcast Today at 4:30pm ET – CAMBRIDGE, Mass., July 12, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, today announced that effective August 8, 2022, Jackie Fouse, Ph.D., will transition to the role of chair of the board of directors and Brian Goff will assume the role of chief executive officer. Mr. Goff will also serve as a member of Agios’ board of directors. Current chairman, David Schenkein, M.D., will remain on the board as a director. “Throughout our history, Agios has engaged in deliberate and thoughtful long-term planning to drive positive impact for our people and patients. The team’s consistent execution – reflected in the divestiture of our oncology portfolio, the approval of our first genetically defined disease medicine and the initiation of pivotal trials in thalassemia and sickle cell disease – put Agios in a position of strength and great promise for the future,” said Dr. Fouse. “The time is right to evolve our leadership and deepen our genetically defined disease skill set to further drive our ability to develop life-changing treatments. Brian’s strong track record of leading rare disease product launches to drive their impact for patients, building and scaling companies’ rare disease capabilities and leading high-performing, diverse teams with strong cultures make him the right next leader for Agios. I look forward to working with him closely as he transitions into his new role and as I continue my commitment to Agios as chair of the board.” “I am delighted and honored to lead Agios at this incredibly exciting time and to have the opportunity to advance its purpose of bringing meaningful new therapies to people with genetically defined diseases,” said Mr. Goff. “Rare and genetically defined diseases have been my focus and passion for the past decade, and Agios is well positioned to profoundly change patients’ lives across a variety of diseases with the recent launch of PYRUKYND® in adult PK deficiency, a promising clinical and preclinical pipeline based on our expertise in cellular metabolism and our talented and dedicated team. I am grateful to Jackie for serving as our new board chair as we carry forward the momentum and the patient-oriented culture she has strengthened at Agios.” Mr. Goff joins Agios with more than thirty years of experience in the biopharmaceutical industry. Most recently he was executive vice president, chief commercial and global operations officer at Alexion Pharmaceuticals, where he was responsible for leading the global commercial and operations teams prior to the company’s acquisition by AstraZeneca in July 2021. Before joining Alexion, Mr. Goff was chief operating officer and a member of the board of directors of Neurovance. Prior to joining Neurovance, Mr. Goff served as Baxalta’s executive vice president and president of the hematology division. He previously served with Baxter Healthcare Corporation as global hemophilia franchise head. Earlier in his career, Mr. Goff held positions of increasing responsibility in sales and marketing with Novartis Pharmaceuticals and the pharmaceutical division of Johnson & Johnson. Mr. Goff has an MBA from the Wharton School at the University of Pennsylvania and a Bachelor of Arts from Skidmore College. Dr. Fouse joined Agios as a member of the board of directors in 2017 and has served as chief executive officer since 2019. She led the company’s strategic pivot to focus on developing and commercializing innovative treatments for genetically defined diseases. In February 2022, the U.S. Food and Drug Administration (FDA) approved the company’s first genetically defined disease product, PYRUKYND® (mitapivat), for the treatment of hemolytic anemia in adults with PK deficiency, the first disease-modifying therapy for this rare, debilitating, lifelong blood disease. Through Jackie’s orchestration of the sale of the company’s commercial, clinical and research-stage oncology portfolio to Servier, Agios received an upfront payment of $1.8 billion, with $200 million in potential future milestone payments. This transaction strengthened the balance sheet and positioned the company to accelerate and advance its genetically defined disease portfolio. “At every step of Agios’ journey, our board and leadership team give careful consideration to what should come next for the company. With the appointment of Brian, we are putting in place the right next CEO who possesses the experience in rare and genetically defined diseases, including hematology, to drive our strategy forward and deepen our ability to deliver for patients and shareholders,” said Dr. Schenkein, current chairman of the Agios board of directors. “Thanks to Jackie’s leadership, Agios is well positioned for its next chapter. She successfully shifted the company focus to genetically defined diseases, strengthened our balance sheet, achieved our first FDA approval in our hemolytic anemia program, continued to foster a culture of connections and respect and thoughtfully addressed issues of social justice. On behalf of the entire board of directors, I want to thank Jackie for her incredible leadership and commitment to patients; she will undoubtedly continue to provide Agios and Brian with valuable strategic guidance and perspective as she takes on the role of chair of the board of directors.” Conference Call Information Agios will host a virtual investor webcast today at 4:30 p.m. ET with Jackie Fouse and Brian Goff to discuss Agios’ CEO transition. The event will be webcast live and can be accessed under “Events & Presentations” in the Investors and Media section of the company’s website at . The archived webcast will be available on the company's website beginning approximately two hours after the event. About Agios Agios is a biopharmaceutical company that is fueled by connections. The Agios team cultivates strong bonds with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver therapies for genetically defined diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company's leadership in the field of cellular metabolism, Agios is advancing a robust clinical pipeline of investigational medicines with active and planned programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency and MDS-associated anemia. In addition to its clinical pipeline, Agios has multiple investigational therapies in preclinical development and an industry-leading research team with unmatched expertise in cellular metabolism and genetics. For more information, please visit the company’s website at . Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the expected benefits of Agios’ chief executive officer succession plan. The words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. Management’s expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation risks and uncertainties related to: the impact of the COVID-19 pandemic on Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of future approved products, and launching, marketing and selling of current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, including with respect to the regulatory submissions for PYRUKYND® (mitapivat), investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures and competitive factors; Agios’ ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain collaborations; the failure of Agios to receive milestone or royalty payments related to the sale of its oncology business, the uncertainty of the timing of any receipt of any such payments, and the uncertainty of the results and effectiveness of the use of proceeds from the transaction with Servier; and general economic and market conditions. These and other risks are described in greater detail under the caption “Risk Factors” included in Agios’ public filings with the Securities and Exchange Commission, or SEC, including the risks and uncertainties set forth under the heading Risk Factors in our filings with the SEC. While the list of factors presented here is considered representative, this list should not be considered to be a complete statement of all potential risks and uncertainties. Any forward-looking statements contained in this press release are made only as of the date hereof, and we undertake no obligation to update forward-looking statements to reflect developments or information obtained after the date hereof and disclaim any obligation to do so other than as may be required by law. Contacts Investors: Holly Manning, 617-844-6630 Senior Director, Investor Relations Holly.Manning@agios.com Media: Jessica Rennekamp, 857-209-3286 Director, Corporate Communications Jessica.Rennekamp@agios.com A photo accompanying this announcement is available at Brian Goff Brian Goff, Incoming CEO of Agios Pharmaceuticals