On August 4, 2022 Madrigal Pharmaceuticals, Inc. (NASDAQ:MDGL), a clinical-stage biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis (NASH), reported its second quarter 2022 financial results (Press release, Synta Pharmaceuticals, AUG 4, 2022, View Source [SID1234617455]).

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Paul Friedman, M.D., Chief Executive Officer of Madrigal, stated, "We believe resmetirom, a once-daily oral medication that targets the steatohepatitis and resulting fibrosis in the liver, has the potential to transform the treatment of NASH. As we enter an important period for the company, I’m confident in our strategic plan and operational readiness: we’ve continued to advance our Phase 3 MAESTRO program, expanded our market development activities in the U.S., and improved our financial position with the term loan agreement announced last quarter. Madrigal is well-positioned for the road ahead."

Becky Taub, M.D., Chief Medical Officer and President of Research & Development of Madrigal, stated, "The MAESTRO-NAFLD-1 results we recently presented at EASL reinforce our understanding of the favorable safety and tolerability profile of resmetirom, and we remain on track for topline data from the pivotal MAESTRO-NASH biopsy study in the fourth quarter. These studies are intended to serve as the foundation for a new drug application filing for resmetirom in the noncirrhotic NASH population in the first half of next year."

Remy Sukhija, Chief Commercial Officer of Madrigal, added, "As we continue to lay the foundation for a potential first-to-market launch in the U.S., our engagement with key stakeholders has accelerated in recent months. In June we launched the ‘NASH Explored’ disease education campaign for healthcare providers and announced expanded partnerships with key patient advocacy groups. Additionally, our Market Access and Medical Affairs teams have conducted NASH disease education sessions with payers that together account for more than 80% of all branded prescriptions in the U.S. All stakeholder groups recognize the serious unmet need for approved therapies to treat NASH with liver fibrosis and extensive market research has reinforced our confidence in the commercial potential of resmetirom."

Financial Results for the Six Months Ended June 30, 2022

As of June 30, 2022, Madrigal had cash, cash equivalents and marketable securities of $211.8 million, compared to $270.3 million at December 31, 2021. The decrease in cash and marketable securities resulted primarily from cash used in operations of $107.3 million, partially offset by the capital raised under the Loan Facility ("Loan Facility") with Hercules Capital, Inc. ("Hercules").

Operating expenses were $70.3 million and $127.9 million for the three month and six month periods ended June 30, 2022, compared to $61.7 million and $114.7 million in the comparable prior year periods.

Research and development expenses for the three and six month periods ended June 30, 2022 were $58.5 million and $106.4 million, compared to $51.6 million and $97.4 million in the comparable prior year periods. The increase is attributable primarily to additional activities related to the Phase 3 clinical trials, and an increase in head count.

General and administrative expenses for the three and six month periods ended June 30, 2022 were $11.8 million and $21.4 million, compared to $10.1 million and $17.3 million in the comparable prior year periods. The increase is due primarily to increases in commercial preparation activities, including an increase in headcount and an increase in non-cash stock compensation.

Interest income for the three and six month periods ended June 30, 2022 was $0.3 million and $0.4 million, compared to $0.1 million and $0.3 million in the comparable prior year periods. The increase in interest income was due primarily to a higher average interest rates in 2022.

Interest expense for the three and six month periods ended June 30, 2022 was $0.8 million and $0.8 million, compared to $0 million and $0 million in the comparable prior year periods. The increase in interest expense was as a result of the Loan Facility we entered with Hercules.

About the Resmetirom Phase 3 Registration Program for the Treatment of NASH

Madrigal is currently conducting two Phase 3 clinical trials, MAESTRO-NASH and MAESTRO-NAFLD-1, to demonstrate the safety and efficacy of resmetirom for the treatment of NASH.

MAESTRO-NASH is a multicenter, randomized, double-blind, placebo-controlled Phase 3 study of resmetirom in patients with liver biopsy-confirmed NASH and was initiated in March 2019. The study targeted enrollment of 900 patients with biopsy-proven NASH (fibrosis stage 2 or 3, at least 450 fibrosis stage 3), randomized 1:1:1 to receive once-daily resmetirom 80 mg, resmetirom 100 mg, or placebo. After 52 weeks of treatment, a second biopsy is performed. The dual primary surrogate endpoints on biopsy are NASH resolution with ≥2-point reduction in NAS (NAFLD Activity Score), and with no worsening of fibrosis OR a 1-point decrease in fibrosis with no worsening of NASH. Either primary endpoint can be achieved for a successful trial outcome. A key secondary endpoint is lowering of LDL-C. The planned target enrollment was announced as completed on June 30, 2021.

The first 900 patients in the MAESTRO-NASH study will continue on therapy after the initial 52-week treatment period; up to another 1,100 patients are to be added using the same randomization plan. The study is expected to continue for up to 54 months to accrue and measure hepatic clinical outcome events including progression to cirrhosis on biopsy (52 weeks and 54 months) and hepatic decompensation events.

MAESTRO-NAFLD-1 was initiated in December 2019 and the 52-week multicenter, randomized, placebo-controlled Phase 3 study of resmetirom in over 1,200 patients with NAFLD, presumed NASH, has completed the double-blind arms and an open-label 100 mg arm. An additional open-label active treatment arm in patients with early (well-compensated) NASH cirrhosis is ongoing. The primary endpoint is to evaluate the safety and tolerability of resmetirom. An open-label extension study (MAESTRO-NAFLD-OLE) is ongoing.

Patients in the 52-week phase of MAESTRO-NAFLD-1 were randomized 1:1:1:1 to receive once-daily resmetirom 80 mg, resmetirom 100 mg, placebo in double-blind arms or resmetirom 100 mg in an open-label arm. MAESTRO-NAFLD-1 (unlike MAESTRO-NASH), did not include a liver biopsy and represents a "real-life" NASH study. Patients with 3 metabolic risk factors were documented with NASH or NAFLD by historical liver biopsy or noninvasive techniques. Using noninvasive measures, MAESTRO-NAFLD-1 was designed to provide incremental safety information to support the NASH indication as well as provide additional data regarding clinically relevant key secondary efficacy endpoints to better characterize the potential clinical benefits of resmetirom on cardiovascular- and liver-related endpoints. These key secondary endpoints included LDL-C, apolipoprotein B, and triglyceride lowering; and reduction of liver fat as determined by MRI-PDFF. Additional secondary and exploratory endpoints were assessed including reduction in liver enzymes, FibroScan, and MRE scores and other NASH biomarkers.

Data from the 52-week portion of MAESTRO-NASH, together with data from MAESTRO-NAFLD-1 and other data, including safety parameters, will form the basis for a potential subpart H submission to FDA for accelerated approval of resmetirom for treatment of NASH.

In May 2022, Madrigal announced plans to expand the resmetirom development program by initiating MAESTRO-NASH Outcomes, a randomized double-blind placebo-controlled study in approximately 700 patients with early NASH cirrhosis to allow for noninvasive monitoring of progression to liver decompensation events. A positive outcome is expected to support the full approval of resmetirom for noncirrhotic NASH, potentially accelerating the timeline to full approval. In addition, this study has the potential to broaden the label for resmetirom to include NASH patients with compensated cirrhosis.

On August 4, 2022 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported business highlights and financial results for the quarter ended June 30, 2022 (Press release, Karyopharm, AUG 4, 2022, View Source [SID1234617454]).

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"I’m pleased with our team’s ongoing commitment to successfully execute against key priorities in the second quarter, achieving more than 75% year-over-year revenue growth and further expanding patient access for selinexor globally following full marketing authorization from the European Commission and recent launches in China and Canada," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "Despite headwinds caused by COVID-19 at the beginning of the year and increased competition for later lines of multiple myeloma treatment, we continue to see increased use of XPOVIO in earlier lines with growth in the community setting. Looking ahead to the remainder of the year, we have several key upcoming milestones, including reporting additional data from our studies of selinexor in front-line myelofibrosis and eltanexor in relapsing/refractory myelodysplastic syndromes."

Second Quarter 2022 and Recent Highlights

XPOVIO Commercial Performance

Achieved U.S. net product revenue for the second quarter of 2022 of $29.0 million, a 44% increase compared to the second quarter of 2021, driven by strong year-over-year growth in new patient starts and refills, with continued shift of XPOVIO into second to fourth lines of therapy.

In the second quarter of 2022, a recovery in new patient starts as compared to the previous quarter was offset by the decline in refills due to COVID-19 related reduction of new patient starts in the beginning of the year and intensified late-line competition in the academic setting.

XPOVIO continued its growth in the community setting, driven by a positive shift in perception and intent to treat metrics.

Increased selinexor’s reach to patients around the world with the full marketing authorization of NEXPOVIO (selinexor) in Europe, which expands the indication in multiple myeloma to patients with multiple myeloma after at least one prior therapy as well as commercial launches in mainland China and Canada by partners Antengene Corporation Limited and FORUS Therapeutics Inc., respectively.
Research & Development (R&D) Highlights for Selinexor and Eltanexor

European Commission granted marketing authorization of NEXPOVIO in combination with once weekly bortezomib (Velcade) and low-dose dexamethasone (XVd) for the treatment of adults with multiple myeloma who have received at least one prior therapy, expanding on the prior approval which was in combination with dexamethasone for the treatment of multiple myeloma in adult patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy.

Results from Phase 1/2 study of selinexor in combination with ruxolitinib in treatment-naïve myelofibrosis were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2022 Annual Meeting, including a generally manageable side effect profile with no dose limiting toxicities and 75% of evaluable patients demonstrating ≥35% spleen volume reduction (SVR 35) at week 12. The most commonly reported Grade 3/4 treatment emergent adverse events were thrombocytopenia (27%), anemia (20%) and neutropenia (20%). These data were also presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress.

Following productive discussions with the U.S. Food and Drug Administration (FDA), the Company is finalizing a partner for a companion diagnostic to be used in a registration-enabling study of selinexor in patients with p53 wild-type endometrial cancer.

FDA granted fast track designation for the development program of eltanexor as monotherapy for the treatment of patients with relapsed or refractory intermediate, high-, or very high-risk myelodysplastic syndromes (MDS), per IPSS-R. In addition, the FDA also granted eltanexor orphan drug designation for the treatment of MDS and selinexor for the treatment of myelofibrosis.

The European Commission granted EU orphan medicinal product designation for eltanexor for the treatment of MDS.
2022 Financial Guidance

Based on its current operating plans, Karyopharm is updating its guidance for full year 2022:

Total revenue to be in the range of $155 million to $165 million.

XPOVIO net product revenue to be in the range of $120 million to $130 million versus previous guidance of $135 million to $145 million.

Non-GAAP R&D and Selling, general and administrative (SG&A) expenses, excluding stock-based compensation expense, to be in the range of $250 million to $265 million versus previous guidance of $265 million to $280 million.

Karyopharm has not reconciled the full year 2022 outlook for non-GAAP R&D and SG&A expenses to full year 2022 outlook for GAAP R&D and SG&A expenses because
Karyopharm cannot reliably predict without unreasonable efforts the timing or amount of the factors that substantially contribute to the projection of stock compensation expense, which is excluded from the full year 2022 outlook for non-GAAP R&D and SG&A expenses.

The Company continues to expect that its existing cash, cash equivalents and investments, and the revenue it expects to generate from XPOVIO product sales, as well as revenue generated from its license agreements, will be sufficient to fund its planned operations into early 2024.
Second Quarter 2022 Financial Results

Total Revenues: Total revenue for the second quarter of 2022 was $39.7 million, up 76% compared to $22.6 million for the second quarter of 2021.

Net product revenue: Net product revenue for the second quarter of 2022 was $29.0 million, up 44% compared to $20.2 million for the second quarter of 2021.

License and other revenue: License and other revenue for the second quarter of 2022 was $10.7 million, compared to $2.4 million for the second quarter of 2021. The increase was primarily attributable to $6.5 million earned in reimbursement of development expenses from the Menarini Group.

Cost of sales: Cost of sales for the second quarter of 2022 were $0.9 million, compared to $1.1 million for the second quarter of 2021. Cost of sales reflects the costs of XPOVIO units sold and third-party royalties on net product revenue.

R&D expenses: R&D expenses for the second quarter of 2022 were $44.3 million, compared to $34.0 million for the second quarter of 2021. The increase was primarily driven by an increase in personnel costs and stock-based compensation, including $3.8 million of severance-related stock-based compensation expense incurred during the three months ended June 30, 2022.

SG&A expenses: SG&A expenses for the second quarter of 2022 were $37.3 million, compared to $36.5 million for the second quarter of 2021. The increase in SG&A expenses was primarily due to an increase in stock-based compensation as a result of $3.5 million of severance-related stock-based compensation expense incurred during the three months ended June 30, 2022.

Interest expense: Interest expense for the second quarter of 2022 was $6.3 million, compared to $5.0 million for the second quarter of 2021.

Net loss: Karyopharm reported a net loss of $49.1 million, or $0.62 per share, for the second quarter of 2022, compared to a net loss of $53.6 million, or $0.71 per share, for the second quarter of 2021. Net loss included non-cash stock-based compensation expense of $15.1 million and $8.1 million for the second quarters of 2022 and 2021, respectively.

Cash position: Cash, cash equivalents, restricted cash and investments as of June 30, 2022, totaled $172.6 million, compared to $235.6 million as of December 31, 2021.

Non-GAAP Financial Information

Karyopharm uses a non-GAAP financial measure, non-GAAP R&D and SG&A expenses, to provide operating expense guidance. Non-GAAP R&D and SG&A expenses exclude stock-based compensation expense. Karyopharm believes this non-GAAP financial measure is useful to investors because it provides greater transparency regarding Karyopharm’s operating performance as it excludes non-cash stock compensation expense. This non-GAAP financial measure should not be considered a substitute or an alternative to GAAP R&D and SG&A expenses and should not be considered a measure of Karyopharm’s liquidity. Instead, non-GAAP R&D and SG&A expenses should only be used to supplement an understanding of Karyopharm’s operating results as reported under GAAP.

Conference Call Information

Karyopharm will host a conference call today, August 4, 2022, at 8:00 a.m. Eastern Time, to discuss the second quarter 2022 financial results and provide other business highlights. To access the conference call, please dial (888) 349-0102 (local) or (412) 902-4299 (international) at least 10 minutes prior to the start time and ask to be joined into the Karyopharm Therapeutics call. A live audio webcast of the call, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived webcast will be available on the Company’s website approximately two hours after the event.

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm’s Selective Inhibitor of Nuclear Export (SINE) compounds to be approved for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with Velcade (bortezomib) and dexamethasone (XVd) in patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in patients with heavily pre-treated multiple myeloma; and (iii) in patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including but not limited to the European Union, the United Kingdom, China, South Korea, Canada and Israel, and is marketed in those areas by Karyopharm’s global partners.

Please refer to the local Prescribing Information for full details.

Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including in endometrial cancer and myelofibrosis.

For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at:

Tel: +1 (888) 209-9326
Email: [email protected]

SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.
Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony‐stimulating factors.
Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.
Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.
Serious Infection: Monitor for infection and treat promptly.
Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.
Embryo‐Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.
Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3‐4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.

The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3‐4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions was infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.
Use In Specific Populations
Lactation: Advise not to breastfeed.

On August 4, 2022 Bio-Techne Corporation (NASDAQ: TECH) reported that its Board of Directors has decided to pay a dividend of $0.32 per share for the quarter ended June 30, 2022 (Press release, Bio-Techne, AUG 4, 2022, View Source [SID1234617453]). The quarterly dividend will be payable August 29, 2022, to all common shareholders of record on August 15, 2022. Future cash dividends will be considered by the Board of Directors on a quarterly basis.

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On August 4, 2022 Bio-Teche Corporation (NASDAQ: TECH) reported Chuck Kummeth’s planned two-year transition from his position as Bio-Techne’s Chief Executive Officer (CEO) effective June 30, 2024 (Press release, Bio-Techne, AUG 4, 2022, View Source [SID1234617452]). The Company’s Board of Directors has begun its search for Mr. Kummeth’s replacement, evaluating both internal and external candidates to assume the CEO position upon Kummeth’s retirement at the end of its fiscal 2024. It is anticipated that Mr. Kummeth will continue to serve on Bio-Techne’s Board of Directors upon his retirement.

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Mr. Kummeth has served as Chief Executive Officer and as a member of the Company’s Board of Directors since April 2013. Under Mr. Kummeth’s leadership, the Company delivered on key strategic initiatives to accelerate its organic growth rate, expand its total addressable market and fortify its position as a leader in the life science tools and diagnostics markets. During Kummeth’s tenure as CEO, Bio-Techne grew its revenue from approximately $311 million in fiscal 2013 to $1.1 billion in fiscal 2022, representing a compounded annual growth rate (CAGR) of over 15%. Additionally, the Company grew from approximately 800 to over 3,000 employees, expanded its product portfolio and total addressable market through the completion of 17 acquisitions, established a best-in-class executive leadership team, and implemented processes and procedures to drive future growth.

"During the last nine years, Chuck has done an extraordinary job accelerating growth, building a strong leadership team and positioning Bio-Techne for the future," said Robert Baumgartner, Bio-Techne’s Chairman of the Board of Directors. "Throughout his tenure as CEO, Chuck and the Board have been focused on building management bench strength. Chuck has successfully built an extremely strong leadership team, including a deep bench of experienced and results-driven executives. Additionally, the Company’s enviable position as a rapidly growing, leading life science tools and diagnostics company positions Bio-Techne to attract top-caliber external candidates. With Chuck at the helm for the next two years, we have time for a thorough evaluation of potential candidates and a seamless transition to his eventual successor."

"I am looking forward to continuing to lead the Company and execute our strategic plan over the next two years. As a Board member, I will play an instrumental role in choosing my successor," commented Chuck Kummeth, President and Chief Executive Officer of Bio-Techne. "I am extremely proud of the Company and team we have built over the last nine years. Bio-Techne is in an ideal position to find the leader to drive the business forward starting in fiscal 2025."

On August 4, 2022 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, reported business highlights and financial results for the second quarter ended June 30, 2022 (Press release, Agios Pharmaceuticals, AUG 4, 2022, View Source [SID1234617451]).

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"As we look ahead to the next chapter for the company, Agios is operating from a position of strength. We are executing our commercial launch of PYRUKYND, the first therapy for a rare, debilitating, lifelong blood disorder; we also have five pivotal trials underway, multiple early-stage studies planned or ongoing, a promising preclinical pipeline and a strong balance sheet providing optionality for the future growth of the business," said Jackie Fouse, Ph.D., chief executive officer at Agios. "As I reflect on my legacy as CEO of Agios, I am proud of the bold and strategic decisions we have made to maximize and accelerate our impact for people with genetically defined diseases. I am tremendously grateful to the Agios team for their unwavering resiliency in the face of unprecedented challenges and heartfelt dedication to patients and each other. I look forward to my new role at Agios as board chair and to supporting Brian as he leverages his expertise to expand Agios’ genetically defined disease capabilities, foster the company’s differentiated culture and drive our next phase of impact."

Second Quarter 2022 & Recent Highlights

Continued to execute U.S. launch of PYRUKYND, generating $3.1 million in U.S. net revenue for the second quarter of 2022, the first full quarter following FDA approval.
Initiated Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND in pediatric patients with PK deficiency who are not regularly transfused and who are regularly transfused, respectively.
Announced that effective August 8, 2022, Dr. Fouse will transition to the role of chair of Agios’ board of directors and Brian Goff will assume the role of Agios’ chief executive officer and member of the board of directors.
Evolved Agios’ research approach to focus on advancing the company’s existing validated preclinical programs and in-licensing or acquiring well-characterized, high-potential assets.
Expanded role of Sarah Gheuens, M.D., Ph.D., to chief medical officer and head of research and development, incorporating research and discovery sciences in addition to her existing chief medical officer responsibilities.
Presented clinical and translational data at the 2022 European Hematology Association (EHA) (Free EHA Whitepaper) Congress, including new data supporting the potential benefits of PYRUKYND treatment in adults with PK deficiency.
Key Upcoming Milestones & Priorities

Agios expects to execute on the following key milestones and priorities in 2022:

Adult PK Deficiency: Receive European Medicines Agency (EMA) regulatory decision for PYRUKYND in adults with PK deficiency by year-end.
Thalassemia: Enroll a meaningful portion of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND in not regularly transfused and regularly transfused adults with thalassemia, respectively, by year-end.
Sickle Cell Disease: Complete enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND in sickle cell disease by year-end.
Myelodysplastic Syndrome: Initiate Phase 2a study of AG-946 in adults with low- to intermediate-risk MDS by year-end.
Data Presentations: Continue to publish clinical and translational data supporting the utility of PK activators across key disease areas and elucidating the burden of disease for PK deficiency, thalassemia and sickle cell disease.
Second Quarter 2022 Financial Results

The financial results discussion compares Agios’ continuing operations. All periods have been adjusted to exclude discontinued operations related to the divested oncology business.

Revenue: Net U.S. product revenue from sales of PYRUKYND for the second quarter of 2022 was $3.1 million. This revenue reflects the first full quarter of PYRUKYND launch, following FDA approval on February 17, 2022. In addition, Agios recognized revenue of $2.5 million dollars in the second quarter of 2022 as an up-front payment associated with the licensing of intellectual property for the company’s Friedreich’s Ataxia preclinical program.

Cost of Sales: Cost of sales for the second quarter of 2022 was $0.4 million.

Non-Operating Income: Non-operating income included approximately $2.7 million from TIBSOVO (ivosidenib) royalties for the second quarter of 2022.

Research and Development (R&D) Expenses: R&D expenses were $74.5 million for the second quarter of 2022 compared to $62.0 million for the second quarter of 2021. The year-over-year increase in R&D was driven primarily by increased headcount and workforce-related expenses, planned increased activity associated with the PAH preclinical program, start-up costs for the AG-946 Phase 2a MDS study, increased spend for the AG-946 Phase 1 trial, and start-up costs for the PYRUKYND pivotal studies in sickle cell disease and pediatric PK deficiency.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $28.3 million for the second quarter of 2022 compared to $29.2 million for the second quarter of 2021. The year-over-year decrease in SG&A expenses was primarily attributable to the completion of the reimbursable transition services Agios provided to Servier, which concluded in the first quarter of 2022, related to the sale of the oncology business.

Net Loss from Continuing Operations: Net loss from continuing operations was $91.8 million for the second quarter of 2022 compared to a net loss of $82.8 million for the second quarter of 2021.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2022, were $1.1 billion compared to $1.7 billion as of June 30, 2021. The year-over-year decrease is attributable to operating expenses and 5.7 million shares of common stock that the company repurchased for $273.4 million during the third and fourth quarters of 2021. Agios expects that its cash, cash equivalents and marketable securities will enable the company to execute its operating plan through major catalysts and to cash-flow positivity without the need to raise additional equity.

Conference Call Information
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second quarter 2022 financial results and recent business activities. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.