On April 29, 2021 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, reported business highlights and financial results for the first quarter ended March 31, 2021.

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"With the recent closing of the sale of our oncology business, we are excited to embrace a focused future in genetically defined diseases," said Jackie Fouse, Ph.D., chief executive officer at Agios. "In the weeks and months ahead, we look forward to a number of important catalysts, beginning with presentations at the EHA (Free EHA Whitepaper) 2021 Virtual Congress, where we will share data from our ACTIVATE and ACTIVATE-T studies of mitapivat in adults with pyruvate kinase (PK) deficiency, as well as data from our Phase 2 study of mitapivat in thalassemia. The PK deficiency data will form the basis of our upcoming submissions for regulatory approval in the U.S. this quarter and in the EU mid-year. Additionally, we look forward to further exploring the impact of mitapivat in thalassemia and sickle cell disease as we launch two Phase 3 studies and a Phase 2/3 study, respectively, later this year. The courage and needs of the patients and families that we seek to serve continue to be our great motivation, and we are proud of our recent educational and patient-focused efforts that aim to make a meaningful impact on rare hemolytic anemia communities."

FIRST QUARTER 2021 & RECENT HIGHLIGHTS
Closed the sale of commercial, clinical and research-stage oncology portfolio to Servier Pharmaceuticals, LLC, on March 31, 2021.

Initiated the repurchase of up to $1.2 billion of outstanding shares, as authorized by the board of directors, and entered into a definitive agreement with Bristol-Myers Squibb Company (BMS) to repurchase 7,121,658 shares of Agios common stock held by BMS and its affiliates for an aggregate purchase price of $344.5 million, or $48.38 per share. Agios expects to execute a meaningful portion of the planned repurchases by year-end through a combination of 10b5-1 plans and open market purchases.

Supported several educational and patient-focused initiatives, including the sponsorship of a health literacy program for sickle cell disease and the launch of a collaboration with 23andMe that led to addition of the very first PK deficiency carrier status report.

KEY UPCOMING MILESTONES

Finalize global regulatory filings for mitapivat in adults with PK deficiency; submit new drug application (NDA) in the U.S. in the second quarter of 2021 and marketing authorization application (MAA) in the EU in mid-2021.
Initiate two Phase 3 studies of mitapivat, ENERGIZE and ENERGIZE-T, in not regularly transfused and regularly transfused adults with thalassemia in the second half of 2021.
Initiate Phase 2/3 study of mitapivat in sickle cell disease by year-end.
Prioritize new indications for pyruvate kinase R (PKR) and pyruvate kinase M2 (PKM2) activator clinical development by year-end.

Data Presentations
Present data from the following clinical studies for presentation at the EHA (Free EHA Whitepaper) Virtual Congress, hosted June 9-17, 2021:
Phase 3 ACTIVATE study of mitapivat in adults with PK deficiency who do not receive regular transfusions
Phase 3 ACTIVATE-T study of mitapivat in adults with PK deficiency who receive regular transfusions
Phase 2 study of mitapivat in adults with α- and β-thalassemia who do not receive regular transfusions
Submit data from ongoing clinical studies of mitapivat in sickle cell disease for presentation at medical meetings in 2021.
Present data from the Phase 1 study of AG-946, the company’s next-generation PKR activator, in healthy volunteers by year-end.

FIRST QUARTER 2021 FINANCIAL RESULTS

Research and Development (R&D) Expenses: R&D expenses for continuing operations were $57.7 million for the first quarter of 2021 compared to $55.4 million for the first quarter of 2020. The year-over-year increase in R&D was driven primarily by start-up costs associated with the Phase 3 studies of mitapivat in thalassemia and sickle cell disease.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses for continuing operations were $33.6 million for the first quarter of 2021 compared to $31.7 million for the first quarter of 2020. The year-over-year increase in SG&A expenses was primarily attributable to one-time workforce expense.

Discontinued Operations: Due to the sale of the oncology business during the first quarter of 2021, we have reclassified the results of the oncology business as discontinued operations, including total revenue of $41.4 million, TIBSOVO net sales of $37 million, and operating expenses of $50.2 million related to discontinued operations.
Net Income: Net income was $1.9 billion for the first quarter of 2021 compared to a net loss of $40.3 million for the first quarter of 2020.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of March 31, 2021 were $2.4 billion compared to $613.1 million as of March 31, 2020. The company expects that its cash, cash equivalents and marketable securities as of March 31, 2021, together with anticipated interest income, future product sales and TIBSOVO royalties, will enable the company to fund its planned operating expenses and capital expenditure requirements 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 first quarter 2021 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 4497151. 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.

On April 29, 2021 Ribon Therapeutics, a clinical stage biotechnology company developing therapeutics targeting stress support pathways, reported an oral presentation on RBN-2397 at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place from June 4 to 8, 2021 (Press release, Ribon Therapeutics, APR 29, 2021, View Source [SID1234578799]). The full meeting program is available at: www.asco.org.

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Abstract Title: A first-in-human phase 1 study of a novel PARP7 inhibitor RBN-2397 in patients with advanced solid tumors
Session Date & Time: Friday, June 4, 2021 at 11:00 AM ET
Session Title: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Abstract ID: 3000

About RBN-2397

RBN-2397, is an orally available small molecule inhibitor of PARP7 that we are developing for the treatment of solid tumors. PARP7 is upregulated in response to cellular stress, including genomic instability in cancers, and acts as a brake on the cellular stress response by negatively regulating the Type I interferon response. By inhibiting PARP7 in tumor cells, RBN-2397 has been shown to directly inhibit cellular proliferation and restore interferon signaling to stimulate an innate and adaptive antitumor immune response. RBN-2397 is currently in a Phase 1 clinical trial as a monotherapy in patients with advanced solid tumors. PARP7 is overexpressed in a number of tumors, including squamous cell carcinoma of the lung, or SCCL, which represents approximately 30% of all non-small cell lung cancers.

On April 29, 2021 Illumina, Inc. (NASDAQ: ILMN) reported that it has filed an action in the General Court of the European Union asking for annulment of the European Commission’s decision asserting jurisdiction to review Illumina’s acquisition of GRAIL (Press release, Illumina, APR 29, 2021, View Source [SID1234578837]). The Commission asserted jurisdiction to review the acquisition under Article 22 of the EU Merger Regulation on April 19, 2021, seven months after the deal was announced.

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"The European Commission’s unprecedented and untimely decision to review this procompetitive acquisition without proper engagement with the parties leaves businesses uncertain as to how the EU Merger Regulation will be applied," said Charles Dadswell, Senior Vice President and General Counsel for Illumina. "The Commission’s actions will stifle innovation, fail patients and increase healthcare costs by needlessly delaying this transaction. The acquisition will allow Illumina to bring GRAIL’s lifesaving testing to more patients, more quickly and at a lower cost."

While the Court process is ongoing, Illumina will continue to work with the European Commission’s Directorate-General for Competition to bring the review to a conclusion as quickly as possible.

On April 29, 2021 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported that Dr. Helen Torley, president and chief executive officer, will present virtually at the BofA Securities 2021 Virtual Health Care Conference on Tuesday, May 11, 2021 at 12:30 p.m. Eastern Time / 9:30 a.m. Pacific Time (Press release, Halozyme, APR 29, 2021, View Source [SID1234578759]).

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An audio-only direct link of the presentation can be accessed through the "Investors" section of www.halozyme.com, and a recording will be made available for 6 months following the event. To access the link, please visit Halozyme’s website approximately 10 minutes prior to the presentation to register and download any necessary audio software.

On April 29, 2021 ChemoCentryx, Inc., (Nasdaq: CCXI), reported financial results for the first quarter ended March 31, 2021 and provided an overview of recent corporate highlights (Press release, ChemoCentryx, APR 29, 2021, View Source [SID1234578781]).

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"Momentum builds with each passing quarter, bringing us closer to our goal of bringing novel, precisely targeted medicine to those that need it most," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "At our R&D Day earlier this month, two world-renowned clinicians outlined the unmet needs in ANCA-associated vasculitis and the data driving their conviction that avacopan could become a landscape-changing therapy. We are well prepared and look forward to providing our views at the FDA Advisory Committee meeting on this topic in just a few days. During our R&D Day we also took the opportunity to establish how our novel small molecule PD-1/PD-L1 inhibitor CCX559 could transcend current limitations in the treatment of cancer. Meanwhile we are progressing toward our next cycle of clinical trials: a Phase III trial of avacopan in patients with severe HS; the initiation of clinical development of avacopan in lupus nephritis, and our first in human studies of the novel orally administered checkpoint inhibitor CCX559 in cancer patients. We look forward to an historic year of 2021 at ChemoCentryx, and we will devote all our energies to making the dream of breakthrough new therapies a reality for patients."

Key First Quarter 2021 Highlights and Recent Developments

In April, the Company held an R&D Day which focused on avacopan in ANCA-associated vasculitis, with a patient describing his journey with the disease, and the Company’s novel checkpoint inhibitor CCX559.

Two leading clinical experts on ANCA-associated vasculitis, Dr. Peter Merkel from the University of Pennsylvania and Dr. David Jayne from the University of Cambridge, explained in detail the widespread effects of this devastating disease and the shortcomings of current therapy with glucocorticoids and other immunosuppressive drugs. Drs. Merkel and Jayne expressed their belief that avacopan could transform the treatment landscape for ANCA-associated vasculitis.

The Company reported new data from the ADVOCATE trial, covering an 8 week period immediately following the 52-week endpoint. During this period from week 52 to week 60, there were 6 relapses out of 158 in the avacopan group and 7 relapses out of 157 in the prednisone group, suggesting a waning of efficacy after drug treatment is stopped. In addition, the estimated Glomerular Filtration Rate (eGFR), which had increased in the avacopan group through week 52, declined in the 8 weeks after patients stopped taking avacopan.

Tausif "Tosh" Butt, who joined the Company in February 2021 as Executive Vice President and Chief Operating Officer, summarized the Company’s preparations and readiness for the

anticipated commercial launch of avacopan shortly after its PDUFA goal date. He reported on primary market research that a survey of 125 rheumatologists and nephrologists indicated that 97% would prescribe avacopan upon FDA approval. The Company plans to focus initially on the estimated eight thousand patients who are newly diagnosed or relapsing with organ or life-threatening disease, out of an estimated eligible patient population for avacopan of twenty thousand patients.

CCX559 was featured in an abstract at the 2021 Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in April. The abstract highlighted laboratory and in vivo data demonstrating potent PD-1/PD-L1 checkpoint inhibition. As a next generation therapy, small molecule inhibitors of PD-L1 may have advantageous properties compared to approved monoclonal antibodies, such as better penetration into solid tumors and reduced immunogenicity. The Company plans to initiate Phase I clinical development of CCX559 in Q2 2021.

In February, The New England Journal of Medicine (NEJM) published the results of the Company’s Phase III ADVOCATE trial of avacopan in ANCA-associated vasculitis, in which avacopan achieved statistical superiority in sustained remission at 52 weeks over prednisone containing standard of care. The same issue of NEJM featured an editorial entitled "Avacopan – Time to Replace Glucocorticoids?".

In February the Japanese New Drug Application for avacopan in the treatment of ANCA-associated vasculitis was accepted for review by the Pharmaceuticals and Medical Devices Agency (PMDA), triggering a milestone payment of $10 million to ChemoCentryx from its partner Vifor Pharma.

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The Company plans to advance avacopan into Phase III development in patients with Hurley Stage III (severe) HS in Q4 2021. In the Phase II AURORA trial, avacopan demonstrated a statistically significant higher response than placebo in Hurley Stage III patients.

The Company plans to initiate clinical development of avacopan in patients with lupus nephritis in Q4 2021.

The Company plans to schedule a meeting with the FDA to discuss evidence of clinical benefit from the ACCOLADE trial of avacopan in the very rare disorder C3 Glomerulopathy (C3G), for which there are no approved therapies. In the trial, avacopan demonstrated statistically significant improvement in renal function as measured by the pre-specified endpoint of eGFR and in the pre-specified secondary endpoint of C3G Histology Index (HI) Disease Chronicity score, compared to placebo over 26 weeks of blinded treatment, but did not achieve a statistically significant improvment in the primary endpoint of the C3G HI Disease Activity Score. Avacopan was safe and well tolerated in C3G patients.

The Company maintained a strong balance sheet, with reported cash, cash equivalents and investments of $424.2 million at March 31, 2021.
First Quarter 2021 Financial Results
Revenue was $10.4 million for the first quarter of 2021, compared to $6.0 million for the same period in 2020. The increase in revenue from 2020 to 2021 was principally attributable to the $10.0 million milestone from Vifor for the February 2021 acceptance of the Japanese NDA for avacopan in the treatment of ANCA vasculitis.
Research and development expenses were $23.4 million for the first quarter of 2021, compared to $19.3 million for the same period in 2020. The increase from 2020 to 2021 was primarily attributable to the manufacture of commercial drug supply in anticipation of the launch of avacopan for the treatment of ANCA vasculitis and costs associated with the advancement of CCX559, the Company’s orally-available small molecule checkpoint (PD-1/PD-L1) inhibitor. These increases were partially offset by lower Phase II related expenses due to the completion of patient enrollment of the avacopan AURORA Phase IIb clinical trial in patients with HS and the discontinuation of further clinical development of CCX140 in FSGS in 2020.
General and administrative expenses were $16.3 million for the first quarter of 2021, compared to $8.8 million for the same period in 2020. The increase from 2020 to 2021 was primarily due to higher employee-related expenses, including those associated with the Company’s commercialization planning efforts, and higher professional fees.

Net loss for the first quarter of 2021 was $29.7 million, compared to net loss of $21.7 million for the same period in 2020.
Total shares outstanding at March 31, 2021 were approximately 69.7 million shares.
Cash, cash equivalents and investments totaled $424.2 million at March 31, 2021. The Company expects to utilize cash, cash equivalents and investments in the range of $145 million to $155 million in 2021.
Conference Call and Webcast
The Company will host a conference call and webcast today, April 29, 2021 at 5:00 p.m. Eastern Time / 2:00 p.m. Pacific Time. To participate by telephone, please dial (877) 303-8028 (Domestic) or (760) 536-5167 (International). The conference ID number is 3963565. A live and archived audio webcast can be accessed through the Investors section of the Company’s website at www.ChemoCentryx.com. The archived webcast will remain available on the Company’s website for fourteen (14) days following the call.