On May 4, 2023 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported business highlights and financial results for the first quarter ended March 31, 2023 (Press release, Agios Pharmaceuticals, MAY 4, 2023, View Source [SID1234630996]).

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"In the first quarter of the year, Agios made significant progress executing across our industry-leading pipeline of PK activators, with clinical studies spanning thalassemia, sickle cell disease, lower-risk MDS and pediatric PK deficiency," said Brian Goff, chief executive officer at Agios. "We closed screening of the Phase 3 studies of PYRUKYND in thalassemia with enrollment expected to be complete later this month, and look forward to the data readout of the Phase 2 portion of the RISE UP study of PYRUKYND in sickle cell disease in the middle of this year."

First Quarter 2023 & Recent Highlights

PYRUKYND U.S. Launch: Generated $5.6 million in U.S. net revenue for the first quarter of 2023, the fourth full quarter following FDA approval. A total of 127 unique patients have completed prescription enrollment forms, representing an increase of 21 percent over the fourth quarter of 2022. A total of 89 patients are on PYRUKYND therapy, representing a 14 percent increase over the fourth quarter of 2022.
Thalassemia: Closed screening of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND in not regularly transfused and regularly transfused adults with thalassemia, respectively.
Leadership: Appointed Jeffrey Capello to the board of directors. Paul Clancy will step down from the board of directors at the end of his term, effective June 13, 2023.
Environmental, Social, and Governance (ESG): Published 2023 ESG Report, which provides corporate sustainability disclosures for the period January 1, 2022 to December 31, 2022.
Other: Servier’s Phase 3 trial of vorasidenib in patients with residual or recurrent IDH mutant low-grade glioma met both its primary endpoint and key secondary endpoints. As part of the divestiture of Agios’ oncology business to Servier, Agios retains rights to a potential $200 million milestone upon FDA approval of vorasidenib and 15% royalties on potential U.S. net sales.
Key Upcoming Milestones & Priorities

Agios expects to execute on the following additional key milestones and priorities by the end of 2023:

Thalassemia: Complete enrollment of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND in not regularly transfused and regularly transfused adults with thalassemia, respectively, by mid-year.
Sickle Cell Disease: Announce data readout from the Phase 2 portion of the RISE UP study of PYRUKYND and go/no-go to Phase 3 decision by mid-year.
Pediatric PK Deficiency: Enroll more than half of patients in the Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND by year-end.
Lower-risk Myelodysplastic Syndromes (LR-MDS): Complete enrollment of the Phase 2a study of novel PK activator AG-946 by year-end.
Pipeline: File investigational new drug (IND) application for phenylalanine hydroxylase (PAH) stabilizer for the treatment of phenylketonuria (PKU) by year-end.
First Quarter 2023 Financial Results

Revenue: Net U.S. product revenue from sales of PYRUKYND for the first quarter of 2023 was $5.6 million. This revenue reflects the fourth full quarter of PYRUKYND launch, following FDA approval on February 17, 2022.

Cost of Sales: Cost of sales for the first quarter of 2023 was $0.6 million.

Research and Development (R&D) Expenses: R&D expenses were $67.3 million for the first quarter of 2023 compared to $70.1 million for the first quarter of 2022. The year-over-year decrease was primarily driven by the $1.5 million of reimbursable transition-related expenses provided to Servier in the first quarter of 2022 related to the sale of the oncology business.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $28.4 million for the first quarter of 2023 compared to $31.5 million for the first quarter of 2022. The year-over-year decrease was primarily attributable to a reduction in workforce-related expenses.

Net Loss: Net loss was $81.0 million for the first quarter of 2023 compared to $94.8 million for the first quarter of 2022.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of March 31, 2023, were $1.0 billion compared to $1.1 billion as of December 31, 2022. Agios expects that its cash, cash equivalents and marketable securities together with anticipated product revenue and interest income will enable the company to execute its operating plan, including funding the currently planned development programs for mitapivat, AG-946 and PAH stabilization and commercializing mitapivat outside of the U.S. through one or more partnerships.

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 2023 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.

On May 4, 2023 Adagene Inc. ("Adagene") (Nasdaq: ADAG), a platform-driven, clinical-stage biotechnology company transforming the discovery and development of novel antibody-based therapies reported achievement of a milestone in its ongoing collaboration with Exelixis for development of novel masked antibody-drug conjugate (ADC) candidates leveraging Adagene’s proprietary SAFEbody precision masking technology (Press release, Adagene, MAY 4, 2023, View Source [SID1234630995]).

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Under the terms of a collaboration and licensing agreement established in 2021, the milestone triggers a $3 million payment to Adagene for successful nomination of the lead SAFEbody candidates for the second of its collaboration programs.

"This milestone reflects our focus on delivering high quality antibody candidates to our technology licensing partners, further validating our platform and world-class antibody engineering expertise," said Peter Luo, Ph.D., Co-founder, Chief Executive Officer, and Chairman of Adagene. "Our collaboration with Exelixis also reflects a strong commitment at Adagene to bringing in non-dilutive funding by leveraging our SAFEbody precision masking and dynamic antibody technologies."

SAFEbody technology is designed to overcome safety and tolerability challenges associated with many antibody therapeutics by using precision masking technology to shield the binding domain of the biologic therapy. This allows for improved tumor-specific targeting of antibodies, while minimizing on-target off-tumor toxicity in healthy tissues, a longstanding challenge with many antibody therapeutics.

Under the terms of the agreement, Adagene received an upfront payment of $11.0 million and Exelixis can nominate two targets for development of SAFEbody candidates during the collaboration. Adagene is eligible for development and commercialization milestones, as well as royalties on net sales of products developed around each of these targets.

In January 2022, Adagene received a $3.0 million milestone payment from Exelixis for the successful nomination of lead SAFEbody candidates for one of the collaboration programs, and an additional $1.1 million upfront payment in June 2022.

Adagene has a network of global technology licensing agreements, including a $2.5 billion collaboration with Sanofi announced in March 2022. In addition to ongoing technology licensing collaborations, Adagene applies its SAFEbody technology to develop candidates for its wholly-owned pipeline of transformative antibody-based therapeutics. The company also has a clinical collaboration with Roche, who is sponsoring and conducting a randomized phase 1b/2 to evaluate the anti-CTLA-4 SAFEbody ADG126 in combination with atezolizumab and bevacizumab in first-line treatment of advanced hepatocellular carcinoma.

On May 3, 2023 Jounce Therapeutics, Inc. (NASDAQ: JNCE) ("Jounce" or the "Company"), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported that Concentra Biosciences, LLC ("Concentra"), through its wholly owned subsidiary Concentra Merger Sub, Inc. ("Concentra Merger Sub"), has successfully completed the previously announced tender offer to acquire all outstanding shares of the common stock of Jounce for $1.85 per share in cash plus a non-tradeable contingent value right (a "CVR") per share (Press release, Jounce Therapeutics, MAY 4, 2023, View Source [SID1234630927]). The $1.85 per share upfront consideration represents a premium of approximately 75% to Jounce’s closing share price immediately prior to the public disclosure of Concentra’s acquisition proposal on March 14, 2023.

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The tender offer expired at 6:00 p.m., Eastern Time, on May 3, 2023. As of the expiration of the offer, 36,367,727 shares of Jounce common stock had been validly tendered and not validly withdrawn from the tender offer, representing approximately 69.09% of the outstanding shares of Jounce common stock. All conditions of the offer were satisfied or waived, and Concentra completed its acquisition of all outstanding shares of Jounce that were validly tendered and not validly withdrawn for the offer price.

Following the closing of the tender offer, Concentra Merger Sub merged with and into Jounce and all shares of Jounce common stock that had not been validly tendered were converted into the right to receive the same $1.85 per share in cash plus one CVR per share (the "Merger"). As a result of the Merger, Jounce became a wholly owned subsidiary of Concentra. Shares of Jounce common stock ceased trading on Nasdaq and Concentra intends promptly to cause such shares to be delisted. Payment will be made promptly to all former Jounce common stockholders regardless of whether they tendered their shares.

On May 3, 2023 iOnctura, a clinical-stage biotech developing selective cancer therapies against targets that play critical roles in multiple tumor survival pathways, reported it has exercised an exclusive option with Clavius Pharmaceuticals, adding the novel oral TGF-β pathway inhibitor, IOA-359, to its pipeline (Poster, iOnctura, MAY 3, 2023, View Source [SID1234640233]). The Company also announces it has, in collaboration with the University of Twente (UT), been awarded a grant from Health Holland and KWF (Dutch Cancer Society), reflecting the potential of IOA-359.

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Under the terms of the licence agreement, iOnctura is solely responsible for the global development and commercialisation of the small molecule inhibitor. The TGF-β pathway plays a critical role in promoting tumor aggressiveness, immune escape and resistance to therapy, making it an attractive target for cancer therapy. Previous attempts to interrupt TGF-β pathway signaling in cancer have been thwarted by drug-associated toxicities and activation of resistance pathways by the tumor. By developing a safe TGF-β pathway inhibitor and characterising the resistance mechanisms that typically arise when targeting the TGF-β pathway alone, iOnctura’s data-driven precision oncology methods are being used to design novel, safe combination treatments that promise to override tumor survival resistance pathways.

Supplementing iOnctura’s internal preclinical investigations, the KWF grant led by Dr Ruchi Bansal, Assistant Professor of Medical Cell BioPhysics, will utilise UT’s unique model system to provide iOnctura with a valuable preclinical pharmacology package for IOA-359.

Catherine Pickering, Chief Executive Officer of iOnctura, said: "IOA-359 is an exciting addition to our preclinical pipeline. TGF-β is an established target in oncology yet we are the first company applying precision methods to intelligently combine targeting this pathway alongside other tumor survival and resistance pathways. We recently demonstrated that the autotaxin/LPA pathway has a role in mediating TGF-β resistance in pancreatic cancer and are excited to further explore combining IOA-359 with our autotaxin inhibitor, IOA-289, preclinically."

United Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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