While Agios Pharmaceuticals Inc. awaits this summer’s PDUFA date for the IDH-mutant glioma drug vorasidenib, the firm unveiled positive data from a global phase III study with oral mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia. (The pyruvate kinase activator was approved as Pyrukynd in February 2022 by the U.S. FDA for hemolytic anemia in adults with a deficiency of the enzyme.) At the same time in brain cancer, Medicenna Therapeutics Corp. during the American Society of Clinical Oncology meeting rolled out phase IIb data from its study with bizaxofusp for unresectable recurrent glioblastoma. Shares of Cambridge, Mass.-based Agios (NASDAQ:AGIO) were trading at $41.10, up $4.76, or 13%. Medicenna (OTC:MDNAF), of Toronto and Houston, was selling for $2.02, down 5 cents, after an early-day spike to $2.17.

Quralis licenses QRL-204 to Lilly for up to $622M total

With its hands full developing QRL-201 targeting STMN2 and QRL-101 targeting Kv7, both in clinical trials for amyotrophic lateral sclerosis (ALS), Quralis Corp. decided to out-license its preclinical splice-switching antisense oligonucleotide for ALS, QRL-204. With interest in neurodegenerative diseases back on the rise, Quralis had plenty of interest from big pharma for the drug that targets UNC13A, eventually deciding to license it to Eli Lilly and Co. for $45 million up front. Quralis will also receive an undisclosed equity investment from the Indianapolis-based pharma and is eligible for up to $577 million in milestone payments, as well as tiered royalties on net sales.

Structure regains ground on oral GLP-1 phase IIa obesity data

Shares of Structure Therapeutics Inc. (NASDAQ:GPCR) were trading up more than 60% at midday on the back of data for GSBR-1290, an oral, GLP-1 receptor agonist it hopes to position in the highly competitive obesity market. Data from a 12-week phase IIa study showed statistically significant weight reduction, with additional data highlighting results from a capsule-to-tablet pharmacokinetic study. The latest readout sees Structure regaining ground lost late in 2023 when initial phase IIa data prompted unflattering comparisons to more advanced oral GLP-1 candidates.

ASCO: Astrazeneca and Puma post strong breast cancer results

Astrazeneca plc’s blockbuster Enhertu continued to garner attention as new data released at the American Society of Clinical Oncology (ASCO) annual meeting showing the antibody-drug conjugate demonstrated strong progression-free survival of 13.2 months in HR-positive, HER2-low and HER2-ultralow metastatic breast cancer patients. Puma Biotechnology Inc. had advances of its own at ASCO with new biomarker findings from a phase II study of alisertib, an aurora protein kinase 2 inhibitor in endocrine-resistant metastatic breast cancer. The Institute of Cancer Research also presented new diagnostic data at ASCO in identifying patients whose breast cancer may relapse. The company’s new blood test found the median lead time from detection of disease to clinical relapse was 15 months.

Flagship Pioneering opens new Singapore hub, plans Seoul office

Cambridge, Mass.-headquartered Flagship Pioneering Inc. opened its Asia-Pacific (APAC) headquarters in Singapore on June 1, BioWorld learned from APAC chair and strategic advisor André Andonian in a May 30 interview. “As an Asia hub, Singapore allows Flagship to address the Singaporean, South Korean and Japanese markets and look opportunistically at other markets,” Andonian said, including China and India, given the geographic proximity. “In the near future, depending on the continuation of the positive momentum … Flagship plans to have the next office open in Seoul,” Andonian said. “I would not be surprised [at a location in South Korea,] say, in the next 12 months.”

CHMP: Re-eval for Translarna; recommendations for Ixchiq, Cejemly

The EMA has been sent back to the drawing board to re-evaluate PTC Therapeutics Inc.’s Duchenne muscular dystrophy therapy Translarna (ataluren), after failing to get the usual rubber stamp following its recommendation in January that the drug’s conditional approval be withdrawn. The Committee for Medicinal Products for Human Use (CHMP) has started to look again at the Translarna data after the European Commission told it to include an assessment of real world evidence. On a more positive note, the CHMP recommended the approval of Ixchiq, the first vaccine against Chikungunya virus infection, and the approval of Cejemly (sugemalimab) for non-small-cell lung cancer.

Genome & Co. shares jump 30% on $426M cancer deal with Debiopharm

South Korea’s Genome & Co. Ltd. (KOSDAQ:314130) shares rose nearly 30% June 3 after it disclosed a potential ₩586.38 billion (US$426 million) licensing deal for its novel antibody-drug conjugate (ADC) candidate with Switzerland’s Debiopharm International SA. Under terms of the deal signed May 31, 2024, Genome & Co. out-licensed exclusive, worldwide rights of its novel target CD239 antibody-based ADC candidate, GENA-111, to Debiopharm for ₩6.88 billion (US$5 million) in a non-refundable, up-front payment, along with potential milestone payments and royalties based on net sales. Specific milestone payments for each stage of development and commercialization were not disclosed, but the total deal could reach ₩586.38 billion, Suwon-si, Gyeonggi-do-based Genome & Co. said in the Korea Exchange filing.

ASCO: ADCs lead the data charge while AI shapes the landscape

While there was much talk about the vast potential of artificial intelligence (AI) during one of the world’s largest research cancer conferences, antibody-drug conjugates (ADCs) continued to grab the lion’s share of attention as Corbus Pharmaceuticals Holdings Inc. and Takeda Pharmaceutical Co. Ltd. released new and positive data. At the annual American Society of Clinical Oncology (ASCO) conference in Chicago, Corbus’ first-in-human study of next-generation Nectin-4-targeted ADC CRB-701 in treating metastatic urothelial cancer was shown to have a 44% overall response rate (ORR) and a 78% disease control rate (DCR). It also produced a 43% ORR and an 86% DCR in cervical cancer to date at doses greater than or equal to 1.2 mg/kg. No dose-limiting toxicities were seen in doses up to and including 4.5 mg/kg.

ASCO: KRAS and lung cancer data from Lilly, Astrazeneca and Catalym

Eli Lilly and Co.’s updated phase I/II study of olomorasib as a monotherapy in patients with KRAS G12C-mutant advanced solid tumors yielded more promise in data released over the weekend at the American Society of Clinical Oncology (ASCO) annual meeting. The progress in KRAS-targeted therapies has been slow but steady, according to Kevan Shokat, a professor of cellular and molecular pharmacology at the University of California, San Francisco, who spoke to ASCO on Sunday about the history of the therapy from bench to bedside. He said KRAS had been an outlier for years but blocking KRAS meant the drug candidate had to find a pocket because it had previously been undruggable.

BioWorld Insider Podcast: ADCs and radiotherapies at the forefront of this year’s
ASCO conference

Chris Barden, a co-managing partner at MPM Bioimpact who manages the firm’s Bioimpact Equities and Oncology Impact funds, shares her insights into the American Society of Clinical Oncology (ASCO) conference in Chicago. She talks about the major trends in oncology, including development of antibody drug conjugates, which is currently the hottest area in cancer treatment.

Also in the news

Abbvie, Astellas, Biogen, Blueprint Medicines, Bristol Myers Squibb, Calliditas, Catalyst, Eisai, Gilead, Grit, Invivyd, Inxmed, Ionis, Larimar, Lixte, Medicenna, Mimivax, Minoryx, Mirum, Nextcure, Novartis, Pfizer, Pharming, Quangang, Rapport, Sanofi, Sifi, SN, Sumitomo, Summit, Takeda, Telix, Ultragenyx, Vanda