Global Roundup: FDA Hits Junshi, HUTCHMED with Complete Response Letters

Shanghai-based Junshi Biosciences was hit with a Complete Response Letter for its cancer treatment, toripalimab.

Along with partner Coherus BioSciences, the company received the CRL from the U.S. Food and Drug Administration for toripalimab in combination with gemcitabine and cisplatin in the first-line treatment of patients with advanced recurrent or metastatic nasopharyngeal carcinoma. The CRL also covered toripalimab monotherapy in the second-line or later treatment of recurrent or metastatic NPC after platinum-containing chemotherapy.

The FDA letter calls for a “quality process change” that Coherus and Junshi Biosciences announced is readily addressable. The two companies will meet with the FDA and anticipate a resubmission of the Biologics License Application later this summer. A review timeline would be at least six months due to travel restrictions related to the COVID-19 pandemic in China.

“Junshi Biosciences is dedicated to the discovery, development and commercialization of innovative new drugs on a global scale,” Ning Li, chief executive officer of Junshi Biosciences said in a statement. “Toripalimab, our PD-1 inhibitor, has demonstrated a compelling clinical profile in studies across multiple tumor types and is currently approved in China for four indications. We fully support our partner, Coherus, in its efforts to seek toripalimab approval in the United States for advanced nasopharyngeal carcinoma, as well as in the subsequent commercial launch, if approved. Our respective teams are working diligently together in a well-coordinated effort to achieve these goals as partners.”

Elsewhere around the Globe:

HUTCHMED: Based in Hong Kong, HUTCHMED was also the recipient of a CRL from the FDA for surufatanib, an oral inhibitor of angiogenesis and immune modulation. The drug was being developed for the treatment of pancreatic and extra-pancreatic neuroendocrine tumors. HUTCHMED said the FDA determined that the current data package, based on two positive Phase III trials in China and one bridging study in the United States, does not support approval in the U.S. at this time. The CRL indicated that a multi-regional clinical trial is required for U.S. approval. Surufatinib received the FDA Fast Track designation from the FDA in April 2020 for the treatment of pNETs and epNETs. Orphan Drug Designation for pNETs was granted in November 2019.

LianBio: China-based LianBio and T posted positive data from a Phase III study assessing TP-03 (lotilaner ophthalmic solution, 0.25%). With the positive data, Tarsus intends to seek approval from the FDA in the second half of 2022. LianBio licensed rights from Tarsus in March 2021 for the development and commercialization of TP-03 in Greater China. LianBio expects to initiate a registrational Phase III clinical trial of TP-03 in the second half of 2022 to support regulatory approval in China.

Lumicks: Amsterdam-based LUMICKS announced a landmark study in Nature demonstrating the capabilities of LUMICKS’ z-Movi Cell Avidity Analyzer to measure cell avidity, a crucial biomarker for understanding immune cell function and for providing superior predictive information of therapeutic efficacy. The study was considered groundbreaking as it exposed a mechanism solid tumors use to evade CAR T cell therapies. Solid tumor cells avoid CAR T cell killing by down-regulating the adhesion strength or “cell avidity” between the effector and its target, which is essential in successfully clearing a solid tumor. Measuring cell avidity should now be an integral part of CAR T cell development programs to help reduce solid tumor clinical trial failure rates. The insufficient cell avidity was determined to be the mechanism underpinning poor in vivo therapeutic behavior of CAR T cells, identifying “avidity escape” as a new avoidance mechanism used by solid tumors.

OSE Immunotherapeutics: France’s OSE Immunotherapeutics SA received a new patent from the European Patent Office that bolsters the protection covering its novel myeloid cell immune checkpoint target, CLEC-1 (a C-type lectin receptor), and its use in cancer treatment. The patent provides a protection until 2037. CLEC-1 is a C-type lectin receptor that has demonstrated the potential to inhibit myeloid cells’ functions and block anti-tumor responsiveness of T-lymphocytes. Myeloid cells have the ability to accumulate in the tumor microenvironment and deregulate the immune activation of T-lymphocytes. CLEC-1 is a new therapeutic target of interest in immuno-oncology.

Imagene AI: Israel-based Imagene AI raised a total of $21.5 million, which includes $3 million in seed funding and $18.5 million in Series A financing. The company is developing AI diagnostic technology capable of delivering real-time molecular analysis. The diagnostics results are able to be received within minutes rather than days or even weeks. According to the company, its technology classifies patterns that cannot be seen by the human eye, evolving genomics, proteomics, and spatial insights to better understand the recurrence of specific cancers and their resistance mechanisms; this enables better stratification of patients in clinical trials and the discovery of novel targets for new drugs.

ACELYRIN, Inc.: ACELYRIN, Affibody AB and Immagene Biopharmaceuticals announced positive results from a Phase II trial of izokibep in 135 patients with psoriatic arthritis. The study met the primary endpoint of ACRO50, as well as meeting secondary endpoints that included PASI response, enthesitis LEEDs improvement, and quality of life improvement. Details of the trial data will be shared by podium presentation at the European Alliance of Associations for Rheumatology Congress on June 3.

Tubulis: Germany-based Tubulis secured €60 million ($63 million) in a Series B financing round. Proceeds from the round will be used to advance Tubulis’ proprietary pipeline of uniquely assembled antibody-drug conjugates (ADCs) towards clinical evaluation and introduce programs addressing a range of solid tumor indications. The proceeds will enable the company to deliver the true therapeutic potential of ADCs through further innovation of novel payload classes and the identification of new cancer targets. Investors include Evotec and Fund+, as well as Bayern Kapital, BioMedPartners, coparion, High-Tech Gründerfonds (HTGF), OCCIDENT and Seventure Partners.

XORTX Therapeutics: In Calgary, Alberta, XORTX dosed the first human subjects in the XRX-OXY-101 bridging pharmacokinetics study. Following dosing, blood sampling and bioanalytical evaluation will be conducted to characterize the pharmacokinetics and bioavailability of the XRx-008 program’s novel proprietary formulations of oxypurinol for future clinical trials development.

Ilya Pharma: Based in Sweden, Ilya Pharma received IND clearance for first-in-class ILP100-Topical immunotherapy treatment of surgical wounds in patients with prediabetes, diabetes and obesity. ILP100-Topical is a first-in-class immunotherapy designed to treat surgical wounds in prediabetes, diabetes, and obesity patients. These patients have weeks to months of delayed healing following surgery and a substantially increased risk of serious wound complications.

Byondis BV: A Dutch clinical stage biopharmaceutical company, Byondis has entered into a licensing agreement with medac GmbH, a privately owned pharmaceutical company based in Germany. Byondis and medac will partner to commercialize Byondis’ lead program, anti-HER2 antibody-drug conjugate (ADC) trastuzumab duocarmazine (SYD985). SYD985 targets a range of HER2-expressing cancers such as metastatic breast and endometrial (uterine) tumors.

MIMETAS: Also based in the Netherlands, MIMETAS has expanded its license to the HUB Organoid Technology to include all organs. The license covers organoid technology developed by Professor Hans Clevers, a pioneer in adult stem cells.

Genenta Science: Based in Italy, Genenta announced preliminary findings from a Phase I/IIa study of Temferon in patients with glioblastoma multiforme patients who have an unmethylated MGMT gene promoter (uMGMT-GBM). The data showed evidence of Temferon’s potential for modulating the tumor microenvironment as well as details of Temferon’s continuing positive safety profile and signals of biological activity. Findings from the study will be presented later this month at the American Society of Gene and Cell Therapy.

CYTENA: Germany’s Cytena and ExcellGene SA of Switzerland entered into a collaboration to streamline cell line development workflows for highly-efficient monoclonal antibody (mAb) and complex therapeutic protein manufacturing, which enables essential improvements to the development workflows of the biopharmaceutical industry.

Immutep Limited: Australia’s Immutep posted new biomarker and exploratory analysis data from its Phase IIb AIPAC trial. The double blind and randomized AIPAC trial evaluated efti in combination with paclitaxel chemotherapy (efti group) compared to placebo plus paclitaxel (placebo group) in 227 patients with HER2-negative/HR-positive metastatic breast cancer. Final OS results were reported in November 2021 in a late breaking abstract at SITC, showing encouraging efficacy in multiple patient subgroups.

Mologic: Based in the U.K., Mologic and Alamar Biosciences forged a collaboration to utilize Alamar’s novel antibodies against SARS-CoV-2 Nucleocapsid (N) protein for Mologic’s future COVID-19 RDTs. Easy-to-use and cost-effective RDTs are a key tool for controlling the spread of COVID-19, including in low resource settings.