Immune-Onc Therapeutics, Inc.

Dupixent is approved by the FDA in five indications namely atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis, eosinophilic esophagitis and prurigo nodularis. Image Credit: SewCreamStudio/ Shutterstock. Sanofi and Regeneron are close to adding chronic obstructive pulmonary disease (COPD) as the sixth indication for its blockbuster therapy Dupixent (dupilumab). The US Food and Drug Administration (FDA) has granted priority review for a supplemental biologics licence application (sBLA) for Dupixent as an add-on maintenance treatment for certain adult patients with uncontrolled COPD. The agency has set a target date of 27 June 2024. Dupixent, a monoclonal antibody that inhibits the signalling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways, is already approved to treat atopic dermatitis , asthma, chronic rhinosinusitis with nasal polyposis, eosinophilic esophagitis and prurigo nodularis. As per Regeneron’s financials, Dupixent pulled in $11.59bn in global sales in 2023. GlobalData forecasts the therapy revenue to maintain its upward trajectory and generate $20.4bn in sales in 2030. GlobalData is the parent company of Pharmaceutical Technology . See Also: FDA generic drug approvals rose in 2023 in bid for improved access Immune-Onc’s antibody therapy collects second FDA orphan designation Dupixent’s sBLA is supported by data from the Phase III NOTUS study (NCT04456673) and the Phase III BOREAS study (NCT03930732). In November 2023, the companies reported that an interim analysis from the NOTUS trial showed that patients with COPD who were treated with Dupixent demonstrated a 34% reduction in moderate or severe exacerbations at 52 weeks. The published data from the BOREAS trial also showed that Dupixent resulted in a 30% reduction in moderate or severe acute COPD exacerbations over 52 weeks. Sanofi and Regeneron are also engaging with regulatory bodies in China and the European Union. Dupixent is currently under review as an add-on maintenance treatment in certain adult patients with uncontrolled COPD by two regulatory agencies, namely China’s National Medical Products Administration (NMPA) and the European Commission. While Dupixent is making strides in COPD, it fell short for the treatment of chronic spontaneous urticaria (CSU) when the FDA rejected the sBLA for the therapy in October 2023, stating that more efficacy data was needed to support Dupixent’s approval in CSU. However, Sanofi and Regeneron plan to resubmit the sBLA, supplemented by data from the Phase III LIBERTY-CUPID Study C trial (NCT04180488). Results from the trial are expected in late 2024. Although last week, Dupixent was approved as a treatment for chronic spontaneous urticaria (CSU) in patients aged 12 years and older by the Japanese Ministry of Health, Labor and Welfare

IO-202 is a monoclonal antibody that targets leukocyte immunoglobulin-like receptor subfamily B member 4 (LILRB4), which can suppress immunity in solid tumours. Image Credit: crystal light / Shutterstock. The US Food and Drug Administration (FDA) has granted an orphan drug designation to Immune-Onc Therapeutics’ IO-202 for the treatment of a rare blood cancer – chronic myelomonocytic leukaemia (CMML). IO-202 had previously received a fast track designation from the US FDA as a treatment of relapsed or refractory (r/r) CMML in 2023. The therapy was also granted both orphan drug and fast track designations as a treatment for acute myeloid leukaemia (AML) by the FDA in 2020 and 2022, respectively. The orphan drug designations allow for an extended market exclusivity of seven years in the US and developmental incentives such as an exemption of FDA application fees and tax credits for qualified clinical testing. The fast track designations enable earlier interactions with the FDA for the pursuit of accelerated approval. The status also opens chances of rolling reviews. IO-202 is a monoclonal antibody that targets leukocyte immunoglobulin-like receptor subfamily B member 4 (LILRB4), which can suppress immunity in solid tumours. It also mediates the T-cell suppression and infiltration of AML cells. It is being investigated as a monotherapy and in combination with chemotherapy, azacytidine, along with targeted therapy AbbVie /Genentech’s Venclexta (venetoclax), in a Phase I trial (NCT04372433). The study is expected to enrol approximately 106 patients with either r/r AML or r/r CMML. It is expected to conclude in 2026, as per ClinicalTrial.gov. See Also: Harmony wins orphan drug tag for pitolisant to treat Prader-Willi syndrome United Therapeutics sues FDA over handling of Liquidia’s NDA A second Phase I trial (NCT05309187) investigating IO-202 in combination with a PD-1 immune checkpoint inhibitor, MSD’s Keytruda (pembrolizumab) is also currently underway. The study enrolled approximately 200 patients with advanced solid tumours and is expected to be completed in April. Monoclonal antibodies have been identified as a key innovation area for cancer therapy by GlobalData, with multiple companies investing to develop antibody therapies for various indications. In December 2023, AbbVie partnered with BigHat Biosciences to identify and develop antibody therapies in oncology and neuroscience indications. GlobalData is the parent company of Pharmaceutical Technology . Almirall is developing an interleukin-21 (IL-21)-hindering monoclonal antibody, NN-8828, for the treatment of immune-inflammatory dermatological ailments. The company acquired the therapy from Novo Nordisk last week for an undisclosed upfront payment and will be entitled to developmental and commercial milestones, as well as tiered royalties on future global sales.

Tyvaso has entered blockbuster territory, seeing global sales of $1.2bn in 2023, up 41% from 2022 sales. Image credit: Shutterstock/viewimage. United Therapeutics has sued the US Food and Drug Administration (FDA), claiming the agency did not follow rules when approving a change to rival Liquidia’s new drug application (NDA). The US biotech accused the agency of permitting Liquidia to “skirt longstanding FDA rules, precedents, and procedures on how pending drug approval applications are handled”, according to a 21 February press release. Commencing litigation is yet another chapter in the long saga between United and federal agencies, centred on the company’s ageing pulmonary arterial hypertension drug Tyvaso (treprostinil). Tyvaso is indicated for pulmonary arterial hypertension (PAH) , for which it was approved in 2009, and pulmonary hypertension associated with interstitial lung disease (PH-ILD), as per its drug label. Tyvaso has entered blockbuster territory, seeing global sales of $1.2bn in 2023, up 41% from 2022 sales. A dry powder inhaler (DPI) formulation, approved for both indications in May 2022, accounted for nearly two-thirds of these 2023 sales. It is estimated to make United $2.7bn by 2029, according to GlobalData’s Pharma Intelligence Centre. See Also: Immune-Onc’s antibody therapy collects second FDA orphan designation Charles River and Wheeler Bio partner on therapeutic discovery GlobalData is the parent company of Pharmaceutical Technology. United’s original patent, which would have granted protection until May 2027, was declared invalid and cancelled by the Patent Trial and Appeal Board (PTAB) in 2022. A year later, United attempted to revive the patent, but a US appeals court upheld the earlier decision, opening the regulatory door for Liquidia’s version of trepostinil, Yutrepia, to enter the market. Liquidia’s drug, also a DPI, was granted tentative approval by the FDA for the treatment of PAH in 2021 – a type of clearance used when patent or exclusivity issues are ongoing. Feathers were ruffled when Liquidia amended its NDA to include PH-ILD as an indication. The FDA delayed the Prescription Drug User Fee Act (PDUFA) date for the drug after the change to the application, with no timeline currently set. United now calls Liquidia’s NDA amendment “unlawful”, stating that a new NDA is needed for the PH-ILD inclusion. Currently, there is only one patent left in the infringement battle. United Therapeutics’ global regulatory affairs executive vice-president Dean Bunce said legal and regulatory precedents are sometimes missed. Bunce said: “We are simply asking that the FDA apply its own rules and precedents consistently to honour the Hatch-Waxman balance struck by Congress between innovators and imitators: Liquidia can rely on United Therapeutics’ innovation to speed its path to market, but the cost of that shortcut is that Liquidia must address the infringement claim against it before rushing to market.” United highlighted that Liquidia avoided a 30-month window in which the FDA cannot approve an NDA amidst patient infringement, as per the Hatch-Waxman Act. The 30-month delay in Liquidia’s PH-ILD indication approval could be enacted if United were successful in its litigation. In a January 2024 statement relating to the NDA amendment, Liquidia said: “The sole remaining patent […] was not issued before Liquidia submitted the NDA for Yutrepia in January 2020 to treat PAH. “Therefore, the Company believes that final FDA approval for Yutrepia will not be subject to any statutory 30-month stay arising from the New Hatch-Waxman Litigation per Section 505(c)(3)(C) of the Federal Food, Drug and Cosmetic Act.”