BO-112

Saniona, a rare disease-focused company based in Sweden, received feedback from the U.S. Food and Drug Administration (FDA) on a path forward for its hypothalamic obesity (HO) drug Tesomet. Based on guidance from the regulatory agency, Saniona is planning a Phase IIb study in this indication in the first half of this year. “There is currently no medicine approved for hypothalamic obesity, a rare disease secondary to hypothalamic injury, characterized by intractable weight gain and complicated by uncontrollable hunger,” Rudolf Baumgartner, Chief Medical Officer and Head of Clinical Development at Saniona said in a statement. “We are encouraged by this feedback from the FDA and look forward to continuing a constructive dialogue with them as we prepare to initiate our Phase IIb clinical trial with Tesomet.” Saniona previously announced that the FDA had highlighted the potential for off-label use of Tesomet in the general obese population. As a result, Saniona submitted a response proposing a Risk Evaluation and Mitigation Strategy (REMS), which can be used to restrict commercial distribution to the appropriate patients suffering from an unmet medical need. According to the company, the FDA indicated overall agreement with this proposal and stated Saniona should demonstrate that HO fulfills the criteria for an unmet medical need. Saniona expects to file an Investigational New Drug (IND) application and initiate its planned Phase IIb clinical trial in HO in the first half of this year. In a 24-week, double-blind, randomized, placebo-controlled Phase II trial in HO, Tesomet was well-tolerated, and treated patients demonstrated statistically significant reductions in body weight and clinically meaningful improvements in waist circumference and glycemic control. These improvements were maintained during an additional 24-week open-label extension, with no clinically meaningful differences in heart rate or blood pressure observed. Saniona is also evaluating Tesomet for the treatment of Prader-Willi syndrome (PWS) and plans to begin a Phase IIb trial in this indication in the first half of this year. Elsewhere around the globe: ClinSpec Diagnostics – Scotland-based ClinSpec reached its round two funding target of £3.5 million by securing an additional investment of £1.1 million. ClinSpec Diagnostics, which is a spinout from the Department of Pure and Applied Chemistry at the University of Strathclyde, develops liquid biopsies for early detection of cancer. Vetter – Germany’s Vetter has opened a new business entity in Shanghai, China. The new office is expected to increase the visibility of Vetter’s presence in China and underlines its importance as an important strategic market to Vetter. China offers a promising injectable pipeline and allows the Contract Development and Manufacturing Organization (CDMO) to offer support to domestic companies that plan on bringing their molecules to the global market, Vetter said. The new office, located in Puxi, Shanghai, follows Vetter’s business development activities in its APAC regional office in Singapore, as well as in its sales offices in Japan and South Korea, further strengthening its footprint in the Asia Pacific region. Wuxi Biologics – China’s WuXi and California-based Exelixis, Inc. have entered into a licensing deal that will support the continued expansion of Exelixis’ oncology biologics pipeline. , Exelixis will make an upfront payment to WuXi Bio in exchange for an exclusive license to a panel of monoclonal antibodies to a preclinically validated target, discovered based on WuXi Bio’s integrated technology platforms, for the development of antibody-drug conjugate, bispecific, and certain other novel tumor-targeting biologics applications. Avacta Group – U.K.-based Avacta Group announced the AffiDX SARS-CoV-2 rapid antigen lateral flow test detects the dominant new variants of the coronavirus, known as the B117, or ‘Kent’, variant, and the D614G variant, as well as the original strain. Avacta has carried out analytical tests with the spike proteins isolated from both the B117 and D614G variants, and has confirmed that its AffiDX® SARS-CoV-2 rapid antigen lateral flow test detects both of these variants as well as the original strain. Also this week, Avacta Group entered into a license agreement with Biokit, a Werfen Company, to incorporate Affimer reagents into a Biokit in-vitro diagnostic (IVD) product. Steba Biotech – Based in Luxembourg, Steba Biotech received Orphan Drug Designation from the FDA for Padeliporfin ImPACT, a potential treatment of adult patients with low-grade and unifocal high-grade Upper Tract Urothelial Cancer (UTUC). The FDA has previously given the green light to the IND application, allowing initiation of the pivotal Phase III clinical trial of Padeliporfin ImPACT in patients with low-grade UTUC, which is expected to begin enrollment later this month. Padeliporfin ImPACT has also received Fast Track designation from the FDA for the treatment of adult patients with low-grade and unifocal high-grade UTUC. Steba is focusing on the development of Padeliporfin ImPACT as an innovative oncology platform with the potential to offer surgery-like efficacy, combined with organ preservation in UTUC and other solid tumors for high-risk surgical patients with unmet needs – either because surgery is not the preferred clinical option (e.g., to delay the loss of a kidney) or the risk of surgery is too high. Nonacus – Genetic testing company Nonacus, based in the U.K., announced the commercial launch of a new service for Whole Genome Sequencing of SARS-CoV-2, developed to enable the U.K. government’s Test-To-Release scheme. During the pandemic, Nonacus expanded its accredited service facility to support a complete SARS-CoV-II testing workflow and developed the new rapid Whole Genome Viral Sequencing library approach with nanopore sequencing. IONTAS Limited – U.K.-based IONTAS and Portugal’s FairJourney Biologics SA entered into an agreement with London-based Quell Therapeutics Limited (Quell) regarding IONTAS / FJB’s proprietary libraries and technology platforms. Quell is harnessing the suppressive capacity of Tregs to develop engineered Treg cell therapies to address several conditions of immune dysfunction. Under the terms of the agreement, IONTAS / FJB will provide Quell with a diverse panel of novel target binders, ranging in affinities, to enable Quell to select functional binders for chimeric antigen receptor (CAR) development against undisclosed targets. The collaboration is non-exclusive and further terms of the agreement are not being disclosed. Mainstay Medical Holdings – Mainstay, based in Ireland, announced the commercial launch in Australia of ReActiv8, its implantable neurostimulation system to treat chronic low back pain. Affibody AB – Affibody AB and Inmagene Biopharmaceuticals were cleared by the FDA to move their interleukin-17 (IL-17) blocker izokibep (ABY-035) to proceed to Phase II clinical development in non-infectious intermediate, posterior, and pan-uveitis, an orphan disease with significant unmet medical need. The study is a one-year Phase II trial in approximately 110 patients with active Uveitis. The trial will evaluate the efficacy, safety and tolerability of izokibep as compared to standard of care. G&L Scientific – U.K.-based G&L Scientific, a regulatory affairs expert, expanded into Europe with new dedicated client services in Spain, Italy, Germany and France. This latest development will further bolster their presence in the European market and complement existing teams in the U.K., the U.S., Canada, and Asia, the company said. Grifols – Spain’s Grifols announced the closing of its acquisition of GigaGen Inc. Griols acquired all outstanding GigaGen stock for $80 million. GigaGen specializes in the early discovery and development of recombinant biotherapeutic medicines. GigaGen’s research focuses on discovering new biological treatments based on antibodies derived from millions of immune system cells obtained from donors. A key asset gained in the deal is GIGA-2050, a recombinant hyperimmune immunoglobulin for the treatment of COVID-19, which is expected to be evaluated in a Phase I trial in the U.S. in spring 2021. Orionis Biosciences – Belgium-based Orionis and The University of Texas MD Anderson Cancer Center launched Project Helios, a research collaboration designed to unlock new drug development opportunities through genome-scale mapping of drug-target interactions. Project Helios will conduct systematic and unbiased mapping of drug interactions across the human proteome with a large portfolio of bioactive, chemically diverse small molecules, including experimental and approved drugs. The findings may provide opportunities to repurpose approved therapies, optimize therapeutic approaches for known targets and develop therapies for novel targets. Biologic Design – Israel’s Biologic Design Ltd. and Eli Lilly announced a research collaboration and license agreement that will leverage Biolojic's AI-based multibody platform to discover and develop a potential novel antibody-based therapy for the treatment of diabetes. Under the terms of the agreement, Lilly will pay research fees associated with the collaboration and additionally, Biolojic is eligible to receive up to a total of $121 million, consisting of potential development and commercialization milestones and a promissory note that may be convertible into Biolojic equity at a future date. Orca Therapeutics – Netherlands-based Orca Therapeutics BV announced that following a review from an Interim Data and Safety Monitoring Board, the company will continue its Phase I/IIa clinical study of ORCA-010 in treatment-naïve patients with localized prostate cancer. Based on this recommendation, ORCA Therapeutics will initiate administration of the highest scheduled dose level of ORCA-010 in the single dose part of the study. Pivotal –A European-wide contract research organization, Pivotal and partner Highlight Therapeutics announced that the first patients have been recruited in a Phase IIa study to assess Highlight’s lead program BO-112 in combination with an anti-PD1 therapy, in patients with unresectable or metastatic melanoma that have previously progressed to checkpoint inhibitors. The study will evaluate the anti-tumoral activity and systemic exposure of repeated intratumoral injections of BO-112 into a tumoral lesion, in combination with intravenously administered anti-PD1. BO-112 has intrinsic anti-tumoral effects, but interestingly acts on several mechanisms involved in resistance to checkpoint inhibitors. ERS Genomics – Ireland’s ERS Genomics Limited, which was formed to provide broad access to the foundational CRISPR/Cas9 intellectual property, announced a non-exclusive license agreement granting Setsuro Tech access to ERS Genomics’ CRISPR/Cas9 patent portfolio for Japan. Setsuro Tech has developed a high-throughput genome editing method for mammalian embryos, termed genome editing by electroporation of Cas9 protein (GEEP). Using this method, the company is able to generate genetically engineered mice at low cost and in a short timeframe. Setsuro Tech is applying CRISPR/Cas9 technology to create genome-edited cell and animal models, based on end user requirements. Evolve Biologics – Ontario-based Evolve Biologics provided an update on its continued progress toward the development and commercialization of its PlasmaCap EBA technology. Evolve continues to progress in its plan for the construction of its first commercial facility in the US and is now in late stages of site selection. The company expects to finalize and announce the future location of its facility by spring 2021.

Highlight Therapeutics and Pivotal work together on Melanoma therapy andlaunch a Phase IIa trial to examine BO-112 efficacy and safety MADRID, Spain, March 02, 2021 (GLOBE NEWSWIRE) -- Highlight Therapeutics, ("Highlight"), a clinical-stage biopharmaceutical company developing RNA-based therapies against cancer, and Pivotal, a Europe-wide full-service CRO, today announced that the first patients have been recruited in a Phase IIa study to assess Highlight's lead program BO-112 in combination with an anti-PD1 therapy, in patients with unresectable or metastatic melanoma that have previously progressed to checkpoint inhibitors. Melanoma is the most malignant tumor of the skin although it can be seldom found in other organs. Incidences of this tumor are rapidly increasing in western countries and, once disseminated, it has been considered an incurable disease with limited therapeutic options. Recently, immunotherapy with anti-PD1 (checkpoint inhibitors) showed encouraging results with 30-36% patients alive at 5 years. Unfortunately, the median PFS (progression free survival) is less than 12 months, mainly due to primary or acquired resistance to anti-PD1 treatment, and most of those patients will die due to the tumor or its complications. "This Phase IIa study is an important step forward in our strategy to develop effective cancer therapies which can be used in combination with checkpoint inhibitors. We are looking to produce a better immunological response in anti-PD1 therapy-sensitive patients, and to induce or maintain responses for those patients that progress or are initially treatment-resistant. BO-112 has the potential to be employed from the beginning of disease treatment and we believe it offers patients a resistant status after immunological treatment," said Dr. Marisol Quintero, PhD, CEO of Highlight Therapeutics. "We are encouraged by the effectiveness already seen in previously treated melanoma patients in the phase I study with BO-112 and we are pleased to be working once more with the highly experienced and dedicated team at Pivotal." This Phase IIa, open-label clinical study is a non-comparative trial implemented in 19 sites across Spain and France. The protocol will include a minimum of 40 non-resectable melanoma patients. This is the third trial with BO-112, following initiation of the phase I trial in 2016. In 2020, a second trial was initiated in gastrointestinal tumors from which the first cohorts have already been successfully completed, and the recruitment of this phase IIa trial has now been initiated in melanoma despite the obstacles presented due to the COVID-19 pandemic. The study will evaluate the anti-tumoral activity and systemic exposure of repeated intratumoral injections of BO-112 into a tumoral lesion, in combination with intravenously administered anti-PD1. BO-112 has intrinsic anti-tumoral effects, but interestingly acts on several mechanisms involved in resistance to Checkpoint inhibitors. "The excellence in clinical research of the clinical investigators´ teams, together with Pivotal's infrastructure and vast experience in the implementation and performance of innovative early phases clinical trials, will allow us to accelerate the research and to quickly test this new treatment regimen," said Dr. Lourdes Huarte, PhD, Senior Vice President of Regulatory and Clinical Operations at Pivotal. "The challenge of this trial was to swiftly implement the study and activate its recruitment in a period negatively impacted by the COVID-19 pandemic. We are delighted to have diligently achieved our first milestone with the recruitment of the first patients in this trial." About Highlight Therapeutics Highlight Therapeutics, formerly known as Bioncotech Therapeutics, is a private, clinical-stage company dedicated to unlocking the full potential of immuno-oncology. Our lead drug candidate BO-112 is a best-in-class RNA-based therapy which has been demonstrated to initiate a powerful immune response, leveraging a unique multi-target approach to turn 'cold' tumors 'hot' and therefore visible to the immune system. It has the potential to rescue patients who are resistant to current checkpoint inhibitor therapy, a very large market opportunity. BO-112 is currently being investigated in a range of clinical trials as a monotherapy and in combination with checkpoint inhibitors. In addition to in-house research, Highlight Therapeutics has a number of external collaborators, including Merck & Co and UCLA. For more information, please visit ; or Contacts About Pivotal Pivotal was founded in 2001 by Dr. Ibrahim Farr on the principle that strategic medical advice and support should be the backbone of all clinical trials. After working for over two decades in the pharmaceutical industry, Dr. Farr recognized the need for a medium-sized CRO with a solid internal medical franchise that could act not only as the "doers" but also as the "co-thinkers" for their clients, through its strategic scientific advice. To date, we are the trusted adviser and counsellor for many companies to deliver maximum value in their drug and medical devices development programs. We are a leading privately held European CRO and, since inception, we have experienced a fast and steady organic growth in Europe. Pivotal´s client portfolio spans major pharmaceutical, biotechnological, medical device and nutrition companies, as well as independent investigators and cooperative groups. We have long-standing relations with over 200 clients. Pivotal has extensive experience across major therapeutic areas and phases I to IV. Our highly customized teams bring to each client a combination of broad industry knowledge and operational excellence, to offer our clients fresh perspectives and breakthrough business insights. Additionally, we have built a strong oncology, innovative therapies, infectious diseases, vaccines, rare diseases and early phases hub that enables us to tackle our customers most difficult challenges, turning recommendations into concrete actions. By remaining true to our core principles and values, our vision is to become our client's preferred outsourcing solution partner. For more information, please visit ; orContact Ms Natalia Farr at Pivotal natalia.farr@pivotalcr.com This information is provided by Reach, the non-regulatory press release distribution service of RNS, part of the London Stock Exchange. 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