Rexmyelocel T (Ixaka)

Italfarmaco Group, based in Milan, Italy, is making headway against Duchenne muscular dystrophy (DMD). The company announced positive results from the Phase III EPIDYS study assessing Givinostat, a histone deacetylase (HDAC) inhibitor that received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration. The study met its primary endpoint, a positive change from baseline in the time to climb four stairs at 18 months compared to placebo. The results demonstrated a slower decline in performing this functional task in the Givinostat-treated group. Additionally, the company noted that key secondary endpoints were also met, consistent with the primary endpoint. Givinostat continued to demonstrate a tolerability pro line with previous studies. In all, the study results show that the addition of Givinostat to steroid treatment leads to clinical benefits for DMD patients. Italfarmaco intends to submit the data to the FDA as well as European regulators for potential approval. Additionally, the company will present the results of the Phase III study for publication in a peer-reviewed journal. “These topline Phase III results in DMD add to the growing dataset we have collected over the last years which have shown positive outcomes with our investigational therapeutic candidate, Givinostat, when taken together with steroids in patients with DMD, providing new hope for boys with DMD, their families and the medical community,” Paolo Bettica, chief medical officer of Italfarmaco Group said in a statement. “There is a tremendous unmet medical need for additional drugs to treat this debilitating rare disease and with these positive results, we intend to meet with regulatory agencies to share these findings and discuss a path forward to submit the complete dataset in a marketing application for potential approval.” Elsewhere around the Globe: Clover Biopharmaceuticals: Based in Shanghai, Clover announced new positive clinical data in individuals who have received a third dose of SCB-2019 (CpG 1018/Alum) as a homologous booster against the Omicron variant of COVID-19. The third dose demonstrated a significant, 19-fold increase in neutralizing antibody levels against the Omicron BA.2 variant compared to pre-booster levels among baseline seronegative participants. Immutep Limited: Australia’s Immutep announced new data from second-line non-small cell lung cancer patients in the Phase II TACTI-002 trial will be presented at the IASLC 2022 World Conference on Lung Cancer in August. EnGeneIC: Also based in Australia, EnGeneIC, is conducting studies of its COVID-19 vaccine that it says provides immunity against all known variants. Trials have shown the novel vaccine works by stimulating an entirely different immune pathway from other vaccines, producing “high affinity” antibodies that neutralize all COVID-19 variants, EnGeneIC said. Thirty-two healthy participants received two doses three weeks apart. Of those, 27 have passed the 28-day safety assessment with no side effects. Critically, they all have high-affinity antibodies capable of neutralizing all COVID-19 mutants, including Omicron. Noema Pharma: Switzerland-based Noema’s Investigational New Drug Application for a Phase IIb study of the company’s PDE10A inhibitor gemlapodect has been greenlit by the U.S FDA. The company will evaluate the drug candidate in Childhood Onset Fluency Disorder, also known as stuttering. The Phase IIb study is expected to read out at the end of 2023. Treos Bio Limited: Based in London, Treos Bio announced the dosing of its first patient in the Phase II OBERTO 301 study of PolyPEPI1018, the company’s lead product candidate. In the trial, PolyPEPI1018 is being combined with atezolizumab, Roche’s PD-L1 inhibitor, for the treatment of patients with late-stage microsatellite stable metastatic colorectal cancer. Ixaka Ltd.: Also based in London, Ixaka Ltd. and Germany’s Minaris Regenerative Medicine GmbH have signed an agreement that enables technology transfer and clinical and commercial manufacture of REX-001, Ixaka’s lead autologous multi-cell therapy. REX-001 is being developed for the treatment of chronic limb-threatening ischemia in patients with diabetes. The scope of the manufacturing agreement includes the scale-up and technology transfer with the goal of supplying the Phase II clinical trials and supporting commercial supplies in the EU and later in the United States. Xenikos B.V.: Located in The Netherlands, Xenikos enrolled the first patient in its pivotal Phase III trial, comparing T-Guard with ruxolitinib for the treatment of patients with Grade III or IV steroid-refractory acute graft-versus-host disease following allogeneic hematopoietic stem cell transplant. Xenikos expects the data from this study to support the submission of a Biologics License Application in the U.S. based on Day 28 data from the first 150 patients. Cyxone: Sweden’s Cyxone filed a submission to the National Institute of Pharmacy and Nutrition, the Hungarian Medical Product Agency, for approval for a Phase IIb study assessing drug candidate rabeximod as a potential treatment for rheumatoid arthritis. Applications will follow this first submission to relevant regulatory authorities in other European countries, the company said. Transgene: Based in France, Transgene and Sweden’s BioInvent International AB announced positive safety and progress in the ongoing Phase I/IIa study assessing BT-001 in patients with solid tumors, including melanoma. The initial data generated in Phase I part A demonstrated that BT-001 alone is well tolerated, with first signs of anti-tumor activity in a hard-to-treat population. BT-001 is based on Transgene’s oncolytic vector and is encoding BioInvent’s proprietary anti‑CTLA‑4 antibody. Confo Therapeutics: Belgium’s Confo Therapeutics has been awarded a €1.7 million grant from Flanders Innovation & Entrepreneurship that will support its discovery programs for novel GPCR candidates with its proprietary ConfoBody technology. MRM Health: Also based in Belgium, MRM Health announced it received regulatory approval from the Federal Agency for Medicines and Health Products in Belgium to initiate a Phase II trial with the novel next-generation optimized consortium therapy, MH002, in patients with Pouchitis, inflammation of the surgically constructed pouch following colectomy. SciSparc Ltd.: Israel-based SciSparc announced that it completed the development of its top-tier drug candidate SCI-110, which will be used in its upcoming Phase IIb study. "Our singular objective in developing SCI-110 is to develop a safe and effective treatment for this devastating, unmet medical need as soon as possible; and this milestone brings us one step closer," SciSparc CEO Oz Adler said.

Italfarmaco is making headway against DMD as it announced positive results from a Phase III study, Clover’s homologous booster against Omicron increases antibodies, and much more. Italfarmaco Group , based in Milan, Italy, is making headway against Duchenne muscular dystrophy (DMD). The company announced positive results from the Phase III EPIDYS study assessing Givinostat, a histone deacetylase (HDAC) inhibitor that received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration. The study met its primary endpoint, a positive change from baseline in the time to climb four stairs at 18 months compared to placebo. The results demonstrated a slower decline in performing this functional task in the Givinostat-treated group. Additionally, the company noted that key secondary endpoints were also met, consistent with the primary endpoint. Givinostat continued to demonstrate a tolerability pro line with previous studies. In all, the study results show that the addition of Givinostat to steroid treatment leads to clinical benefits for DMD patients. Italfarmaco intends to submit the data to the FDA as well as European regulators for potential approval. Additionally, the company will present the results of the Phase III study for publication in a peer-reviewed journal. “These topline Phase III results in DMD add to the growing dataset we have collected over the last years which have shown positive outcomes with our investigational therapeutic candidate, Givinostat, when taken together with steroids in patients with DMD, providing new hope for boys with DMD, their families and the medical community,” Paolo Bettica, chief medical officer of Italfarmaco Group said in a statement. “There is a tremendous unmet medical need for additional drugs to treat this debilitating rare disease and with these positive results, we intend to meet with regulatory agencies to share these findings and discuss a path forward to submit the complete dataset in a marketing application for potential approval.” Elsewhere around the Globe : Clover Biopharmaceuticals: Based in Shanghai, Clover announced new positive clinical data in individuals who have received a third dose of SCB-2019 (CpG 1018/Alum) as a homologous booster against the Omicron variant of COVID-19. The third dose demonstrated a significant, 19-fold increase in neutralizing antibody levels against the Omicron BA.2 variant compared to pre-booster levels among baseline seronegative participants. Immutep Limited: Australia’s Immutep announced new data from second-line non-small cell lung cancer patients in the Phase II TACTI-002 trial will be presented at the IASLC 2022 World Conference on Lung Cancer in August. EnGeneIC: Also based in Australia, EnGeneIC, is conducting studies of its COVID-19 vaccine that it says provides immunity against all known variants. Trials have shown the novel vaccine works by stimulating an entirely different immune pathway from other vaccines, producing “high affinity” antibodies that neutralize all COVID-19 variants, EnGeneIC said. Thirty-two healthy participants received two doses three weeks apart. Of those, 27 have passed the 28-day safety assessment with no side effects. Critically, they all have high-affinity antibodies capable of neutralizing all COVID-19 mutants, including Omicron. Noema Pharma: Switzerland-based Noema’s Investigational New Drug Application for a Phase IIb study of the company’s PDE10A inhibitor gemlapodect has been greenlit by the U.S FDA. The company will evaluate the drug candidate in Childhood Onset Fluency Disorder, also known as stuttering. The Phase IIb study is expected to read out at the end of 2023. Treos Bio Limited: Based in London, Treos Bio announced the dosing of its first patient in the Phase II OBERTO 301 study of PolyPEPI1018, the company’s lead product candidate. In the trial, PolyPEPI1018 is being combined with atezolizumab, Roche’s PD-L1 inhibitor, for the treatment of patients with late-stage microsatellite stable metastatic colorectal cancer. Ixaka Ltd.: Also based in London, Ixaka Ltd. and Germany’s Minaris Regenerative Medicine GmbH have signed an agreement that enables technology transfer and clinical and commercial manufacture of REX-001, Ixaka’s lead autologous multi-cell therapy. REX-001 is being developed for the treatment of chronic limb-threatening ischemia in patients with diabetes. The scope of the manufacturing agreement includes the scale-up and technology transfer with the goal of supplying the Phase II clinical trials and supporting commercial supplies in the EU and later in the United States. Xenikos B.V.: Located in The Netherlands, Xenikos enrolled the first patient in its pivotal Phase III trial, comparing T-Guard with ruxolitinib for the treatment of patients with Grade III or IV steroid-refractory acute graft-versus-host disease following allogeneic hematopoietic stem cell transplant. Xenikos expects the data from this study to support the submission of a Biologics License Application in the U.S. based on Day 28 data from the first 150 patients. Cyxone: Sweden’s Cyxone filed a submission to the National Institute of Pharmacy and Nutrition, the Hungarian Medical Product Agency, for approval for a Phase IIb study assessing drug candidate rabeximod as a potential treatment for rheumatoid arthritis. Applications will follow this first submission to relevant regulatory authorities in other European countries, the company said. Transgene: Based in France, Transgene and Sweden’s BioInvent International AB announced positive safety and progress in the ongoing Phase I/IIa study assessing BT-001 in patients with solid tumors, including melanoma. The initial data generated in Phase I part A demonstrated that BT-001 alone is well tolerated, with first signs of anti-tumor activity in a hard-to-treat population. BT-001 is based on Transgene’s oncolytic vector and is encoding BioInvent’s proprietary anti‑CTLA‑4 antibody. Confo Therapeutics: Belgium’s Confo Therapeutics has been awarded a €1.7 million grant from Flanders Innovation & Entrepreneurship that will support its discovery programs for novel GPCR candidates with its proprietary ConfoBody technology. MRM Health: Also based in Belgium, MRM Health announced it received regulatory approval from the Federal Agency for Medicines and Health Products in Belgium to initiate a Phase II trial with the novel next-generation optimized consortium therapy, MH002, in patients with Pouchitis, inflammation of the surgically constructed pouch following colectomy. SciSparc Ltd.: Israel-based SciSparc announced that it completed the development of its top-tier drug candidate SCI-110, which will be used in its upcoming Phase IIb study. “Our singular objective in developing SCI-110 is to develop a safe and effective treatment for this devastating, unmet medical need as soon as possible; and this milestone brings us one step closer,” SciSparc CEO Oz Adler said.