A two-drug immunotherapy combination evaluated at Roswell Park Comprehensive Cancer Center for treating cancers is expected to become available to cancer patients with COVID-19 through a clinical trial at Roswell Park. The FDA authorized clinical researchers at the center to conduct a study assessing the safety and effectiveness of giving both rintatolimod (branded Ampligen and manufactured by AIM Immunotech Inc.) and interferon alfa. According to researchers, patients with cancer and COVID-19 have a risk of severe illness up to five times higher than people without cancer. The planned phase I/IIb trial is set to enroll about 40 patients in two stages. Phase I will see 12 to 24 patients receiving both rintatolimod and interferon alfa-2b at escalating doses. Once that initial phase is complete, further study participants will be randomized to two arms: one receiving the two-drug combination and a control group who will not receive rintatolimod or interferon alfa but will receive best available care. The study will evaluate the extent to which the therapy will promote clearance of the SARS-CoV-2 virus from the upper airway.
Aerpio Pharmaceuticals Inc., of Cincinnati, restructured a licensing deal with San Diego-based Gossamer Bio Inc. for its HIF-1 alpha stabilizer, GB-004. Terms of the amended agreement include a $15 million immediate payment to Aerpio and $90 million in milestone payments related to regulatory approvals and commercial sales. Aerpio is also eligible to receive tiered royalties on sales of licensed products at percentages ranging from the low to mid-single digits. GB-004 is an oral, gut-targeted HIF-1 alpha stabilizer that has been shown to improve disease indices in multiple models of inflammatory bowel disease, the company said. Aerpio made news in 2018 via the $420 million deal with Gossamer to pursue mucosal healing in inflammatory bowel disease and to use Aerpio's hypoxia induced factor-1 alpha stabilizer, once-daily GB-004, and related compounds to bring about longer-term remissions. The arrangement was made with GB004 Inc., a wholly owned subsidiary of Gossamer, with the firm collecting $20 million up front, with as much as $400 million more to come if development, regulatory and sales goals are met.
Beam Therapeutics Inc., of Cambridge, Mass., reported data during the virtual American Society of Gene and Cell Therapy meeting from its direct editing program for alpha-1 antitrypsin deficiency, showing that using Beam's adenine base editors (ABEs) to directly correct the PiZ mutation resulted in an average of 16.9% correction of beneficial alleles at seven days and 28.8% at three months. That significant increase over the period suggests that corrected hepatocytes may have a proliferative advantage relative to uncorrected cells. In addition, treated mice demonstrating decreased alpha-1 antitrypsin (A1AT) globule burden within the liver and a durable increase in serum A1AT, roughly 4.9-fold higher than in controls, was observed at three months. In vivo data were reported from the company’s HPFH sickle cell disease program, which aims to recreate naturally occurring single-base changes in the gamma globin gene promoters (HBG1 and HBG2) that disrupt repressor binding and lead to increased expression of gamma globin, which is half of the fetal hemoglobin (HbF) tetramer. Beam observed more than 90% editing and a more than 65% increase in gamma globin levels compared to less than 1.5% in unedited cells in mice at 16 weeks post-transplant. Beam was able to replicate those findings with a second donor at 18 weeks post-transplant. In addition, the data showed successful editing of CD34+ cells from a homozygous sickle patient, demonstrating greater than 60% increase in gamma globin levels with a concomitant decrease to less than 40% in sickle beta globin levels in vitro. The data demonstrate that ex vivo delivery of ABEs achieved precise editing, resulting in long-term engraftment and therapeutically relevant increases in target gene expression.
Boehringer Ingelheim GmbH, of Ingelheim, Germany, and CDR-Life AG, of Schlieren-Zurich, Switzerland, entered a collaboration and licensing agreement to research and develop antibody fragment-based therapeutics for geographic atrophy, a progressive, irreversible retinal disease that occurs in patients with age-related macular degeneration. The two will use antibody fragment-based technology, which retains the specificity of an antibody while significantly reducing the size of the molecule, and apply it to the eye via intravitreal injection. Under the terms, Boehringer will receive an exclusive, worldwide license to develop certain compounds based on CDR-Life´s technology against a specific target and will be responsible for global development and commercialization. CDR-Life will be eligible to receive up to CHF474.5 million (US$488 million) in up-front and success-based milestone payments, as well as research funding and royalties on sales.
Cipla Ltd., of Mumbai, India, said it signed a nonexclusive licensing agreement with Gilead Sciences Inc., of Foster City, Calif., to manufacture and distribute remdesivir, which has an emergency use authorization from the FDA to treat COVID-19. Cipla can manufacture and market the product in 127 countries, including India and South Africa, under Cipla's brand name.
New data from Citius Pharmaceuticals Inc., of Cranford, N.J., show that its mesenchymal stem cells (MSC) secrete higher levels of anti-inflammatory proteins compared to MSCs derived from bone marrow. Comparative secretome analysis showed overexpression of multiple neuroprotective and anti-inflammatory factors, including CXCL1, VEGF-A and CXCL5, the company said. Earlier this year, Citius signed an exclusive option agreement to in-license NC-MSCs for acute respiratory distress syndrome, including in COVID-19 patients, from Novellus Inc., of Cambridge, Mass.
Cmab Biopharma Inc., of Suzhou, China, and Qurebio Ltd., of Shanghai, agreed to develop and manufacture Qurebio's bispecific antibody, Q-1802, which is being studied for treating solid tumors such as gastric cancer. The filing package and drug product conforms to requirements necessary to submit INDs in both China and the U.S., the companies said.
Creoptix AG, of Zurich, Switzerland, said it will collaborate with Adriano Aguzzi, professor for neuropathology at the University of Zurich, to characterize immunological responses to SARS-CoV-2 such as IgA, IgM and IgG antibodies, which signal whether an individual has been exposed to the virus. To identify blood samples, the team will use a bioanalytical instrument that enables antibody detection in even pure serum or plasma.
Privately held Cytoagents Inc., of Pittsburgh, said it received a $1.6 million grant from the NIH to develop its lead drug candidate, GP-1681, for treating severe influenza and COVID-19 by targeting hypercytokinemia, more commonly known as cytokine storm. GP-1681 is a small-molecule inhibitor of cytokine release in activated human immune cells that addresses an unmet need for therapeutic treatment. It has been shown to safely modulate the natural immune response by tamping down various cytokines and addresses the underlying cause of life-threatening symptoms associated with cytokine storm, the company said.
Forward Pharma A/S, of Copenhagen, Denmark, said that due to current precautionary measures amid the pandemic, a European Patent Office hearing that's part of its ongoing efforts to resolve an intellectual property dispute key to it securing royalty payments from Cambridge, Mass.-based Biogen Inc., has been postponed until Feb. 2, 2021.
Genespire Srl, of Milan, and the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) have agreed to jointly study and develop gene therapies that exploit gene editing and lentiviral vector technologies developed by SR-Tiget. Genespire was granted an exclusive global license for the research, development and commercialization of gene therapies for metabolic diseases based on SR-Tiget’s alloantigen-free, microRNA-regulated lentiviral vectors, which the companies said allow for stable liver gene therapy even for diseases with early onset, requiring administration at a young age. Genespire was also granted exclusive licenses and options to the results of a joint research and development program with SR-Tiget in T-cell and hematopoietic stem cells to address genetic diseases, in particular primary immunodeficiencies, it said. Financial terms of the agreement were not disclosed.
Genocea Biosciences Inc., of Cambridge, Mass., said it would file an IND for GEN-011, an investigational T-cell therapy, before the end of the second quarter. Initial clinical data on the tumor-targeted candidate are expected to follow in the first half of 2021.
Hetero Labs Ltd., of Hyderabad, India, said it entered a licensing agreement with Gilead Sciences Inc., of Foster City, Calif., for manufacturing and distribution of remdesivir for the treatment of COVID-19. Under the terms, Hetero will be supplying remdesivir in 127 countries, including India, subject to regulatory approvals in respective countries. Financial terms were not disclosed.
Jaguar Health Inc., of San Francisco, and its wholly owned subsidiary, Napo Pharmaceuticals Inc., jointly entered an accounts receivable purchase agreement with Oasis Capital LLC pursuant to which Oasis agreed to purchase all of the company's accounts receivable related to the April 2020 sales of Mytesi (crofelemer) to Cardinal Health Inc. The April 2020 accounts receivable have a gross value of $2.7 million. Oasis paid about $1 million. Oasis will return to Jaguar within five days any amount that exceeds the sum of the purchase price and a fee of 5.45%.
Legend Biotech Corp., of Somerset, N.J., and Noile-Immune Biotech Inc., of Tokyo, said they entered a license agreement whereby Legend will have the right to develop CAR T and/or TCR T-cell therapies incorporating Noile-Immune’s PRIME (proliferation-inducing and migration-enhancing) technology secreting both IL-7 and CCL19. The PRIME technology is designed to improve proliferation and trafficking into solid tumors of both engineered CAR T and/or TCR T cells, as well as the patient’s own T cells. Under the multiyear collaboration agreement, Legend and Noile-Immune will work together on up to two select cancer targets. Legend will gain the right to incorporate the PRIME technology into its CAR T and/or TCR T-cell programs and will provide to Noile-Immune a total of up to $70 million per selected target for certain development, regulatory and commercial milestone payments. Noile-Immune will also be entitled to receive mid-single-digit royalties on net sales of any resulting products.
Lipocine Inc., of Salt Lake City, said a preclinical study of LPCN-1144, an oral prodrug of bioidentical testosterone, found it improved high fat diet-induced nonalcoholic steatohepatitis features and hepatic fibrosis in a rabbit model of the disease. LPCN-1144 treatment-related reduction in key liver inflammation and fibrosis markers was comparable to that reported for obeticholic acid under a similar preclinical methodology, the company said.
Mind Medicine Inc., of Toronto, said it entered a clinical trial agreement with Maastricht University to undertake a phase IIa trial for lysergic acid diethylamide (LSD) in adults with attention deficit hyperactivity disorder. Mindmed previously established a microdosing division to develop a portfolio of clinical trials studying the use of subperceptual amounts of psychedelic substances for medical purposes. The trial is expected to begin by the end of the year.
Mylan NV, of Hertfordshire, U.K., licensed from Foster City, Calif.-based Gilead Sciences Inc. rights to manufacture and distribute remdesivir in 127 low- and middle-income countries, including India. The agreement is nonexclusive, allowing for multiple licensees to ensure extensive access once the drug is approved, the companies said. Financial terms were not disclosed. Remdesivir is the 10th medicine licensed to Mylan by Gilead.
Opiant Pharmaceuticals Inc., of Santa Monica, Calif., said it now expects to file an NDA for OPNT-003 in early 2021, due to the impact of the COVID-19 pandemic. The company also reported its first quarter earnings, which included revenues of $4.3 million driven by Narcan nasal spray royalties of $4.2 million, an increase of 12% year-over-year. Projected royalties for the full-year 2020 are $26.2 million. The company ended the quarter with cash and cash equivalents of $32.2 million and no debt, it said.
Oxford Biomedica plc, of Oxford, U.K., said its recent U.K. Medicines & Healthcare products Regulatory Agency (MHRA) inspection resulted in the issue of a certificate of GMP compliance for the first two GMP manufacturing suites and supporting area such as warehouse, cold chain facilities and QC laboratories, in its new Oxbox manufacturing facility. The approval by the MHRA of the first two suites enables commercial production of batches to commence for partner programs within the coming weeks.
Passage Bio Inc., of Philadelphia, presented preclinical data at the American Society of Gene and Cell Therapy meeting showing that, in the Twitcher mouse model of Krabbe disease, CSF delivery of AAVhu68 encoding GALC showed substantial increases in GALC enzyme activity, improved myelination of peripheral nerves, improved neuromotor function and increased survival. In the naturally occurring Krabbe canine model, a single ICM injection of AAVhu68 encoding GALC showed normalization of GALC activity, reduction of CSF psychosine levels, normalization of peripheral nerve conduction velocity, improvement in brain myelination, reduction in brain inflammation, phenotypic correction and increased survival. Treatments in both the mouse and canine models were shown to be well-tolerated with no observed toxicities. Passage is developing PBKR-03, an AAVhu68 GALC gene therapy, for infantile Krabbe disease. The company anticipates submitting an IND in the second half of this year.
Point Biopharma Inc., of Toronto, said it selected PSI CRO to manage its phase III study of PNT-2002, a 177Lu-PSMA radiotherapeutic for the treatment of metastatic castrate-resistant prostate cancer.
Simcere Pharmaceutical Group, of Nanjing, China, and Canadian biotech Primary Peptides Inc. disclosed a collaboration and exclusive license agreement to develop and commercialize an innovative product for the treatment of stroke. Under the terms, Simcere receives exclusive rights to develop and commercialize in China, including the mainland, Hong Kong, Macau and Taiwan, while Primary Peptides retains rights in all other markets. Further details and financial terms were not disclosed.
Stoke Therapeutics Inc., of Bedford, Mass., reported preclinical data in a virtual poster at the American Society of Gene and Cell Therapy meeting, which demonstrated in vitro and in vivo target engagement and protein up-regulation in OPA1 protein-deficient cells. OPA1 protein deficiency is the primary cause of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. Results from Stoke’s TANGO antisense oligonucleotides (ASOs) showed dose-dependent decreases in non-productive OPA1 mRNA and increases in OPA1 protein expression. In vivo increases in OPA1 protein levels in the retina of wild-type rabbits were observed and the test ASO was well-tolerated for up to 15 days after intravitreal injection.
The Sabin Vaccine Institute said it received an additional $20 million from the Biomedical Advanced Research and Development Authority (BARDA) to advance development of vaccines against Ebola Sudan and Marburg viruses through phase II trials. In September 2019, BARDA awarded Sabin a $128 million development contract and provided the initial funding award of $20.5 million. The second $20 million award will enable the manufacture and release of clinical vaccine material developed by Reithera Srl, of Rome. The funding will also support nonclinical studies to evaluate efficacy and immune response.