Acelrx Pharmaceuticals Inc., of Redwood City, Calif., disclosed an agreement to market Dsuvia (sufentanil), a sublingual opioid tablet in a single-dose applicator, within the dental and oral surgery markets in the U.S. exclusively through Zimmer Biomet Inc., of Warsaw, Indiana. The agreement expands the U.S. availability of the non-invasive, sublingual analgesic for use by dental health care professionals in medically supervised settings who currently use injectable opioids for surgical analgesia. The product avoids the high peak plasma levels and short duration of action observed with bolus intravenous administration, the company said.
Amag Pharmaceuticals Inc., of Waltham, Mass., signed an exclusive licensing agreement with Norgine BV, of Amsterdam, to develop and commercialize ciraparantag in Europe, Australia and New Zealand. Ciraparantag is in development for use in patients treated with direct oral anticoagulants and low-molecular-weight heparin when reversal of the anticoagulant effect of those products is needed for emergency surgery, urgent procedures or due to life-threatening or uncontrolled bleeding. The deal brings a $30 million up-front payment to Amag with the potential for $260 million if development and commercial milestones are reached, plus royalties.
Brainstorm Cell Therapeutics Inc., of New York, achieved its first milestone in developing an innovative exosome-based platform technology for the treatment of severe COVID-19 infection. Results from a study in a mouse model of lipopolysaccharide (LPS)-induced ARDS showed that intratracheal administration of Nurown-derived exosomes resulted in a statistically significant improvement in multiple lung parameters. Those included the clinically relevant factors of functional lung recovery, reduction in pro-inflammatory cytokines and attenuation of lung damage, Brainstorm said.
Dynavax Technologies Corp., of Emeryville, Calif., and Medigen Vaccine Biologics Corp., of Taipei, Taiwan, plan to develop an adjuvanted vaccine candidate to protect against COVID-19. The collaboration is evaluating the combination of Medigen’s stable prefusion form of the SARS-CoV2 recombinant spike protein with Dynavax’s adjuvant, CpG 1018, which is in Dynavax’s FDA-approved adult hepatitis B vaccine. Preclinical studies demonstrated the vaccine candidate adjuvanted with CpG 1018 generated strong immune responses in animals, the companies said.
Emmaus Life Sciences Inc., of Torrance, Calif., said it opened a regional office in Dubai to reach clinicians and patients groups at hospitals throughout the Middle East and North Africa (MENA). Those partnerships are intended to accelerate the company’s path to registration and commercialization of sickle cell disease drug Endari (L-glutamine oral powder) in the MENA region.
G1 Therapeutics Inc., of Research Triangle Park, N.C., created a license agreement for lerociclib with Eqrx Inc., of Cambridge, Mass., which gains exclusive rights for lerociclib in the U.S., Europe, Japan and all other global markets, excluding the Asia Pacific region, except Japan. G1 will receive $20 million cash up front and could receive development and commercial milestone payments of up to $290 million, plus tiered royalties ranging from mid-single digits to midteens based on annual net sales of lerociclib, a differentiated oral CDK4/6 inhibitor being developed for use in combination with other targeted therapies in certain breast and lung cancers.
Humanetics Corp., of Minneapolis, said it received funding from the National Institute of Allergy and Infectious Diseases (NIAID), part of the NIH, to conduct a study of BIO-300 in COVID-19 patients. The study will focus on patients treated for severe COVID-19 disease, many of whom were discharged from the hospital. The patients risk progressive complications that could lead to long-term impairment of lung function. BIO-300 is being developed by Humanetics as a medical countermeasure to protect the body from harm caused by ionizing radiation.
Intravacc, of Bilthoven, the Netherlands, and Leiden University said they developed an animal replacement method for vaccine testing. Inactivated model vaccines were treated with an enzyme that plays an important role in the first steps of the immune response. The enzyme, cathepsin S (CS), breaks down vaccines and pathogens into fragments that are recognized by immune cells. The inactivation step with formaldehyde affects the rate of breakdown by CS. It was assumed that the breakdown of inactivated vaccines would be slowed down by CS, but the opposite was the case, researchers said. It is possible to accurately and sensitively measure vaccine inactivation by quantifying the formation of vaccine fragments during breakdown by CS, the researchers added.
Kaleido Biosciences Inc., of Lexington, Mass., said it initiated a controlled clinical study with Massachusetts General Hospital to evaluate Microbiome Metabolic Therapy (MMT) candidate KB-109 added to supportive self-care (SSC) for outpatients with mild to moderate COVID-19. It is the second clinical study Kaleido has initiated in that patient population. The non-IND study aims to include about 50 COVID-19-positive outpatients to be randomized 1-to-1 to either receive SSC (the control group) or SSC plus KB-109 for two weeks and then followed for three weeks. Patients will be evaluated for safety, physiologic effects, and gut microbiota structure and function. MMTs are designed to modulate the metabolic output and profile of the microbiome by driving the function and distribution of the gut’s existing microbes.
A prospective study found potential utility of Haifa, Israel-based Memed Ltd.'s host immune technology for treating severe COVID-19. Initial data from the prospective study showed continuous and rapid measurements of IP-10, a biomarker associated with inflammatory disease including infectious diseases and immune dysfunction, can be a resource for predicting disease severity and progression, monitoring inflammatory status and personalizing treatment strategies for patients with severe COVID-19, Memed said.
Nabriva Therapeutics Inc., of Dublin, reported preclinical results showing the anti-inflammatory activity of lefamulin. In a mouse model of lipopolysaccharide (LPS)-induced lung neutrophilia, lefamulin showed anti-inflammatory properties similar to common corticosteroid dexamethasone. Pretreatment with lefamulin at doses of 35 mg/kg, 70 mg/kg and 140 mg/kg administered subcutaneously 30 minutes before an intranasal LPS administration challenge was associated with almost complete reduction in total cell and neutrophil recruitment to the lungs at four hours post challenge compared with the vehicle control group (no treatment). Lefamulin also demonstrated reductions in total cell and neutrophil counts that were similar to those observed with dexamethasone at all doses tested. Lefamulin, the company’s pleuromutilin antibiotic, is approved by the FDA under the brand name Xenleta for the treatment of community-acquired bacterial pneumonia in adults. Nabriva also said it has been awarded grant funding from the Austrian Research Promotion Agency to support additional in vitro and animal experiments designed to investigate and further characterize the anti-inflammatory and immunomodulatory properties of lefamulin, as well as the company’s library of pleuromutilin compounds.
Noxopharm Ltd., of Sydney, reported preclinical data from two independent research groups confirming that idronoxil, the active ingredient in the company’s immuno-oncology drug candidate, and sphingosine-1-phosphate inhibitor, Veyonda, activate cancer-fighting immune cells and then enable their entry into microtumors. Noxopharm said other preclinical and clinical data, together with the new research data, leads the company to believe it is close to claiming the first drug capable of converting tumors from cold to hot across multiple cancer types in a well-tolerated way.
Onconova Therapeutics Inc., of Newtown, Pa., said preclinical data published in Molecular Cell support rigosertib’s role in modulating RAS cellular signaling. Study authors found that non-clinical-grade rigosertib used in in vitro studies by others contained impurities and degradation products in sufficient quantities to impact cellular function. In contrast, clinical-grade rigosertib, which has been used in all clinical studies to date, is free of that impurity. Using vincristine as a control, the ability of both compounds to bind tubulin was then measured. In vitro assay results indicated that both non-clinical-grade rigosertib and the vincristine control showed tubulin binding activity, while clinical-grade rigosertib did not exhibit detectable tubulin-binding activity. Furthermore, cell lines expressing mutant beta-tubulin remained sensitive to rigosertib.
Ovid Therapeutics Inc., of New York, and the University of Connecticut School of Medicine entered a research collaboration and license agreement to accelerate the development of a next-generation short hairpin RNA (shRNA)-based therapeutic for Angelman syndrome and potentially other indications. Under the terms, Ovid will work with UConn and gain exclusive access to identified genetic sequences for a potential shRNA-based therapeutic. The most common cause of Angelman syndrome is the loss of function of the gene that codes for ubiquitin protein ligase E3A (UBE3A), which plays a critical role in nerve cell communication, resulting in impaired tonic inhibition. An shRNA-based therapeutic may address that underlying genetic cause by reducing the expression of UBE3A-antisense, potentially restoring the function of UBE3A. That approach may be used in combination with OV-101 (gaboxadol), Ovid’s small-molecule delta-selective GABAA receptor agonist, to restore tonic inhibition and address the underlying symptomology of individuals with Angelman syndrome. OV-101 is currently being evaluated in the pivotal phase III NEPTUNE trial in Angelman syndrome, with top-line results expected in the fourth quarter of 2020.
Synaffix BV, of Amsterdam, and ADC Therapeutics SA, of Epalinges, Switzerland, said they expanded their existing collaboration to explore additional applications, including DAR1, of Synaffix’s site-specific conjugation technologies. Under the expanded terms, ADC has been granted nonexclusive rights for two additional programs, which brings the total number of programs using Synaffix’s ADC technologies to five. ADC also gains access to the latest innovative extensions of Synaffix’s Glycoconnect platform, including DAR1 technology that enables stable attachment of just a single drug per antibody. Synaffix is eligible to receive up-front, milestone and royalty payments tied to each program. Further financial details are not disclosed.
The U.K. government increased its COVID-19 investment by £100 million (US$127.45 million) to fund a cell and gene therapy catapult manufacturing innovation center to accelerate mass production of a COVID-19 vaccine. The Braintree, U.K., center will open in December 2021 with capacity to produce millions of doses each month. The initiative upgrades an existing facility, complementing the Vaccines Manufacturing and Innovation Centre under construction in Oxfordshire, U.K.
United Therapeutics Corp., of Silver Spring, Md., said the U.S. Patent and Trademark Office issued a new patent on July 21, relating to pulmonary arterial hypertension drug Tyvaso (treprostinil) inhalation solution, which expires May 14, 2027, and covering a method of administering treprostinil via inhalation, including claims covering the dosing regimen used to administer Tyvaso. United also filed an amended complaint against Liquidia Technologies Inc., of Research Triangle Park, N.C., in its pending patent infringement litigation in the U.S. District Court for the District of Delaware to include a claim for infringement of the new patent. The lawsuit was initiated in June, based on a 505(b)(2) NDA filed by Liquidia seeking FDA approval of LIQ-861, a dry powder inhalation formulation of treprostinil.
X-Chem Inc., of Waltham, Mass., said it met a key development milestone in its collaboration with Orexia Ltd., of London, a company focused on developing orexin positive modulators using structure-based drug design capabilities. After reaching that development milestone, Orexia exercised an option to exclusively license the orexin receptor type 2 (OX2R) program from X-Chem, which comprises multiple novel OX2R-specific small-molecule orexin positive modulators. Orexia will develop those molecules and is solely responsible for conducting clinical trials with drug candidates derived from licensed compounds, and retains the exclusive global rights to commercialize any resulting products. X-Chem received a license payment and is eligible for future payments based on the achievement of specified research and development milestones, as well as royalties on the sale of approved products.