Aeterna Zentaris Inc., of Charleston, S.C., said it settled a class-action lawsuit pending in the U.S. District Court for the District of New Jersey that alleged the company and certain current and former officers and directors violated the Securities Exchange Act of 1934 in connection with public statements regarding the safety and efficacy of the adult growth hormone deficiency therapy Macrilen (macimorelin) and the prospects for FDA approval of the NDA. The settlement payment will be funded entirely by the company’s insurers, subject to execution of final documents and approval by the district court.
Aldeyra Therapeutics Inc., of Lexington, Mass., said it was prioritizing late-stage ocular disease programs in allergic conjunctivitis, dry eye disease and proliferative vitreoretinopathy while placing on hold development of topical dermal reproxalap, an acetaldehyde dehydrogenase inhibitor, to treat ichthyosis associated with Sjogren-Larsson syndrome and ADX-1612 (ganetespib), an Hsp90 inhibitor, JAK2/STAT inhibitor and apoptosis stimulator, to treat post-transplant lymphoproliferative disorder. The initiatives are expected to extend the company’s cash runway through year-end 2021.
Chembio Diagnostics Inc., of Hauppauge, N.Y., said it formed a global strategic partnership with Lumiradx Ltd., of London, to develop point-of-care diagnostic tests on the platforms of both companies to detect the COVID-19 virus and IgM and IgG antibodies. Financial terms were not disclosed.
Clearside Biomedical Inc., of Alpharetta, Ga., said Arctic Vision Shanghai Biotechnology Co. Ltd., of Shanghai, acquired an exclusive license to commercialize and develop Xipere (triamcinolone acetonide) in China, Hong Kong, Macau, Taiwan and South Korea. Clearside is set to receive up to $35.5 million in development and sales milestones, including $4 million up front and additional payments for achieving specified events prior to and including receipt of FDA approval of Xipere. Clearside also will be entitled to tiered royalties of 10% to 12% based on annual net sales of Xipere in greater China and South Korea. The suspension formulation of the corticosteroid triamcinolone acetonide for suprachoroidal administration with Clearside’s SCS microinjector is in development as a targeted treatment of macular edema associated with uveitis. Arctic Vision also gained the right to pursue development and commercialization of Xipere for additional ophthalmic indications in its licensed territory, with Clearside’s consent.
Dendreon Pharmaceuticals LLC, of Seal Beach, Calif., reported findings from a subanalysis of its Proceed registry comparing overall survival (OS) in African American (AA) and Caucasian (CAU) men with metastatic castrate-resistant prostate cancer (mCRPC) who were treated with Provenge (sipuleucel-T) in a real-world setting. When comparing PSA-matched AA (n=107) to CAU (n=222) men with a baseline PSA less than or equal to the median (29.48 ng/mL), AA men in the registry showed a median OS of 54.3 months vs. 33.4 months for CAU men, or an improvement of 20.9 months and a 48% relative risk reduction in death. The Proceed registry enrolled nearly 2,000 patients with mCRPC who received Provenge between 2011 and 2014 in treatment settings and followed them for three years. The data were published online in Prostate Cancer and Prostatic Diseases.
Denovo Biopharma LLC, of San Diego, said it discovered a genetic biomarker associated with response to the treatment of depression seen in DB-104. Denovo licensed the triple reuptake inhibitor, which targets transporters for dopamine, serotonin and norepinephrine, from Albany, N.Y.-based Molecular Research Inc. following its return from Bristol Myers Squibb Co., of New York, which had conducted two phase IIb trials in treatment-resistant depression.
Edesa Biotech Inc., of Toronto, said it formed a collaborative research project with the National Research Council of Canada (NRC) to develop immunotherapies for vitiligo and other indications. Under the agreement, NRC scientists will produce multiple monoclonal antibodies for Edesa to identify a lead candidate to take into IND-enabling studies. The NRC agreed to grant Edesa an exclusive worldwide license for antibodies arising from the project. Edesa expects to complete the project’s initial phase by year-end 2020. Financial terms were not disclosed.
Emergent Biosolutions Inc., of Gaithersburg, Md., said it initiated development of candidates to treat and prevent COVID-19, using its hyperimmune platforms. COVID-HIG, manufactured from human plasma with antibodies to SARS-CoV-2, will be developed as a potential treatment for severe hospitalized patients and to protect at-risk individuals. In parallel, COVID-EIG, manufactured from plasma of immunized horses with antibodies to SARS-CoV-2, will be developed as a potential treatment for severe hospitalized patients. Emergent plans to use its existing infrastructure and capabilities it deployed for a recently completed phase II trial of influenza hyperimmune FLU-IGIV to treat individuals hospitalized with influenza A.
Flagship Pioneering, of Cambridge, Mass., said it launched Repertoire Immune Medicines, created by the combination of two existing Flagship companies – Cogen Immune Medicines and Torque Therapeutics. The new company has the capability to decipher human subject-derived antigen T-cell receptor (TCR) codes from healthy or diseased tissues. Those complexes dictate T-cell activation or exhaustion, and their immunological codes can be used to design and clinically test a multitude of unprecedented therapeutic products based on precedented and specific mechanisms of T-cell killing of antigen-presenting tumor cells or infected cells, the company said. Repertoire has developed a suite of technologies that allows characterization of the immune synapse. By coupling single cell technologies with cellular and acellular antigen libraries, the company decodes CD4+ and CD8+ TCR-antigen specificity across selected T-cell subsets from patients and from healthy individuals. Repertoire scientists created and developed technologies for the Decode discovery and Deploy product platforms that allow characterization of the immune synapse and the ability to rationally design, and clinically develop, multiclonal immune medicines. The company is engaged in its first dose-escalation safety trial with an autologous T-cell product, TRQ15-01, which leverages its technology platforms to prepare the patient’s T cells and link them to an IL-15 nanogel immune modulator. The CEO of Repertoire is John Cox, who most recently led the spin-off of Bioverativ from Cambridge, Mass.-based Biogen Inc., and its growth and subsequent acquisition by Paris-based Sanofi SA.
Hepion Pharmaceuticals Inc., of Edison, N.J., said a translational research study investigating the effects of its drug candidate, CRV-431, on lung tissue obtained from a patient with idiopathic pulmonary fibrosis (IBF) reported positive findings. In the study, conducted by Fibrofind Ltd., diseased lung tissue was obtained from an IPF patient undergoing a lung transplant and maintained for six days ex vivo. CRV-431 was administered to lung tissue samples at concentrations of 1 µM or 5 µM on the final four days of culture. Additional lung samples were treated with Esbriet (pirfenidone, Roche Holding AG) or Ofev (nintedanib, Boehringer Ingelheim GmgH). Drug effects were evaluated by daily measurement of secreted markers of fibrosis and inflammation. CRV-431 at 5 µM decreased gene expression by an average of 45% (range 20%-84% reduction), which was similar to the effects observed with pirfenidone (46% average reduction) dosed at concentrations 500 times greater. The daily average decrease in secreted disease markers produced by 5 µM CRV-431 was 28% (range 5%-61%). The most potent secretion effect of 5 µM CRV-431 was a 61% reduction in the daily average production of the IPF biomarker, MMP7, which was twice the magnitude of nintedanib (31% reduction) and similar to pirfenidone (65% reduction).
Immunoprecise Antibodies Ltd., of Victoria, British Columbia, a therapeutic antibody discovery CRO, said its approach utilizing polytope monoclonal antibody therapy, from its discovery platforms and artificial intelligence capabilities with partner, Evqlv Inc., is being used in the development of a universal COVID-19 therapy.
Innovation Pharmaceuticals Inc., of Wakefield, Mass., said research procedures that one of the 12 U.S. regional biocontainment labs (RBLs) will follow when testing brilacidin, the company's lead defensin mimetic drug candidate, will be against SARS-CoV-2, the coronavirus responsible for COVID-19. Staff scientists at the RBL will evaluate brilacidin's potential inhibitory efficacy against SARS-CoV-2 in lung epithelial cell lines, as well as its potential inhibitory effect in viral replication of the alphavirus (with Venezuelan equine encephalitis virus as a prototype) in different cell lines. If brilacidin shows promise, testing will be expanded to include additional mechanism-of-action studies to determine brilacidin's specific inhibitory activity across the lifecycle of the SARS-CoV-2 virus – viral entry, viral RNA and viral release.
Mallinckrodt plc, of Staines-upon-Thames, U.K., said it is currently evaluating the limited published evidence suggesting a potential role for inhaled nitric oxide (iNO) as a supportive measure in treating those patients infected with coronavirus (SARS-CoV-2) and having associated pulmonary complications. The company indicated it has engaged with the FDA, the NIH and the Biomedical Advanced Research and Development Authority; however, the safety and efficacy of iNO for pulmonary complications associated with coronavirus have not been established. The company markets iNO as Inomax (nitric oxide) gas for inhalation in the U.S. for the treatment of term and near-term neonates with hypoxic respiratory failure associated with pulmonary hypertension. While nitric oxide has not been specifically studied or used to treat COVID-19, in vitro it has demonstrated an inhibitory effect on the replication cycle of severe acute respiratory syndrome-related coronavirus (SARS-CoV).
Medicago Inc., of Quebec City, said it has produced a virus-like particle (VLP) of the coronavirus just 20 days after obtaining the SARS-CoV-2 gene. Production of the VLP is the first step in developing a vaccine for COVID-19, which will now undergo preclinical testing for safety and efficacy. Once that is completed, the company expects to discuss with the health agencies for the start of human trials by July or August. The company is also using its technology platform to develop antibodies against SARS-CoV-2 in collaboration with the Laval University’s Infectious Disease Research Centre headed by Gary Kobinger, who helped develop a vaccine and treatment for Ebola. Those SARS-CoV-2 antibodies could potentially be used to treat people infected by the virus.
Novocure Ltd., of St. Helier, Jersey, said preclinical data on its tumor treating fields (TTF) in combination with anti-PD-1 therapy were published in Cancer Immunology, Immunotherapy. The study suggests that TTF therapy can induce anticancer immune response and provide the first evidence for the immunostimulatory effects of tumor TTF-induced cell death. Specifically, the study evaluated whether TTF-mediated cell death can elicit antitumor immunity. The data demonstrated that damage-associated molecular patterns were released in TTF-treated cancer cells, and calreticulin was exposed on the cell surface. TTF also promoted the engulfment of cancer cells by dendritic cells and dendritic cell maturation in vitro, as well as recruitment of immune cells in vivo.
Olix Pharmaceuticals Inc., of Suwon, South Korea, said preclinical data published in Molecular Pharmaceutics indicated that L-type calcium channel blockers (CCBs) might have potential in augmenting RNAi therapeutics. Findings showed L-type CCBs increased the endocytic cellular uptake of cell-penetrating asiRNA (cp-asiRNA), and the small molecules substantially improved the potency of cp-asiRNAs, not only in vitro but also in vivo on rat skin.
PDL Biopharma Inc., of Incline Village, Nev., discussed as part of its earnings report the ongoing plan to dissolve the company. PDL has taken steps to monetize the assets of the company and distribute net proceeds to its stockholders in the form of share repurchases, cash dividends or other distributions. The company has engaged financial advisors to evaluate the sale of the entire company, or the sale or distribution of its holdings of San Diego-based Evofem Biosciences Inc. common stock, the portfolio of royalty assets and the company's Noden and LENSAR subsidiaries.
Pluristem Therapeutics Inc., of Haifa, Israel, signed a collaborative agreement with the BIH Center for Regenerative Therapy and the Berlin Center for Advanced Therapies at Charite’ University of Medicine Berlin to expand its existing framework and research agreement and conduct a joint project evaluating the therapeutic effects of Pluristem’s patented PLX cell product candidates for potential treatment of the respiratory and inflammatory complications associated with the COVID-19 coronavirus. PLX cells are allogeneic mesenchymal-like cells that have immunomodulatory properties that induce the immune system’s natural regulatory T cells and M2 macrophages, and thus may prevent or reverse the dangerous overactivation of the immune system. The collaboration with Charité researchers will allow the firm to expedite its program to potentially enable the use of PLX cells to treat patients infected with COVID-19 that have respiratory and immunological complications, Pluristem said.
Predictive Oncology Inc., of Minneapolis, said it will launch a new artificial intelligence platform for vaccine and drug development targeting coronaviruses and acute respiratory syndromes through an operating agreement with Soluble Therapeutics Inc., of Birmingham, Ala. The company has also signed a letter of intent with Inventabiotech Inc., of San Antonio, Texas, to acquire Soluble, its assets and its HSCTM technology, a high-throughput second viral coefficient determination system. Using Soluble’s HSCTM technology and its six machines should allow the running of more than 12,000 computer simulations per machine to help generate new diagnostics, vaccines and therapeutics. Soluble’s HSCTM technology has demonstrated efficacy with both new and existing vaccine and drug programs, Predictive noted.
Rubius Therapeutics Inc., of Cambridge, Mass., disclosed in its full-year 2019 financial results its plan to focus on development of its oncology and autoimmunity pipeline going forward. As a result, the company said it is deprioritizing its RTX-134 program for phenylketonuria (PKU) and other rare disease programs, citing unanticipated delays primarily due to continued manufacturing challenges at the company’s CMO and anticipated high cost associated with producing chronic, high-dose therapy for enzyme deficiencies. The current phase Ib trial in PKU will be discontinued. Rubius said future capital investments and improvements in manufacturing efficiency, together with enhancements to the off-the-shelf, allogeneic cell therapy-focused Red Platform, may enable the firm to revisit chronic, high dose-dependent conditions in the future. As of Dec. 31, the company had cash, equivalents and investments totaling $283.3 million. Shares of Rubius (NASDAQ:RUBY) fell $2.56, or 39%, to close March 12 at $3.99.
New data from Sareum Holdings plc, of Cambridge, U.K., describes the anticancer effect of the checkpoint kinase 1 (CHK1) inhibitor SRA-737 in multiple human lung and colorectal cancer cells, when used in combination with small molecules blocking function of a family of proteins involved in DNA replication and repair. The study establishes the basis for doubling up on drugs targeting systems for DNA repair, and shows the approach could be effective against cancers that do not respond to single-drug treatment. The data also show ways to choose patients that could benefit from existing CHK1 inhibitors such as SRA-737.
Sutro Biopharma Inc., of South San Francisco, said Merck & Co. Inc., of Kenilworth, N.J., extended the research term of the collaboration's first cytokine-derivative program by one year, which includes a payment to Sutro of an undisclosed amount. Sutro's collaboration with Merck was made public in July 2018 to develop therapeutics for cancer and autoimmune disorders. The advancement of a cytokine-derivative product candidate toward IND-enabling studies marks another important step forward, Sutro said.
J.S. Cole and Associates will be the exclusive financial advisor for Austin, Texas-based TFF Pharmaceuticals Inc.’s partnering efforts in Japan. TFF’s platform is designed to improve solubility and absorption of poorly water-soluble drugs. The company’s two lead candidates are voriconazole and tacrolimus inhalation powders. It plans to increase its pipeline by collaborating with large pharmaceutical partners. J.S. Cole provides business development and marketing research guidance.
Tyligand Bioscience Ltd., of Shanghai, and Context Therapeutics LLC, of Philadelphia, are collaborating to develop, manufacture, register and commercialize onapristone extended release. Tyligand will be solely responsible for designing and optimizing a manufacturing process to meet Context’s development and commercialization needs, and standards for quality, safety and cost. Upon completion of performance-based milestones, Tyligand will be granted the exclusive right and will be solely responsible for developing and commercializing onapristone extended release in China, Hong Kong and Macau. Context will be eligible to receive royalties on net sales in Macau. Context will retain rest of world commercialization rights. Onapristone extended release is being evaluated in patients with progesterone receptor-positive ovarian and endometrial cancers in the phase II ONWARD 220 trial.
Vir Biotechnology Inc., of San Francisco, said it plans to collaborate with Cambridge, Mass.-based Biogen Inc. to develop and manufacture human monoclonal antibodies to treat COVID-19 patients. Subject to a final agreement, Biogen would continue cell line development, process development and clinical manufacturing to advance development of Vir’s antibodies. Vir has identified a number of monoclonal antibodies that bind to SARS-CoV-2 that were isolated from individuals who had survived a SARS infection. The company is conducting research to determine if its antibodies, or additional antibodies that it may be able to identify, can be effective as treatment and/or prophylaxis against SARS-CoV-2.
Zelluna Immunotherapy A/S, of Oslo, Norway, and Sweden’s Karolinska Institute will collaborate to develop T-cell receptor natural killer products to treat solid cancers. The collaboration will support a multiyear research program. Zelluna will have exclusive rights to develop and commercialize products resulting from the collaboration through a commercial license with the institute.