The Allen Institute and Biomarin Pharmaceutical Inc., of San Rafael, Calif., said they formed a collaboration that will use technologies developed at Allen to create gene therapies to treat rare genetic diseases of the central nervous system. The licensed technology was developed by researchers at the Allen Institute for Brain Science with the goal of studying and classifying individual brain cell types by engineering AAVs to carry genes that switch on in specific types of neurons or other cells in the brain. A key component of the engineered AAVs is a molecular "Zip code," or enhancer, that ensures gene expression is restricted to the correct cellular address. Biomarin will receive an exclusive license to each program for research, development and commercialization. Financial terms were not disclosed.
Cannabics Pharmaceuticals Inc., of Tel Aviv, Israel, disclosed plans to develop a melanoma treatment, which produced antitumor activity on melanoma cell lines. Cannabics plans to start in vivo studies in animal models of melanoma.
Dermtech Inc., of La Jolla, Calif., said it will collaborate with researchers from the Stanford University School of Medicine, led by principal investigator Kavita Sarin, on a study of longitudinal non-invasive cytokine monitoring in people with hidradenitis suppurativa, a chronic skin condition characterized by recurrent swollen and painful lesions in the armpit, groin, anal and breast regions. The three-year study will use Dermtech’s non-invasive sample collection and precision genomics platform to identify and evaluate biomarkers over time in people with the condition, potentially leading to improved decisions about treatment.
Eli Lilly and Co., of Indianapolis, said its development program for mirikizumab will focus exclusively on Crohn's disease and the ulcerative colitis, an indication where the company reported in March 2021 that the phase III Lucent-1 study met its primary endpoint of clinical remission and all key secondary endpoints. Although the Oasis program in psoriasis generated positive results, with safety and efficacy similar to other IL-23p19 inhibitors, Lilly said in its quarterly earnings update that it does not plan to submit any applications for mirikizumab in the indication.
Essa Pharmaceuticals Inc., of Houston, said it formed a trial collaboration and supply agreement with Bayer AG, of Leverkusen, Germany, to evaluate Essa’s lead candidate, EPI-7386, an N-terminal domain androgen receptor inhibitor, in combination with Bayer's androgen receptor inhibitor, darolutamide, in people with metastatic castration-resistant prostate cancer. The agreement allows Bayer to sponsor and conduct a phase I/II study, expected to begin in 2021, to evaluate the combination. Essa will supply EPI-7386 for the trial and retain rights to the agent.
Grifols SA, of Barcelona, said it signed an agreement with the government of Andorra to establish a global hub in the tiny country. The facility, the Pyrenees Immunology Research Center, will focus on developing treatments for immune system disorders that can result in diseases including autoimmunity disorders, cancer and emerging infectious diseases. It will also host and sponsor conferences, symposiums and educational programming to promote broader awareness of immunological pathologies. Completion is set for 2023.
Homology Medicines Inc., of Bedford, Mass., said preclinical data demonstrated that its AAVHSCs delivered vectors at a high efficiency to the liver and secreted antibodies throughout the body, resulting in sustained expression levels consistent with C5 antibody therapeutics in a humanized murine model. The proof of concept unlocks potential for a one-time treatment that leverages the liver to produce fully functional antibodies, the company said. The data also demonstrated that its AAVHSCs efficiently transduced the liver and reached relevant tissues, including crossing the blood-brain and peripheral-nerve barriers with one I.V. administration, the company added.
Preclinical data from Neuraly Inc., of Germantown, Md., showed that NLY-01, an engineered exendin-4, glucagon-like peptide-1 receptor (GLP-1R) agonist, can selectively block β-amyloid-induced activation of microglia through up-regulated GLP-1R, inhibit formation of reactive astrocytes and preserve neuronal viability, resulting in improved spatial learning and memory. NLY-01 is entering a phase IIb trial in Alzheimer’s disease while also being evaluated in a phase II Parkinson’s disease trial.
New results of a study of OK-201 from Okyo Pharma Ltd., of London and Boston, showed the non-opioid analgesic candidate, delivered topically in a mouse neuropathic corneal pain model, demonstrated potential to treat acute and chronic ocular pain. The reduced corneal pain response was similar to that of gabapentin, a commonly used oral drug for neuropathic pain, the company added. OK-201 is a lipidated cyclized bovine adrenal medulla analogue.
Pfizer Inc., of New York, said it acquired privately held San Diego-based Amplyx Pharmaceuticals Inc., whose lead compound fosmanogepix, APX-001, is being developed to treat invasive fungal infections. Financial terms were not disclosed. Fosmanogepix is in phase II trials evaluating the safety and efficacy of intravenous and oral formulations to treat life-threatening invasive fungal infections caused by molds, yeasts and rare molds such as Aspergillus spp, Candida spp including Candida auris, Fusarium spp and Scedosporium spp.
Point Biopharma Inc., of Indianapolis, said the U.S. Nuclear Regulatory Commission issued a materials license for a new production facility in Indianapolis. Point is renovating its 80,000-square-foot radiopharmaceutical manufacturing center which, when complete, will make it one of the largest, GMP radioligand manufacturing facilities in the world, the company said. The materials license authorizes handling of nuclear material in chemical and/or physical form, enabling Point to work with a variety of radioisotopes on site plus complete testing and qualification of its operations. The company said it expects the facility will begin to provide supply for its phase III trial targeting metastatic castration-resistant prostate cancer later in 2021.
Silence Therapeutics plc, of London, achieved a research milestone as part of its ongoing RNAi collaboration with Mallinckrodt plc, of Dublin, for complement-mediated diseases, triggering a further $2 million payment to Silence. The milestone relates to preclinical development work on the SLN-500 C3 targeting program, highlighting the successful ongoing collaboration between the two companies. Silence continues to work with Mallinckrodt to progress IND-enabling studies for SLN-501, the first nominated product candidate.
Soligenix Inc., of Princeton, N.J., said preclinical data presented at the annual Conference on Vaccinology Research demonstrated the potency of the Civax COVID-19 vaccine development program in mice and nonhuman primates. Utilizing its heat-stabilization technology, in conjunction with a clinically tested adjuvant (Covaccine HT), Soligenix and its collaborators at the University of Hawaii at Manoa are developing a heat-stable subunit vaccine, targeted to enable ambient shipping and storage.
Triplet Therapeutics Inc., of Cambridge, Mass., disclosed preclinical data in Huntington’s disease (HD) for its first clinical candidate, TTX-3360, including the therapeutic target and route of administration, in a virtual presentation at the CHDI Foundation’s 16th annual HD Therapeutics Conference. TTX-3360, an antisense oligonucleotide, is the first therapeutic candidate with the potential to modify the course of HD and other repeat expansion disorders by targeting the DNA damage response pathway.
Twist Bioscience Corp., of South San Francisco, and Vivlion GmbH, of Frankfurt, Germany, disclosed a collaboration for the generation of gRNA libraries for CRISPR applications. Vivlion will purchase Twist Oligo Pools to generate and sell precision CRISPR libraries for functional genomics research. Vivlion’s 3Cs technology bypasses standard cloning methods such as PCR amplification, allowing direct conversion of Twist’s Oligo Pools into gRNA libraries that maintain Twist’s uniformity.
VBI Vaccines Inc., of Cambridge, Mass., said preclinical data of its enveloped virus-like particle vaccine candidate, VBI-2902, has been submitted for peer-review to a scientific journal and will be available on the online preprint server bioRxiv. The data demonstrate that the candidate expressing a modified, prefusion form of the SARS-CoV-2 spike protein elicited a highly potent and focused neutralizing antibody response, and conferred protective benefit in Syrian golden hamsters challenged with COVID-19, assessed in terms of clinical disease (loss of body weight) and lung inflammation.
Vifor Pharma Group, of St. Gallen, Switzerland, said the first patient has been enrolled in the large scale registry CARE-HK in HF (cardiovascular and renal treatment in heart failure [HF] patients with or at high risk for hyperkalemia [HK]). The non-interventional clinical study aims to better understand renin-angiotensin-aldosterone system inhibitors treatment decisions in clinical practice, potential barriers to achieving optimal guideline-directed care in HF patients with or at high risk for HK, and to assess how Vifor’s Veltassa (patiromer) may be used in the management of this patient population.