Aerpio Pharmaceuticals Inc., of Cincinnati, is reviewing its strategic alternatives following disappointing phase IIb data for AKB-9778 in moderate to severe nonproliferative diabetic retinopathy earlier this year. The company is considering an acquisition, company sale, merger, business combination, asset sale, in-license, out-license or other strategic transaction. Aerpio ended June with $48.2 million in cash resources. Shares of Aerpio (NASDAQ:ARPO) closed up 6.8 cents, or 14.7%, to 53 cents on Monday. (See BioWorld, March 19, 2019.)

Alkermes plc, of Dublin, is collaborating with Fred Hutchinson Cancer Research Center to run the phase II ION-01 study testing ALKS-4230, a fusion protein targeting the intermediate-affinity interleukin-2 receptor complex, with anti-PD-1 Keytruda (pembrolizumab, Merck & Co. Inc.) in patients with advanced or recurrent head and neck squamous cell cancer who did not achieve complete remission with an anti-PD-(L)1 antibody treatment. The primary endpoint of the study is response rate. Duration of response, progression-free survival, time to progression and overall survival will be measured as secondary endpoints in the study, which will start in the fourth quarter of 2019.

Amygdala Neurosciences Inc., of San Francisco, was awarded a $1.35 million grant from the NIH's National Institute on Alcohol Abuse and Alcoholism to test the toxicity of its aldehyde dehydrogenase 2 inhibitor, ANS-6637, in rats and monkeys. Current toxicology data support three-month dosing, while the new studies will support dosing of six months or longer. ANS-6637 is currently in phase II development for alcohol use disorder, and Amygdala has plans to start phase II studies in opioid use disorder and smoking cessation.

Applied Genetic Technologies Corp., of Gainesville, Fla., will present data on its adeno-associated virus (AAV) vectors for ocular gene therapy at the European Society of Gene and Cell Therapy in Barcelona, Spain. In nonhuman primates, the presence of high serum anti-AAV antibodies didn't impact the efficiency of AAV transduction in the retina. The presence of preexisting anti-AAV antibodies also didn't increase inflammation.

Armata Pharmaceuticals Inc., of Marina Del Rey, Calif., received a $1.3 million research and development tax incentive cash rebate from the Australian Tax Office for its R&D spending in Australia in 2018.

Arqule Inc., of Burlington, Mass., reported preclinical data for its pan-AKT inhibitor, miransertib (ARQ-092), at the 2019 American Society of Human Genetics Annual Meeting in Houston. In a mouse model of PIK3CA-driven vascular malformations (VMs), miransertib prevented the formation of VMs by suppressing vascular growth and endothelial cell proliferation. There was also regression of VMs that had already formed. Miransertib is currently being tested in the registrational MOSAIC study in patients with Proteus syndrome and PIK3CA-related overgrowth spectrum disorders.

Crispr Therapeutics AG, of Zug, Switzerland, and Bayer AG, of Leverkusen, Germany, plan to have their joint venture, Cambridge, Mass.-based Casebia Therapeutics Inc., operate under Crispr Therapeutics with Bayer having opt-in rights for two undisclosed products at IND submission. The companies expect the transaction will close in the fourth quarter of 2019, although it's still subject to negotiation and execution of definitive agreements. (See BioWorld, Jan. 26, 2017.)

Foamix Pharmaceuticals Ltd., of Rehovot, Israel, and Leo Pharma A/S, of Ballerup, Denmark, settled their patent litigation with an affiliate of Teva Pharmaceuticals Industries Ltd., of Jerusalem, over Finacea (azelaic acid) foam for the treatment of rosacea. Details of the settlement agreement are confidential.

Homology Medicines Inc., of Bedford, Mass., reported preclinical data for its gene therapy programs for the treatment of metachromatic leukodystrophy (MLD) and phenylketonuria (PKU) at the American Society of Human Genetics 2019 Meeting in Houston. In a murine model of MLD, a single administration of HMI-202 produced therapeutic levels of human ARSA enzyme. At 48 weeks, the treatment modulated LAMP-1, myelin and lymphocyte protein transcript and sulfatides in central nervous system tissue. In nonhuman primates, ARSA protein levels confirmed crossing of the blood-brain barrier and blood-nerve barrier. In the Pah-enu2 murine model of PKU, a single administration of HMI-102 normalized Phe levels, which were sustained for 48 weeks. Downstream biochemical metabolites, including tyrosine, melanin and 5-HIAA, were increased. Weight-based dosing from murine model to the nonhuman primate model was confirmed. A five-year retrospective study of PKU patients at two specialized U.S. clinics showed Phe concentrations remained above the therapeutic threshold while treated with the current standard of care.

Immunicum AB, of Stockholm, reported preclinical data for ilixadencel, an off-the-shelf, cell-based immune primer. In the CT26 tumor model in mice, 60% of 10 mice treated with mouse-ilixadencel and anti-CTLA4 antibodies were alive at the end of the study, including two with a complete response and three which had almost reached a complete response. Of the 10 mice treated with anti-CTLA4 and anti-PD-1 antibodies, 20% were alive, including one which had almost reached complete response.

Moberg Pharma AB, of Stockholm, granted Seoul-based Dongkoo Bio & Pharma Co. Ltd. exclusive rights to market, distribute and sell MOB-015 in South Korea. Moberg continues its production and supply duties. MOB-015, a topical formulation of terbinafine, is designed to treat onychomycosis, a fungal infection of the toe or fingernail. No financial terms were disclosed.

Ph Pharma Co. Ltd., of Seoul, South Korea, and Immunome Inc., of Exton, Pa., are collaborating to discover antibody-drug conjugates (ADCs) against multiple oncology targets. Immunome will be responsible for antibody discovery and Ph Pharma will conjugate its ADC payloads and test the ADC candidates. Ph Pharma will have the right to develop and commercialize the first development candidate from the collaboration; a selection process will determine rights to subsequent candidates. The company that selects the candidate will pay the other company development, regulatory and commercial milestones of up to $100 million per product, as well as royalties. The companies will share revenue from sublicensing to third parties.

Progenics Pharmaceuticals Inc., of New York, sent a letter to shareholders urging them to vote against a consent solicitation by Velan Capital LP, of Alpharetta, Ga., which owns 11.7% of Progenics. The consent solicitation seeks to remove three current members of Progenics' board and elect five director nominees supported by Velan.

Seelos Therapeutics Inc. and Vyera Pharmaceuticals LLC, both of New York, have agreed to not conduct a phase III trial of SLS-002, an intranasal racemic ketamine for patients with suicidality in post-traumatic stress disorder and major depressive disorder. Instead, the companies will convert the one-time cash payment obligation owed to Vyera to a series of cash and stock payments over the next nine months. This amending of a previous agreement follows feedback from an FDA type C meeting, as Seelos now plans to conduct a phase II proof-of-concept trial in suicidality in major depressive disorder.

Tetra Bio-Pharma Inc., of Ottawa, Ontario, a cannabinoid-focused company, said it is preparing to begin the treatment portion of its phase III trial of PPP-011, an inhaled, synthetic THC/CBD, and to advance its program for PPP-003, a cannabinoid formulation to treat eye conditions such as painful dry eye and uveitis. PPP-011 is being evaluated for improving the quality of life in patients with advanced cancer and uncontrolled symptoms. The company anticipates approval of the candidate in advanced cancer pain by late 2020. A launch of phase I and II studies in uveitis and painful dry eye is planned for early 2020.

Tiaki Therapeutics Inc., of Cambridge, Mass., reported preclinical data at Neuroscience 2019 in Chicago, describing its approach to targeting microglia, the immune cells of the brain, to address inflammatory-driven CNS disorders. Using the company's systems biology platform, Tiaki scientists have identified targets that mitigate the neuroinflammatory transcriptomic signature observed in patients with neurodegenerative disease. That neuroinflammatory signature was validated by comparing transcriptomes from either the whole slice (all CNS cell types) or isolated microglia to transcriptomes from patients with Alzheimer's disease. Bioinformatics analysis demonstrated a significant correlation between the gene expression signature observed in the adult ex vivo brain slice assay and gene expression data from Alzheimer's disease patients.

Symdeko (tezacaftor/ivacaftor and ivacaftor), from Vertex Pharmaceuticals Inc., of London, is now reimbursed to Australian patients with cystic fibrosis (CF), ages 12 and older, who are homozygous for the F508del mutation or who have one copy of the F508del mutation and another responsive residual function mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In addition, Vertex's Orkambi (lumacaftor/ivacaftor) is now also reimbursed for the treatment of children with CF, ages 2 to 5, who have two copies of the F508del mutation in the CFTR gene.