Correct, these papers are not written in layman's terms for investors to understand, they are for scientists and industry..
I suggest that people go back and click on the "Full text" version and reread it a few times, there's a lot that even we can take in..here's a sample...
"DISCUSSION
PKX-001 is an anti-aging glycopeptide. It is a small (544.2 Daltons), synthetic analog of the larger (>2,600 Daltons) family of compounds called Anti-Freeze Glycoproteins (AFGPs). These proteins occur naturally in Arctic and Antarctic fish, and other, cold-climate, dwelling invertebrates to enable cell and tissue function at subzero temperatures (DeVries, 1983, Bang et al 2013). Research in other species have shown much promise for AFGPs in the preservation of biological materials, and organ transplantation (Amir et al, 2005; Matsumoto et al, 2006, Deller 2014). Large-scale research has been limited however due to AFGPs’ costly extraction from un-sustainable sources, instability and large molecular size.
PXK-001 has been investigated via in vitro cell culture and in animal models under a variety of conditions and showed significant cytoprotective properties. In vitro studies in various cell lines has demonstrated significantly increased cell survival when PKX-001 is added to culture media and cells then exposed to various stressors (US Patent Nos. US20090311203). Supplementation with PKX-001 during in vitro culture of isolated human and murine islets has been shown to improve potency of transplanted islets and attenuate long-term tacrolimus-induced graft dysfunction (Gala-Lopez et al, 2016).
Our previous study using ex vivo human retina has also suggested that PKX-001 can help with human cell survival (Yanai et al, 2015). In that study we developed a novel culture system with human retina sitting on a bed of human retinal pigment epithelium derived from human embryonic stem cells, thus, mimicking the subretinal space. PPCs were sandwiched between the neurosensory retinal explant and retinal pigment epithelium. When PPCs were pretreated for 24 hours with PKX-001 (4 mg/mL), an almost 3-fold increase in PPC viability was observed at the end of a 10-day culture period.
Our current study extends this previous ex vivo study. Pre-treating PPCs with PKX-001 before subretinal transplantation results in improvement in PPC survival for up to 6 months. Most significantly though, signs of PPC maturation: objective functional benefits (electrodiagnostics and optical coherence tomography) and immunohistochemistry suggests that PKX-001 significantly improve PPC maturation into functional photoreceptors in a retinal dystrophy animal model. Despite that both S-opsin and L/M opsin were expressed in pre-implantation PPC we only observed their expression in vivo at the 6 month point and only in PPCs pre-treated with PKX-001. In addition, STEM121, a marker of viable human cells was only observed at six months and only in PKX-001 pre-treated PPCs. We interpret this as a sign that on subretinal injection PPC basal metabolism might be significantly suppressed by the hostile host environment and that at the 6 month timepoint, PKX-001 has significantly aided recovery to allow for photoreceptor maturation.
Currently, in vivo studies of PPC transplantation have produced mixed results in animal studies (Pearson RA et al., 2012; Barber et al., 2013; Gonzalez-Cordero A et al., 2013), and very limited benefits in human studies (Jin ZB et al., 2019, Wang Y et al., 2020). There is therefore a pressing need to improve transplantation methodology. The problems can be subdivided into two broad areas, issues of quality of PPCs and the hostile environment of the degenerating retina (Ikelle L et al., 2020). The most successful strategies so far have been improving the quality of PPC manufacture (Hirami Y et al., 2009, Jin ZB et al., 2012, West EL et al., 2012) a focus on immunosuppression (Nazari, H et al., 2015; Zhu J et al., 2017) and improvements in surgical techniques (Wang Y et al., 2020). A number of other strategies are also emerging. Small molecules are in development to overcome cross-species cell contamination. (Osakada F et al., 2009). Also, siRNAs that disrupt outer limiting membrane integrity and chondroitinase that digest chondroitin sulfate proteoglycans deposited as a result of glial scarring have also been used to improve the degenerating retinal environment for PPC transplantation (Barber et al., 2013). PKX-001 now add to these methodologies. Our studies suggest that PKX-001 works by ameliorating the toxicity of degenerating retina through a reduction of prostaglandin E2, a mediator that has been shown to stimulate cell death in several model systems (Takadera et al., 2004; Ricciotti et al., 2011; Miyagishi et al., 2013; Yannai et al., 2015).
In conclusion, in this preliminary in vivo study, PKX-001 significantly improves survival of PPCs in an animal model of inherited retinal disease. This study however was undertaken in a relatively small number of animals and further studies are needed to better quantify the effectiveness of the small molecule. Studies in other animal models are also needed since it has been shown that the genotype of an animal model can significantly influence the success of stem cell integration (Barber et al., 2013). In addition, more studies are needed to determine the precise mechanism of action underpinning the effectiveness of PKX-001. It has been shown that in some cases, functional restoration after transplantation can be attributed to RNA or protein transfer between graft and host photoreceptors instead of transplanted photoreceptors migrating and integrating into the photoreceptor layer of recipients (Santos-Ferreira T et al., 2016, Pearson RA et al., 2016, Nickerson PEB et al., 2018). It needs to be determined if this impacts on the method of action of PKX-001."
