On August 26, 2020 Kinnate Biopharma Inc., a biopharmaceutical company focused on the discovery and development of small molecule kinase inhibitors for difficult-to-treat, genomically-defined cancers, reported that it has raised $98 million in a Series C financing (Press release, Kinnate Biopharma, AUG 26, 2020, View Source [SID1234564036]). The financing was led by RA Capital Management with participation from additional new investors: Viking Global Investors; Venrock Healthcare Capital Partners; Fidelity Management & Research Company, LLC; Janus Henderson Investors; Surveyor Capital (a Citadel company); Boxer Capital of Tavistock Group; Logos Capital; and an investment fund associated with SVB Leerink. Existing investors Foresite Capital, OrbiMed, Nextech Invest, and Vida Ventures also participated in the round. In December 2019, Kinnate closed a $74.5 million Series B financing.

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"In just over two years, Kinnate has built a proprietary pipeline of kinase inhibitors from the ground up, and this significant financing positions us to advance at least one of our lead product candidates into clinical development in the first half of next year," said Nima Farzan, CEO of Kinnate. "We are pleased to expand our syndicate of world-class healthcare investors during this exciting time of growth for the company. Their expertise will be invaluable as we execute on our vision of increasing the availability of targeted therapies for the many cancer patients who currently lack access to effective precision medicines."

Through its deep expertise in structure-based drug discovery, translational research, and patient-driven precision medicine, Kinnate has developed a wholly owned kinase inhibitor portfolio of new targeted therapy candidates that aim to help patients who are non-responders or express resistance to currently available precision medicines. The company’s lead preclinical programs include small molecule inhibitors targeting specific classes of BRAF kinase mutations (Class II and Class III mutations) and FGFR2 or FGFR3 alterations that aim to overcome resistance commonly limiting the efficacy of existing therapies. The company is also advancing a number of other research programs. To help advance these programs, Kinnate is working with leaders at experienced precision medicine cancer centers including Massachusetts General Hospital Cancer Center and the UC San Diego Moores Cancer Center.

"The rapid advancement of genomic technologies has enabled the identification of many more people who may benefit from genomically-targeted therapies. However, because of non-response or resistance mutations, many fail to benefit from these potentially life-saving medicines," said Peter Kolchinsky, PhD, managing partner at RA Capital Management. "We are proud to support the Kinnate team and see tremendous potential for their lead programs to significantly increase the number of people who could benefit from targeted precision oncology therapeutics."

On August 26, 2020 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes, reported that it will be presenting at the LD 500 Virtual Investor Conference on Tuesday, September 1, 2020 at 1:20 p.m. ET. Rodney Varner, Chairman and Chief Executive Officer of Genprex, will present virtually to an online audience (Press release, Genprex, AUG 26, 2020, View Source [SID1234564035]).

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Mr. Varner will deliver a Company overview and provide updates on its product pipeline, including its lead drug candidate, GPX-001, which received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for use in combination with AstraZeneca’s Tagrisso in late stage lung cancer patients with EGFR mutations whose tumors progress on Tagrisso. Mr. Varner will also provide an overview of the Company’s preclinical diabetes gene therapy candidate that may have the potential to cure Type 1 and Type 2 diabetes.

The presentation will be webcast live and available for replay on the LD 500 conference website here and via the investor relations section of the company’s website at Genprex.com.

Event: The LD 500 Virtual Conference

Presentation Date: Tuesday, September 1, 2020

Presentation Time: 1:20 p.m. ET – Track 2

Registration Link: https://ld-micro-conference.events.issuerdirect.com/

Webcast Link: https://bit.ly/31b1i20

The LD 500 will take place September 1-4, 2020 and will feature some of the most prominent companies in the micro-cap world, alongside interviews and keynotes with small-cap leaders.

View Genprex’s LD 500 profile here: https://bit.ly/2CHzOrt

On August 26, 2020 Novartis reported that, at primary analysis, the Phase III ASCEMBL study met its primary endpoint of statistically significant superiority in major molecular response (MMR) rate at 24 weeks for asciminib (ABL001) vs. bosutinib1 (Press release, Novartis, AUG 26, 2020, View Source [SID1234564031]). The study evaluates asciminib – a novel investigational treatment specifically targeting the ABL myristoyl pocket (STAMP) – in adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine-kinase inhibitors (TKIs)1. Patients with failure or intolerance to the most recently administered TKI therapy were included in the trial1.

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"Our ability to treat patients with TKIs changed CML care forever. However, the risk of disease progression is a reality for many patients – especially those who experience resistance to sequential TKI therapy or those who cannot adhere to treatment due to the daily impact of intolerable side effects16," said John Tsai, MD, Head of Global Drug Development and Chief Medical Officer, Novartis. "We are incredibly grateful to the patients and investigators around the world who participated in this study. These results with asciminib are a testament to our commitment to further transform CML care – this time through STAMP inhibition, by exploiting a natural regulatory mechanism of the ABL kinase."

Data from the ASCEMBL trial will be submitted for presentation at an upcoming medical meeting, and results will be shared with regulatory authorities. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for asciminib.

Findings from earlier studies of asciminib were presented during past annual meetings of the European Hematology Association (EHA) (Free EHA Whitepaper), and some details can be found here13,14.

About asciminib (ABL001)
Asciminib (ABL001) is an investigational treatment specifically targeting the ABL myristoyl pocket (STAMP). As a STAMP inhibitor, asciminib may help address tyrosine-kinase inhibitor (TKI)-resistance and intolerance in later treatment lines of chronic myeloid leukemia (CML)8-14, and it is being studied in several clinical trials in hopes of helping patients across multiple treatment lines of CML7,10,11,13-15,17.

About ASCEMBL
ASCEMBL is a Phase III, multicenter, open-label, randomized study comparing the oral investigational treatment asciminib (ABL001) versus bosutinib in patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine-kinase inhibitors (TKIs)1. Patients with failure or intolerance to the most recently administered TKI therapy were included in the trial1.

About Novartis Commitment to CML
Our ongoing research in Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) has helped transform the disease from a fatal leukemia to a chronic condition in most patients. Novartis maintains an unwavering commitment to scientific innovation and access to care for patients worldwide. As an organization committed to patients, Novartis continues to reimagine CML care by pursuing ambitious goals with courage, passion and commitment for the global CML community.

On August 26, 2020 Cellesce reported that a new paper exploring the application of patient-derived organoids (PDOs) in the study of novel inhibitors of stem cell activity has recently been published in the journal PLOS ONE (Badder et al., 2020) (Press release, Cellesce, AUG 26, 2020, View Source [SID1234564030]).

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The study utilised 3D image-based morphometric analysis to quantify over 600 different features from individual organoids following treatment with TNKSi. While the morphometric analysis approach mirrored the trend seen in traditional biochemical assays, importantly this more sophisticated method was able to detect subtle alterations in growth and morphology in response to TNKSi with much greater accuracy. This leads to the conclusion that whilst traditional biochemical assays still have value in detecting compounds that merit further investigation in early stage drug discovery, combining these with 3D morphological analysis could be the key to unlocking the full potential of organoids in predictive drug testing at a much larger scale.

The study was led by Cellesce founding director Professor Trevor Dale’s Cardiff University-based academic research group working together with Cellesce and other partners. It describes the derivation of a novel set of colorectal cancer PDOs. The PDO models are then used as a platform to test the response of colorectal cancer to Wnt pathway modulation using small molecule inhibitors of the tankyrase protein (TNKSi). The work utilises a range of analysis techniques and highlights 3D quantitative image analysis in particular as having the potential to greatly enhance the high throughput prediction of compound efficacy in pre-clinical testing.

In recent years, there has been a shift within the drug discovery industry to focus on the development of compounds targeting ‘cancer stem cell’ populations within tumours. Historically, conventional chemotherapeutics have aimed to target the tumour bulk, to kill as many tumour cells as possible; the effects of which are usually to drive tumour regression in the short-term, albeit with greater side-effects – and a high chance of patient relapse. It is now widely understood that, in order to permanently prevent tumour growth, the initiating cancer stem cell population must be removed or inhibited. In the patient, this might have a relatively small impact initially on overall tumour size, but a longer term more effective treatment caused not by killing the cells, but by a more subtle change in the behaviour of the cells within the tumour.

The study of such targeted compounds has led to demand for better predictive model systems. While historical drug discovery has relied heavily on the predictive power of 2D cancer cell lines, their lack of cellular heterogeneity and relevant phenotypic behaviour leaves them largely unsuited for the study of cancer stem cell inhibitors, and far from ideally placed for anti-cancer drug development in general.

PDOs – which retain intra-tumoral complexity and, crucially, stem cell function – are now gaining increasing momentum as predictive in vitro models in the drug discovery field, with the potential to reduce compound attrition rates and development costs, ultimately increasing the number of successful compounds available for use in the clinic. A more complex model, the study argues, demands a more comprehensive method of analysis that is capable of capturing the complete range of changes that may occur in response to treatment.

The organoid lines generated for this study are licensed for sale by Cellesce in large scale validated batches produced using Cellesce’s patented bioprocess. Cellesce PDOs:

Are vialled ready for plating straight into the desired format
Come with full protocols and technical support
A custom expansion service is available with:
Culture optimisation and banking options
Large scale expansion with custom vialling
Cellesce has produced an Application Note that summarises the paper’s findings.

On August 26, 2020 InDex Pharmaceuticals Holding AB (publ) reported that interim report January – June 2020 (Press release, InDex Pharmaceuticals, AUG 26, 2020, View Source [SID1234564029])

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Focus on phase III preparations

"The commercial preparations have confirmed our belief in the potential of cobitolimod, where we estimate that the annual sales at a successful commercialisation can reach more than USD 1 billion," says Peter Zerhouni, CEO of InDex Pharmaceuticals.

Period April – June 2020

Net sales amounted to SEK 0.0 (0.0) million
Operating loss amounted to SEK –15.6 (–17.7) million
Result after tax amounted to SEK –15.6 (–17.7) million, corresponding to SEK –0.17 per share (–0.26) before and after dilution
Cash flow from operating activities amounted to SEK –32.8 (–13.6) million
Period January – June 2020

Revenues amounted to SEK 0.0 (0.0) million
Operating result amounted to SEK –39.6 (–34.9) million
Result after tax amounted to SEK –39.6 (–34.9) million, corresponding to SEK –0.45 per share (–0.51) before and after dilution
Cash flow from operating activities amounted to SEK –54.7 (–32.0) million
Cash and cash equivalents at the end of the period amounted to SEK 70.6 (50.5) million
Number of employees at the end of the period was 7 (7)
Number of shares at the end of the period was 88,781,275
All comparative amounts in brackets refer to the outcome during the corresponding period 2019.

Significant events during April – June 2020

InDex received positive responses from FDA and EMA regarding phase III development of cobitolimod for the treatment of moderate to severe ulcerative colitis
The annual general meeting in InDex Pharmaceuticals Holding AB was held on Monday April 20, 2020. Board members Wenche Rolfsen (also chairman), Uli Hacksell, Lennart Hansson and Stig Lökke Pedersen were re-elected, and Marlene Forsell and Yilmaz Mahshid were elected as new ordinary board members
Significant events after the reporting period

No significant events have occurred after the end of the reporting period
Other events

InDex announced that the successful results of the CONDUCT study will be presented orally at two leading medical conferences
CEO statement
Our focus remains on the phase III preparations with cobitolimod in ulcerative colitis. To assist, we have expanded our circle of medical advisors by setting up a North American advisory board. It is fantastic that we have succeeded to attract six of the top clinical experts in inflammatory bowel disease to support the continued development and positioning of cobitolimod with a focus on the US market. The group’s first meeting in June clearly showed that they see a potential prominent role for cobitolimod in the future treatment of ulcerative colitis. We continue the work to finalize the phase III design and the associated financing. Contingent on how the Covid-19 pandemic evolves, we plan to start the study in the second quarter of 2021.

We have also had a US consultancy firm to detail the positioning as a complement to the market research we conducted during the first quarter of this year, which has provided us with a deeper understanding of the future market. The commercial preparations have confirmed our belief in the potential of cobitolimod, where we estimate that the annual sales at a successful commercialisation can reach more than USD 1 billion. More than 2 million people have ulcerative colitis in the US, Europe and Japan, and of these about 60 percent are estimated to suffer from moderate to severe ulcerative colitis. Approximately 55 percent of this group are considered to have left-sided colitis and more than half do not respond to conventional treatment and need other alternatives. With an assumed price in the US, Europe and Japan in line with the most recently launched products, it corresponds to an addressable market for moderate to severe left-sided ulcerative colitis of over USD 9 billion annually.

Since May, there have been several important news regarding competing development projects in phase III in ulcerative colitis. One project has been discontinued and others have reported less promising clinical results. Several of these drug candidates will probably not reach the market in the end. Cobitolimod has a completely different mechanism of action and safety profile than these substances, and for InDex the news mainly mean reduced future competition. It will also be very interesting to get more data from these studies that we can learn from for our own development.

Our phase IIb results from the CONDUCT study will be presented orally in October at two leading gastroenterology conferences; the UEGW in Europe and the ACG in the US. These events are great opportunities for us to further reach the international scientific community with the successful results, as well as potential partners and specialist investors.

It is very inspiring with the support from all our medical advisors. They point out cobitolimod’s unique combination of efficacy and safety as well as the novel mechanism of action. I look forward to an eventful autumn for InDex with the continued phase III preparations.

Publication
This information is information that InDex Pharmaceuticals Holding AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation (MAR). The information was