On September 3, 2020 Hutchison China MediTech Limited ("Chi-Med") (Nasdaq/AIM: HCM) reported that it has initiated a Phase II study of HMPL-453, its novel small molecule inhibitor targeting fibroblast growth factor receptors ("FGFR"), in patients with advanced intrahepatic cholangiocarcinoma ("IHCC"), which is a type of liver cancer (Press release, Hutchison China MediTech, SEP 3, 2020, https://www.chi-med.com/chi-med-initiates-a-phase-ii-trial-of-hmpl-453-in-patients-with-advanced-ihcc-in-china/ [SID1234564383]).

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The clinical study is a single-arm, multi-center, open-label study, evaluating the efficacy, safety and pharmacokinetics of HMPL-453 in patients with advanced IHCC with FGFR2 fusion that had failed at least one line of systemic therapy.

The primary outcome measure is objective response rate (ORR). Secondary outcome evaluations include preliminary efficacy measures, such as disease control rate (DCR), time to response (TTR), duration of response (DoR), progression-free survival (PFS), and overall survival (OS). Additional details may be found at clinicaltrials.gov, using identifier NCT04353375.

About IHCC
IHCC is a cancer that develops in the cells within the bile ducts[1]. With over 390,000 new cases every year, China accounted almost 50% of the world’s annual incidence of liver cancer in 2018.[2],[3] After hepatocellular carcinoma ("HCC"), IHCC is the second most common primary hepatic malignancy accounting for 10% to 20% of newly diagnosed liver cancers[4]. Approximately 10-15% of IHCC patients have tumors that harbor FGFR2 fusion[5],[6]. Long-term survival for patients with IHCC is worse than for HCC, which may be related to a high propensity for regional and distant metastases as well as the lack of effective systemic therapy options[4].

About FGFR
FGFRs are a sub‑family of receptor tyrosine kinases. Activation of FGFR signaling pathways is central to several biological processes. In normal physiology, FGF/FGFR signaling is involved in embryonic development (organogenesis and morphogenesis), tissue repair, angiogenesis, neuroendocrine and metabolism homeostasis. Given its complexity and critical role in a number of important physiological processes, aberrant FGFR signaling has been found to be a driving force in tumor growth, promotion of angiogenesis, as well as conferring resistance to anti‑tumor therapies.

About HMPL‑453
HMPL‑453 is a novel, highly selective and potent small molecule inhibitor targeting FGFR 1, 2 and 3. In pre‑clinical studies, HMPL‑453 demonstrated superior potency and better kinase selectivity as compared to other drugs in the same class, as well as a favorable safety profile. Enrollment has been completed for the dose escalation of the Phase I study of HMPL-453 in China (clinicaltrials.gov identifier NCT03160833). Enrollment of a Phase II study is ongoing in patients with advanced malignant mesothelioma in China (clinicaltrials.gov identifier NCT04290325).

On September 3, 2020 AbbVie (NYSE: ABBV) reported that it will participate in the Morgan Stanley 18th Annual Global Healthcare Conference on Wednesday, September 16, 2020 (Press release, AbbVie, SEP 3, 2020, View Source [SID1234564382]). Richard A. Gonzalez, chairman and chief executive officer, will present at 10 a.m. Central time.

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A live audio webcast of the presentation will be accessible through AbbVie’s Investor Relations website at investors.abbvie.com. An archived edition of the session will be available later that day.

On September 2, 2020 Rigel Pharmaceuticals (Nasdaq: RIGL) reported that Raul Rodriguez, the company’s president and chief executive officer, is scheduled to participate in a panel discussion on COVID-19 during Citi’s 15th Annual BioPharma Virtual Conference taking place September 9-10, 2020 (Press release, Rigel, SEP 2, 2020, View Source [SID1234569922]).

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Citi Panel Details:
Panel Topic: State of Play for COVID-19 Therapeutics
Date: Wednesday, September 9
Time: 9:50 a.m. Eastern Time

To access the event live or the archived webcast, go to the Investor Relations section of the company’s website at www.rigel.com. Please connect to Rigel’s website several minutes prior to the start of the live webcast to ensure adequate time for any software download that may be necessary.

On September 2, 2020 Oblato, Inc reported that The U.S. Food and Drug Administration has awarded Rare Pediatric Disease Designation (RPDD) for diffuse intrinsic pontine glioma (DIPG) and Orphan Drug Designation for treatment of malignant glioma to OKN-007, an investigational drug discovered at the Oklahoma Medical Research Foundation (Press release, Oblato, SEP 2, 2020, View Source [SID1234564622]).

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DIPG is a fast-growing pediatric cancer that starts in the brain stem. It is one of several sub-categories of malignant gliomas, deadly cancers of the brain and spinal cord.

"We are very pleased to receive the successful designations from the FDA for our proprietary compound OKN-007," said Oblato President and Chief Executive Officer Won S. Yang. These designation programs provide for special status and priority review of regulatory applications for new therapies for rare pediatric or "orphan" diseases, conditions that affect limited patient populations.

According to the National Brain Tumor Society, approximately 26,000 Americans will be diagnosed with primary malignant brain tumors this year. Of those, DIPG.org reports that up to 300 will be cases of DIPG.

OKN-007 was initially discovered by OMRF scientists Rheal Towner, Ph.D., and Robert Floyd, Ph.D. Oblato acquired all rights to OKN-007 from OMRF, and the company is currently testing the investigational drug in a Phase 2 clinical study of 56 patients suffering from recurrent glioblastoma, the most aggressive form of glioma. The patients are being treated with the drug in combination with another medication, temozolomide, at eight sites in the U.S.

In pre-clinical studies at OMRF, Towner has also shown that OKN-007 inhibits growth of human DIPG tumors implanted in experimental models. Oblato is planning to begin clinical trials in DIPG patients in 2021. "Right now, there is no effective treatment for this deadly brain cancer," said Towner.

Going forward, Oblato and OMRF will continue collaborating, with a focus on improving treatment for patients suffering from a variety of solid-tumor cancers.

"OMRF and Oblato are committed to a single goal: helping patients overcome these life-threatening illnesses," said OMRF Director of Technology Ventures Andrew Westmuckett, Ph.D. "Our hope is that OKN-007 can transform the therapeutic landscape."

On September 2, 2020 Merck and Pfizer reported that The National Institute for Health and Care Excellence (NICE) has now published final guidelines endorsing NHS use of immunotherapy Bavencio (avelumab) in combination with axitinib as a first-line treatment for kidney cancer (Press release, Merck & Co, SEP 2, 2020, View Source [SID1234564591]).

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Current NHS treatment for untreated advanced renal cell carcinoma (RCC) includes sunitinib, pazopanib, tivozanib or cabozantinib.

NICE concluded that clinical trial evidence shows that, for people with untreated advanced RCC, Bavencio plus axitinib increases how long people live without their disease getting worse compared with sunitinib, while early data also suggest the therapy increases survival.

However, it says this is uncertain because the final trial results are not available yet, and the Institute also noted the lack of trials comparing Bavencio/axitinib directly with other current options.

Bavencio plus axitinib has the potential to be cost effective, but more evidence is needed to address these uncertainties and update the economic model, which is why its use will be funded via the Cancer Drugs Fund while further data is collected.

Bavencio is an immune checkpoint inhibitor targeting PD-L18 and axitinib is an antiangiogenic VEGF-targeted TKI, their complementary mechanisms of action targeting two key pathways that tumours use to grow.

The combination was made available as part of the Early Access to Medicines Scheme (EAMS) in August 2019, which has allowed more than 150 patients to gain earlier access to the treatment throughout the UK.