On July 21, 2021 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported it will release results for the second quarter 2021 on Wednesday, July 28, 2021, after the market closes (Press release, Vanda Pharmaceuticals, JUL 21, 2021, View Source [SID1234585038]).

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Vanda will host a conference call at 4:30 PM ET on Wednesday, July 28, 2021, during which management will discuss the second quarter 2021 financial results and other corporate activities. To participate in the conference call, please dial 1-866-688-9426 (domestic) or 1-409-216-0816 (international) and use passcode 5298904.

The conference call will be broadcast simultaneously and archived on Vanda’s website, www.vandapharma.com. Investors should go to the website at least 15 minutes early to register, download, and install any necessary audio software.

A replay of the call will be available on Wednesday, July 28, 2021, beginning at 7:30 PM ET and will be accessible until Wednesday, August 4, 2021, at 7:30 PM ET. The replay call-in number is 1-855-859-2056 for domestic callers and 1-404-537-3406 for international callers. The passcode number is 5298904.

On July 21, 2021 Amplia Therapeutics Limited (ASX: ATX), ("Amplia" or the "Company"), a company developing new approaches for the treatment for cancer and fibrosis, reported it has received the unblinded results of its successful Phase 1 clinical trial of AMP945 (Press release, Amplia Therapeutics, JUL 21, 2021, View Source;[email protected] [SID1234585022]). The results confirm the Company’s preliminary analyses and show that, in the trial, AMP945 was safe and well tolerated at all doses tested, supporting its progression into later stages of clinical development in both cancer and fibrosis. Further details about the trial and its results are provided below.

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About the Phase 1 Trial
The clinical trial, designated as AMP945-101 and entitled "A Phase I, randomised, double blind, placebo-controlled study of the safety, tolerability and pharmacokinetics of single and repeat doses of AMP945 administered orally to healthy adult volunteers", was conducted at Nucleus Network in Melbourne. The study was conducted under a protocol approved by the Alfred Hospital Human Research Ethics Committee (HREC) in September 2020.

The Primary Endpoints for the trial were focused on the safety and tolerability of orally administered AMP945 which were assessed by evaluating the nature, incidence and severity of adverse events, withdrawals, physical examinations, vital signs, ECGs and safety laboratory test results including assessment of biochemical and haematological markers. Secondary endpoints assessed the pharmacokinetics of AMP945.

Overall, the trial recruited 56 healthy volunteers who were dosed with either single or multiple doses of AMP945 or placebo. Single and multiple doses of AMP945 up to 125mg and 100mg respectively, were given. Multiple doses were taken once daily for seven days. To study the effect of food on absorption of AMP945, one cohort of participants was given AMP945 both before and after food. Participants in the trial ranged in age from 18-65.

Safety and Tolerability
AMP945 was well tolerated at all doses given and there were no withdrawals or serious adverse events recorded in the trial. Adverse events were generally mild or moderate and were distributed evenly across participants assigned to AMP945 or placebo. Mild headache was the most frequently observed adverse event and the majority of safety findings were considered as either not related or unlikely to be related to AMP945. Events that were considered ‘possibly’ related to AMP945 included one incidence of diarrhoea, two incidences of headache, one taste disorder and one hot flush. There were no clinically significant changes in vital signs, clinical or laboratory parameters associated with AMP945. No adverse safety signals or dose-related trends were detected in any of the parameters measured.

Pharmacokinetics
AMP945 was delivered via capsules, taken with a glass of water. Following oral dosing, the plasma half-life of AMP945 was approximately 20 hours indicating that AMP945 was both orally bioavailable and could be administered once daily. Plasma levels of AMP945 exceeded the concentrations required to inhibit the intended target (FAK) with maximum plasma levels (Cmax) being achieved after approximately 2-4 hours post dose. Exposure parameters, including Cmax and area under the curve (AUC), all increased in a dose-dependent manner. There was no evidence of any food effect on the absorption of AMP945. Studies are ongoing to measure the inhibitory activity of AMP945 on FAK in skin punch biopsies and to assess the plasma metabolite profiles of AMP945.

Amplia’s CEO and Managing Director, Dr John Lambert, commented that "This Phase 1 trial has delivered exactly what we wanted: AMP945 has been shown to have a safety and tolerability profile suitable for progressing it into Phase 2 trials in both pancreatic cancer and pulmonary fibrosis. We are now planning those trials and expect to start the Phase 2 trial in pancreatic cancer around the end of 2021. With the successful completion of this Phase 1 trial Amplia has hit yet another significant milestone and I would like to thank our team, Amplia’s investors and the volunteers who stepped forward to participate in this successful trial." This ASX announcement was approved and authorised for release by the Board of Amplia Therapeutics

On July 21, 2021 Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), a leading drug development company in the field of brain cancer, reported that Glioblastoma (GBM) Awareness Day and calls for a greater focus on research from industry, academia, and government in order to better treat the disease (Press release, Kazia Therapeutics, JUL 21, 2021, View Source [SID1234585040]).

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GBM is the most common and most aggressive form of primary brain cancer, and it is expected that approximately 13,000 Americans will receive a GBM diagnosis in 2021. Unlike many other cancers there has been little progress made in the treatment of this disease and the standard of care has remained largely unchanged since the turn of the century.

Current treatment for GBM includes surgery, radiotherapy and chemotherapy with temozolomide. However, around two-thirds of patients receive no meaningful benefit from temozolomide. The median survival from diagnosis remains approximately 16 months and has improved little in the last twenty years.

Kazia CEO, Dr James Garner, said, "Enormous progress has been made in the treatment of so many cancers, but GBM has thus far remained intractable, with outcomes for patients relatively unchanged in two decades. However, the future for this disease looks much more promising, with new insights into the biology of GBM and a renewed focus from academia and industry combining to explore new therapeutic frontiers. We remain wholly confident that this disease can be beaten, and we call on all stakeholders – clinicians, researchers, government, industry, and patient advocates – to redouble their efforts on improving the prognosis for patients with glioblastoma."

About GBM Awareness Day

Glioblastoma Awareness Day takes place on July 21, 2021, with a specific focus on:

Increasing public awareness of glioblastoma
Honoring the individuals who have lost their lives to glioblastoma, a devastating disease, or are currently living with it
Supporting efforts to develop better treatments for glioblastoma that will improve the long term prognosis and quality of life of individuals diagnosed with the disease
Expressing support for the individuals who are battling brain tumors, as well as the families, friends, and caregivers of those individuals
Urging a collaborative approach to brain tumor research, which is a promising means of advancing the understanding and treatment of glioblastoma
Encouraging continued investment in glioblastoma research and treatments, including through the Glioblastoma Therapeutics Network and other existing brain tumor research resources
About Kazia’s Paxalisib

Kazia Therapeutics is at the forefront of potential new therapies for glioblastoma with the drug paxalisib, a brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway. Licensed from Genentech, Inc, in October 2016, paxalisib is currently completing a phase II study which has shown promising indications of clinical efficacy in interim analyses. Paxalisib has been granted orphan designation and Fast Track designation by FDA.

GBM AGILE

The GBM AGILE clinical trial, sponsored by the Global Coalition for Adaptive Research, a 501(c)(3) non-profit organization, commenced recruitment in June 2019. GBM AGILE aims to accelerate the development of new therapies for GBM via an adaptive study design that can explore multiple potential therapies in parallel. To date, three investigational drugs have joined the study: Bayer’s regorafenib, Kintara Therapeutics’ VAL-083, and Kazia Therapeutics’ paxalisib.

To date, GBM AGILE has screened over 650 patients. This progress is expected to accelerate as new sites in new territories come on stream.

For further information on GBM Awareness Day please visit: View Source

On July 21, 2021 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a precision oncology company targeting virus-associated malignancies, reported that the U.S. Food and Drug Administration (FDA) has cleared Viracta’s Investigational New Drug (IND) application to proceed into a Phase 1b/2 trial in patients with EBV+ recurrent or metastatic nasopharyngeal carcinoma (RM-NPC) and other EBV+ solid tumors (Press release, Viracta Therapeutics, JUL 21, 2021, View Source [SID1234585007]). The global Phase 1b/2 trial is designed to evaluate the safety and preliminary efficacy of Viracta’s all-oral combination regimen in advanced EBV+ solid tumors (including EBV+ RM-NPC), and in combination with the PD-1 inhibitor pembrolizumab in EBV+ RM-NPC. Initiation of the trial is expected in the second half of 2021.

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"Clearance of this IND application is a crucial milestone that underscores the broader applicability of our therapeutic approach to treating patients with EBV-associated malignancies," said Lisa Rojkjaer, M.D., Chief Medical Officer of Viracta. "EBV is the primary etiologic agent for NPC, one of the most commonly reported head and neck cancers worldwide. Patients with RM-NPC have a poor prognosis, with no standard treatment options for second or later lines of therapy, and median overall survival of less than 20 months. PD-L1 is known to be highly expressed by NPC and preliminary response rates of 20%-30% have been reported with PD-1 inhibitors for RM-NPC. Given the encouraging activity seen in our Phase 1b/2 trial of nanatinostat and valganciclovir in patients with EBV+ recurrent lymphomas, we now turn to evaluating this combination in patients with other EBV+ malignancies and explore potential synergies with checkpoint inhibition."

Ivor Royston, M.D., President and Chief Executive Officer of Viracta, added, "Expansion into our solid tumor program is an important component of our clinical and corporate strategy, and one that could significantly expand our addressable patient population and unlock significant value to shareholders. Our all-oral combination regimen represents a promising new therapeutic approach to patients with EBV+ solid tumors, and I’m very pleased that the program is progressing as planned and look forward to its continued advancement."

The Phase 1b dose escalation portion of the trial is designed to evaluate safety, pharmacokinetics, and determine the recommended Phase 2 dosing regimen of nanatinostat and valganciclovir for expansion in patients with EBV+ solid tumors. In Phase 2, the safety, preliminary efficacy and potential pharmacodynamic markers of nanatinostat and valganciclovir together and in combination with pembrolizumab will be evaluated in EBV+ RM-NPC patients.

About Nanatinostat

Nanatinostat (VRx-3996) is an orally available histone deacetylase (HDAC) inhibitor being developed by Viracta. Nanatinostat is selective for specific isoforms of Class I HDACs, which is key to inducing viral genes that are epigenetically silenced in EBV-associated malignancies. The nanatinostat and valganciclovir combination is being investigated in multiple subtypes of relapsed/refractory EBV+ lymphoma in two ongoing Phase 2 trials, one of which is a registration-enabling global, multicenter, open-label basket trial.

On July 21, 2021 Merus N.V. (Nasdaq: MRUS), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics and Triclonics), reported the novel mechanism of action (MOA) of MCLA-145, the Company’s clinical stage Biclonics T-cell agonist (Press release, Merus, JUL 21, 2021, View Source [SID1234585023]). MCLA-145 binds with high affinity and specificity to PD-L1 and CD137. The unique immunostimulatory mechanism of action of MCLA-145 was published in Nature Communications on June 21, 2021, titled "A human CD137×PD-L1 bispecific antibody promotes anti-tumor immunity via context dependent T cell costimulation and checkpoint blockade."

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"Our recent Nature Communications publication is another example of the power and potential of our unique Biclonics platform technology allowing for high throughput functional screening to discover novel Biclonics —human, common light chain, IgG antibodies in the bispecific format—that can unlock innovative biology," said Cecile Geuijen, Chief Scientific Officer. "Preclinically, MCLA-145 has been observed to potently activate immune effector cells, only in the context of the tumor microenvironment, and can simultaneously block inhibitory signals in the same immune cell population."

MCLA-145 was identified by screening hundreds of bispecific IgG antibodies for immune activation via PD-L1 engagement among other characteristics. MCLA-145 was shown to potently activate T cells even in the presence of suppressive conditions. Furthermore, MCLA-145 was shown to enhance T cell priming and to promote long-term T cell immunity. These in vitro findings were translated to in vivo experiments where MCLA-145 anti-tumor activity was superior to the current standard immune checkpoint inhibitor comparators and linked to recruitment and intratumor expansion of CD8+ T cells.

MCLA-145 is currently being evaluated in a phase 1 open-label, multicenter dose escalation study, including a planned safety dose expansion phase, in patients with solid tumors. MCLA-145 is the first drug candidate co-developed under Merus’ global collaboration and license agreement with Incyte, which permits the development and commercialization of up to 11 bispecific and monospecific antibodies from our Biclonics platform. Merus retains full rights to develop and commercialize MCLA-145, if approved, in the United States; and Incyte holds full rights to develop and commercialize MCLA-145 outside the United States.

About MCLA-145
Discovered through an unbiased functional screening of multiple immunomodulatory target combinations, MCLA-145 is a Biclonics T-cell agonist that binds with high affinity and specificity to human PD-L1 and CD137 in preclinical models. The unique immunostimulatory profile of MCLA-145 derives from the potential to potently activate immune effector cells in the context of the tumor microenvironment while simultaneously blocking inhibitory signals in the same immune cell population.