On June 1, 2024 Ankyra Therapeutics, a clinical-stage oncology company developing anchored immunotherapies to improve the therapeutic window for immuno-oncology drugs, reported that it is presenting a Trial in Progress poster for the phase 1 ANCHOR clinical trial at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held May 31 – June 4, 2024 in Chicago, IL (Press release, Ankyra Therapeutics, JUN 1, 2024, https://www.businesswire.com/news/home/20240601270271/en/Ankyra-Therapeutics-Announces-Trial-in-Progress-Poster-Presentation-at-2024-ASCO-Meeting-and-Approval-of-ANK-101-Phase-1-Protocol-Amendment [SID1234643967]).

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Ankyra Therapeutics has developed an anchored drug-delivery platform based on linking immunotherapy drugs to aluminum hydroxide. The linked compounds are delivered locally to the tumor, where they are retained for several weeks promoting anti-tumor activity without systemic toxicity. Ankyra’s lead asset, ANK-101, is comprised of interleukin-12 with an alum-binding peptide that enables direct linkage with aluminum hydroxide. The ANCHOR study is a multi-institution, first-in-human, phase I clinical trial of ANK-101 in patients with superficially accessible solid tumors who have progressed after standard therapy was initiated in February 2024. The primary objectives of the study are to determine the safety, tolerability, and recommended dose of ANK-101 for further studies. The trial is active at five clinical study sites: Massachusetts General Hospital in Boston, MA; Providence Cancer Institute in Portland, OR; University of Pittsburgh in Pittsburgh, PA; Princess Margaret Hospital in Toronto, ON; and the National Cancer Institute (NCI), part of the National Institutes of Health, in Bethesda, MD. The poster presentation will include the rationale for the trial, study objectives, clinical trial design, and an update on accrual. The study will also evaluate pharmacokinetics, immune biomarkers, and quality of life.

In addition, Ankyra has received clearance from both the U.S. Food and Drug Administration (FDA) and Health Canada to amend the phase I clinical trial to include a second dose-escalation and expansion part to evaluate ANK-101 for injection into solid tumors located in visceral organs. This part will run concurrently with the current Phase I study with dosing to begin once patients in the superficial part have cleared a 21-day dose-limiting toxicity (DLT) observation period.

"We are delighted with the progress in the current phase I study which will help establish the initial safety profile and dosing selection for ANK-101," stated Howard L. Kaufman, MD, CEO of Ankyra Therapeutics. Dr. Kaufman also stated that "this study is the first step to realize the potential for anchored immunotherapy to deliver high doses of immunotherapy without systemic toxicity, which could represent an important advance in how we deliver effective cancer treatment in a safer manner." Joe Elassal, MD, Chief Medical Officer at Ankyra added "the recent approval to extend ANK-101 to visceral tumors is especially exciting as we can now study a broader patient population that could extend the potential indications for ANK-101 treatment".

For patients interested in enrolling in this clinical trial at NCI, please call NCI’s toll-free number: 1-800-4-Cancer (1-800-422-6237) (TTY: 1-800-332-8615); visit the website: View Source; and/or email: [email protected].

Title: A phase 1, open-label, dose escalation study on the safety and tolerability of ANK-101 in advanced solid tumors
Session Type: Poster Session
Session Title: Developmental Therapeutics – Immunotherapy
Track: Developmental Therapeutics – Immunotherapy
Sub Track: New Targets and New Technologies (IO)
Session Date and Time: Saturday June 1, 2024 9:00 AM CDT
Location: Hall A
Poster Board Number: 158a
Published Abstract Number: TPS2689
Citation: J Clin Oncol. 42, 2024 (suppl 16; abstr TPS2689)

The poster will be available on the publications section of Ankyra’s website after the meeting at View Source

About ANK-101

ANK-101 is an anchored drug complex composed of interleukin-12 (IL-12) linked to aluminum hydroxide. ANK-101 enables local delivery of functional IL-12 to the tumor microenvironment where it remains biologically active for several weeks but does not diffuse into the systemic circulation, thereby avoiding systemic toxicity. Treatment with ANK-101 in animal models has been associated with recruitment and retention of CD8+ T cells, NK cells and M1 macrophages activating innate and adaptive anti-tumor immunity. ANK-101 is being evaluated for the treatment of advanced solid tumors alone and in combination with anti-PD-1 agents. The Phase 1 first-in-human, open-label clinical trial of ANK-101 as a monotherapy (NCT:06171750) consists of a dose escalation portion that will evaluate the safety and tolerability of ANK-101, followed by dose expansion cohorts.

On June 1, 2024 Tempus, a leader in artificial intelligence and precision medicine, reported an expansion of its collaboration with AstraZeneca, which leverages Tempus Next to arm physicians with technology that supports the delivery of guideline-directed biomarker testing for patients with non-small cell lung cancer (NSCLC) (Press release, Tempus, JUN 1, 2024, View Source [SID1234643966]). The magnitude and frequency of updates to oncology guidelines each year make it difficult for the updates to be rapidly incorporated into patient treatment plans. As part of a larger, strategic collaboration, the two companies are now expanding upon a pilot program that utilizes Next, Tempus’ care pathway intelligence platform, to help physicians determine if their patients with NSCLC may benefit from guideline-directed molecular testing, including testing for epidermal growth factor receptor (EGFR) mutations.

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EGFR is a biomarker that presents in some patients with NSCLC and that has specific targeted therapies associated with it. Currently, ~30-40% of eligible NSCLC patients do not receive appropriate testing1. In September 2023, Tempus and AstraZeneca initiated a pilot program to leverage Next to identify this specific care gap at participating provider sites, using AI to scan unstructured clinical data to understand which patients may be eligible for testing based on clinical guidelines, and notifying treating physicians to inform care. The pilot’s initial success within the first six months prompted a new expansion of the program to support deployment of the care pathway model in a total of 15 provider sites.

"Next was designed to ensure all patients have access to treatment plans based on the most up-to-date guidelines by equipping physicians with cutting-edge tools in their existing workflows to support the delivery of evidence-based care," said Chris Scotto DiVetta, Senior Vice President, AI Applications at Tempus. "We were excited to see initial success in this program and look forward to contributing to a future where advanced technology and medical expertise unite to improve outcomes for all patients."

Tempus Next is designed to accelerate the adoption of precision medicine and enhance patient outcomes. The solution integrates seamlessly with EMRs in order to analyze a comprehensive suite of data—including clinical notes, molecular information, and imaging—to pinpoint certain deviations from care guidelines. Participating health systems receive regular updates, supporting guideline-directed care for their patients. To learn more, visit tempus.com/oncology/care-pathway-solution.

On June 1, 2024 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported long-term follow-up results, including the first-ever overall survival (OS) findings, from the Phase 2b HERIZON-BTC-01 clinical trial of zanidatamab in previously treated, unresectable, locally advanced, or metastatic HER2-positive biliary tract cancer (BTC) (Press release, Jazz Pharmaceuticals, JUN 1, 2024, View Source [SID1234643956]). These data will be featured at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in a poster presentation during the Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary session on June 1, 2024, at 1:30-4:30 p.m. CDT. Zanidatamab is a human epidermal growth factor receptor 2 (HER2)-targeted bispecific antibody being studied in multiple solid tumors.

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For the trial’s primary endpoint, results demonstrated that a confirmed objective response rate (cORR) by independent central review (ICR) was maintained at 41.3% (95% confidence interval (CI): 30.4, 52.8) and one additional patient achieved a complete response (n=2; 2.5%) since initial findings were presented at the ASCO (Free ASCO Whitepaper) Annual Meeting in 2023. The median duration of response (DoR), one of the trial’s key secondary endpoints, increased by approximately 2 months to 14.9 months (95% CI: 7.4, not reached), compared to the previously reported findings. In this data cut, zanidatamab demonstrated a median estimated OS, another secondary endpoint, of 15.5 months (95% CI: 10.4, 18.5) in all patients with HER2+ BTC, 18.1 months (95% CI:12.2, 23.2) in patients with IHC 3+ tumors, and 5.2 months (95% CI: 3.1, 10.2) in patients with IHC 2+ tumors. Results highlight the clinically meaningful benefits of sustained and durable responses with continued treatment with zanidatamab.

"Patients with BTC are typically diagnosed when their disease is at an advanced stage, which is associated with a poor prognosis," said Shubham Pant, M.D., M.B.B.S. professor in the Department of Gastrointestinal Medical Oncology and Investigational Cancer Therapeutics at The University of Texas MD Anderson Cancer Center. "Historically, overall survival with standard-of-care chemotherapy in second-line patients with advanced BTC is reported to be between 6-9 months1 so there is a significant unmet need for targeted therapies that can potentially improve survival. The deep and durable responses seen in this study signal the potential to fill a critical unmet patient need with zanidatamab, a chemotherapy-free option, among patients with HER2-expressing cancers."

"We are encouraged by the updated results from the pivotal HERIZON-BTC-01 trial demonstrating sustained clinical activity in previously treated patients with advanced HER2-positive BTC. Results from HERIZON-BTC-01 were included in the Biologics License Application (BLA) for zanidatamab, which was granted Priority Review by the FDA earlier this week, as well as in the Marketing Authorization Application for zanidatamab which was recently submitted to the European Medicines Agency," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "Clinical development of zanidatamab for the treatment of advanced HER2-positive BTC continues with HERIZON-BTC-302 (NCT06282575), the ongoing, global, randomized Phase 3 trial of zanidatamab in combination with standard-of-care therapy in the first-line setting for patients with HER2-positive BTC. We also look forward to investigating zanidatamab’s potential in other HER2-expressing solid tumors, including in cases resistant to prior HER2-targeted therapies."

Trial Results

Results from this long-term analysis of the Phase 2b HERIZON-BTC-01 trial (NCT04466891) indicate that zanidatamab monotherapy demonstrated sustained and durable antitumor responses in previously treated patients with HER2-positive unresectable, locally advanced, or metastatic BTC and support the clinically meaningful benefit of continued treatment with zanidatamab. The safety profile in all enrolled patients remained manageable with favorable tolerability compared with the initial analysis. Two (2.3%) patients discontinued treatment due to treatment-related adverse events (TRAEs).

The trial evaluated zanidatamab (20 mg/kg IV once every 2 weeks) in patients with HER2-positive, locally advanced unresectable, or metastatic BTC who had received prior gemcitabine-containing therapy. Patients with prior HER2-targeted therapy use were excluded from the trial. All patients were required to have centrally confirmed HER2-amplified tumors (assessed by in situ hybridization). Patients (n=87) were assigned into two cohorts based on tumor IHC status: Cohort 1 (n=80) included patients who were IHC 2+/3+ (HER2-positive) and Cohort 2 (n=7) included patients who were IHC 0/1+. The median duration of follow-up was 21.9 (16-34) months. Tumors were assessed every 8 weeks per RECIST v1.1. Updated efficacy analyses include only Cohort 1, while safety analyses include Cohorts 1 and 2.

As of July 28, 2023, data from HER2-positive BTC patients enrolled in Cohort 1 (n=80) demonstrated:

With longer follow-up, the cORR was maintained from the initial analysis (n=33; 41.3%) (95% CI: 30.4, 52.8), with one additional complete response (n=2; 2.5%).
Although the trial was not designed to detect treatment effects by HER2 status, as previously reported, in a pre-planned subgroup analysis of cORR by HER2 expression, responses were observed in patients with IHC 3+ tumors (cORR: 51.6% [95% CI: 38.6%-64.5%]) and IHC 2+ tumors (cORR: 5.6% [95% CI: 0.1-27.3%])2.
A two-month increase in the median DoR to 14.9 months (95% CI: 7.4, not reached).
In patients with IHC3+ tumors, the median DoR was 14.9 months (95% CI: 7.4, not reached).
The DOR in the 1 responder with IHC 2+ tumors was 7.5 months.
A median OS (95% CI) of 15.5 months (95% CI: 10.4, 18.5).
The median OS in patients with IHC 3+ was 18.1 months (95% CI: 12.2, 23.2).
The median OS in patients with IHC 2+ was 5.2 months (95% CI: 3.1, 10.2).
Median progression-free survival (PFS) was maintained (5.5 months [95% CI: 3.6, 7.3]) compared with the initial analysis, which had a data cutoff of October 10, 2022.
In patients with IHC 3+ tumors, the median PFS was 7.2 months (95% CI: 5.4, 9.4) months.
In patients with IHC 2+ tumors, the median PFS was 1.7 months (95% CI: 1.0, 3.3) months.
As previously reported for Cohorts 1 and 2, zanidatamab demonstrated a manageable and tolerable safety profile, with no new safety signals identified and no deaths that were treatment related. TRAEs leading to dose reductions remained infrequent. Serious TRAEs occurred in eight (9.2%) patients. One patient experienced serious TRAEs since the initial analysis (alanine aminotransferase increased and aspartate aminotransferase increased).Treatment discontinuation rate was 2.3% and no additional patients discontinued treatment due to TRAEs since the initial analysis.

About BTC
BTC, including gallbladder cancer and intrahepatic and extrahepatic cholangiocarcinoma, account for <1% of all adult cancers globally and are often associated with a poor prognosis.3,4 The human epidermal growth factor receptor 2 (HER2) is a well-validated target for antitumor therapy in other cancers. Across the U.S., Europe, and Japan, approximately 12,000 people are diagnosed with HER2+ BTC annually.5,6,7,8

About Zanidatamab
Zanidatamab is an investigational HER2-targeted bispecific antibody that can simultaneously bind two non-overlapping epitopes of the HER2 receptor, known as biparatopic binding. This unique design and increased binding results in multiple mechanisms of action, including dual HER2 signal blockade, removal of HER2 protein from the cell surface, and immune-mediated cytotoxicity leading to encouraging antitumor activity in patients. Zanidatamab is being developed in multiple clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is being developed by Jazz and BeiGene, Ltd. (BeiGene) under license agreements from Zymeworks, which first developed the molecule.

The U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review of the Biologics License Application (BLA) for zanidatamab with a Prescription Drug User Fee Act (PDUFA) action date of November 29, 2024. Zanidatamab was also granted Breakthrough Therapy designation in patients with previously treated HER2 gene-amplified biliary tract cancers (BTC) and given two Fast Track designations: one as a single agent for refractory BTC and one in combination with standard of care chemotherapy for 1L gastroesophageal adenocarcinoma (GEA). Additionally, zanidatamab has received Orphan Drug designations from FDA for the treatment of BTC and GEA, as well as Orphan Drug designation from the European Medicines Agency for the treatment of BTC and gastric cancer. Zanidatamab was also granted Breakthrough Therapy designation from the Center for Drug Evaluation (CDE) in China.

On June 1, 2024 Immunofoco Biotech, a company dedicated to developing cell therapy products for solid tumors, reported that the clinical research data for two of its products have been accepted for presentation at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Immunofoco, JUN 1, 2024, View Source [SID1234643954]). The company will showcase the latest clinical trial findings for IMC001 in a poster presentation and for IMC002 in an online presentation during the ASCO (Free ASCO Whitepaper) event, scheduled from May 31 to June 4, 2024.

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Outstanding safety and efficacy data of IMC002, an autologous CLDN18.2-targeting CAR-T, in CLDN18.2+ advanced solid tumors (e16012)

Claudin 18.2 (CLDN18.2) is a promising therapeutic target for treating advanced solid tumors. IMC002 is an autologous CLDN18.2-targeting CAR-T cell directed by a high specific anti-CLDN18.2 VHH, the specificity of which was confirmed by a membrane proteome array assay. Preclinical studies showed that IMC002 exhibited high tumor specificity, potent anti-tumor efficacy, and excellent safety profile in both cellular and animal models.

As of January 17, 2024, three patients with advanced unresectable gastric cancer received IMC002 infusion. There was no dose-limiting toxicity (DLT) or serious adverse events (SAE) reported. All three patients experienced manageable cytokine release syndrome (CRS) at grade 1. In addition, all adverse events recovered quickly. Preliminary efficacy data showed that all three patients exhibited stable disease as their best overall response, as evaluated by RECIST 1.1 criteria. Among the two patients who received a dosage of 3 million CAR-T cells/kg, successful radical surgeries were performed at week (W) 11 and 44 post-IMC002 infusion respectively when CAR-T treatment reduced the tumor burden. Notably, pathological complete response (pCR) was achieved in one patient with surgery at W44. Noteworthy expansion of CAR-T cells in peripheral blood was observed in all three patients, with peak CAR+cell counts recorded between day 7-11 in the peripheral blood post-infusion. Moreover, CAR-T cell infiltration in the tumor tissues was detected in the surgical samples obtained at weeks 11 and 44. Significant increases of serum levels of IFN-y, IL-10, IP-10, IL-2 and IL-6 were observed in all three patients. The trial remains ongoing.

IMC002 demonstrated a promising safety profile, along with encouraging anti-tumor effects, including observed pCR and sustained expansion within tumor tissues, even at the low dosage levels among patients with advanced CLDN18.2+ gastric cancer. In addition, our trial has successfully facilitated a surgical treatment intervention following CAR-T therapy, enabling the downstaging of previously deemed unresectable gastric cancers.

Efficacy of EpCAM CAR-T IMC001 in advanced gastric cancers (Poster #4043)

An exciting phase I clinical trial was presented, showcasing the innovative application of CAR-T cell therapy targeting EpCAM for the treatment of advanced gastrointestinal (GI) cancers. Led by a collaborative team of physicians and scientists from Shanghai Changhai Hospital, The First Affiliated Hospital of Zhejiang University, and Suzhou Immunofoco Biotechnology Co., Ltd, the trial demonstrated promising safety and preliminary efficacy findings for IMC001.

Between August 18, 2021, and May 8, 2023, a total of 11 patients underwent IMC001 CAR-T cell infusion. As of the cutoff date (March 31st, 2024), infusions of low- and middle-dose IMC001 demonstrated a favorable safety profile. Among 10 evaluable patients, the disease control rate was 90%, with one patient in the low-dose group (1/3, 33.3%) and two in the middle-dose group (2/5, 40%) achieved a partial response (PR). The middle-dose was determined as the Recommended Dose, the median progression-free survival (PFS) was 18.1 weeks (95% CI 7.97, –) and the median overall survival (OS) was 55.1 weeks (95% CI 23.78, –) for this group; within the middle-dose group, 3/5 patients survived for more than 10 months. One patient in the middle-dose group achieved a confirmed PR by Week 24, leading to a radical gastrectomy at Week 27, and the patient had survived for more than 22 months by the cutoff date. Analysis of tumor immune microenvironment suggested that an inflamed tumor environment may favor the anti-tumor effects of IMC001.

In this phase I dose-escalation trial, IMC001 exhibited a favorable safety profile and encouraging efficacy in patients with advanced, pre-treated gastric cancer (GC). Further investigation is needed to further evaluate the potential of IMC001 for patients with advanced GC.

Dr. Tianhang Luo, a distinguished chief physician from Shanghai Changhai Hospital and principal investigator for the IMC001 and IMC002 trials, commented, "We have witnessed remarkable cases at our institution where patients, after receiving infusion with IMC001 or IMC002 CAR-T cell products, became eligible for surgical treatment by downstaging previously unresectable gastric cancers. Notably, there was a case where a patient treated with IMC002 underwent surgery about 10 months after CAR-T infusion, and the pathological examination of the resected tumor tissues showed only fibrous and adipose tissues without any tumor cells. This indicates successful infiltration of CAR-T cells into the solid tumor, effectively eliminating the tumor cells. A single infusion of CAR-T cells achieved disease control for over a year, with the tumor shrinking to complete remission, greatly reducing the need of other medication for the patient. This demonstrates the tremendous potential of CAR-T cell therapy in treating solid tumors."

Dr. Minmin Sun, Founder, Chairman, and CEO of Immunofoco Biotech, stated in an interview: "IMC002 and IMC001 have shown good safety and significant anti-tumor effects in treating patients with advanced gastric cancer. CAR-T cells have shown ongoing expansion within tumor tissues, with one patient achieving complete remission upon pathological examination. These encouraging results have greatly strengthened our commitment to expanding the scope of clinical trials and quickly advance to registrational phase II clinical trials. We firmly believe that IMC002 & IMC001 will offer more innovative treatment options to cancer patients worldwide."

On June 1, 2024 Sapience Therapeutics, Inc., a clinical-stage biotechnology company focused on the discovery and development of peptide therapeutics to address oncogenic and immune dysregulation that drive cancer, reported new clinical and biomarker data from its ST101 Phase 2 study in GBM at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Sapience Therapeutics, JUN 1, 2024, View Source [SID1234643953]). ST101 is a first-in-class antagonist of C/EBPβ, currently being evaluated in patients with recurrent and newly diagnosed GBM in the Phase 2 portion of an ongoing Phase 1-2 clinical study (NCT04478279).

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Dr. Fabio M. Iwamoto, Division of Neuro-Oncology, New York-Presbyterian/Columbia University Irving Medical Center, and Principal Investigator of the ST101-101 clinical study, said, "ST101’s clinical activity in GBM patients in the expansion and window-of-opportunity studies is highly encouraging, showing durable responses, a compelling survival benefit, and remodeling of the tumor microenvironment to an immune-active state. I am pleased to present this data at ASCO (Free ASCO Whitepaper) and look forward to ST101 advancing into Phase 2b combination studies."

Dr. Abi Vainstein-Haras, Sapience’s Chief Medical Officer, added, "ST101’s impressive clinical results and favorable safety profile add to the growing body of data supporting C/EBPβ as a promising target for the treatment of GBM. Our biomarker results demonstrate a clear impact on the tumor microenvironment, providing rationale to explore ST101 in combination with immune-oncology agents, such as checkpoint inhibitors. We look forward to advancing a comprehensive clinical development program for ST101 with the goal of extending survival and improving outcomes for patients with this devastating disease."

Oral presentation highlights include:

Presentation Title: "Efficacy and biomarker analysis of phase 2 (P2) and window-of-opportunity (WoO) cohorts of patients with glioblastoma (GBM) treated with ST101, an inhibitor of the transcription factor C/EBPβ"
Abstract Number for Publication: 2011
Session Type and Title: Clinical Science Symposium – Advancing Trial Design: Illuminating Tumor Evolution in Central Nervous System Cancer
Date and Time: 6/1/2024, 3:00 PM-4:30 PM CDT
Presenting Author: Fabio M. Iwamoto, MD, Division of Neuro-Oncology, New York-Presbyterian/Columbia University Irving Medical Center

ST101 has the potential to be a well-tolerated treatment option for patients with GBM
Outcome data presented from multiple cohorts of GBM patients (n=42)
Main study: monotherapy in recurrent GBM, n=30
2 patients with PRs, on study treatment for 55 weeks and 59+ weeks
7 patients with SD with on study treatment for a range of 13-79 weeks
53% OS and 40% OS at 9 and 12 months, respectively
Window-of-Opportunity Study: ST101 monotherapy in recurrent GBM, n=6
2 PRs (1 unconfirmed), 1 ongoing
2 SD, 1 ongoing
Median OS ~12 months; 3/6 patients alive (41-62 wks)
Window-of-Opportunity Study: combination of ST101 with standard-of-care (radiation and temozolomide) in newly diagnosed GBM, n=6
1 CR, duration ~1 year and ongoing
4 SD, 3 ongoing
5/6 patients alive (25-57 wks)
Biomarker data presented from Window-of-Opportunity study cohorts
ST101 crosses the BBB and penetrates tumor tissue as shown by IHC
Target (C/EBPβ) engagement and degradation shown by IHC
Modulation of the tumor immune microenvironment to promote anti-tumor activity
The slide presentation is available under the Presentations tab on the Sapience Therapeutics website.

About ST101

ST101, a first-in-class antagonist of C/EBPβ, is currently being evaluated in patients with newly diagnosed and recurrent GBM (ndGBM and rGBM) in the Phase 2 portion of an ongoing Phase 1-2 clinical study (NCT04478279). In an ongoing window-of-opportunity sub-study, ST101 is being evaluated as a monotherapy in rGBM and in combination with radiation and temozolomide in ndGBM, with patients receiving ST101 before and after surgical resection. ST101 has been granted Fast Track designation for rGBM from the U.S. FDA and orphan designations for glioma from the U.S. FDA and the European Commission.