On March 25, 2020 Ono Pharmaceutical Co., Ltd. reported that ONO received a manufacturing and marketing approval of Velexbru (generic name: tirabrutinib hydrochloride) Tablet 80mg ("Velexbru"), a Bruton’s tyrosine kinase ("BTK") inhibitor, for the treatment of recurrent or refractory primary central nervous system lymphoma in Japan (Press release, Ono, MAR 25, 2020, View Source [SID1234584586]).

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This approval is based on the results from a multi-center, open-label, uncontrolled Phase I/II study (ONO-4059-02), evaluating an efficacy and safety of Velexbru in 44 patients with recurrent or refractory primary central nervous system lymphoma (PCNSL), receiving Velexbru orally once daily. In this study, the overall response rate (ORR) assessed by an independent review committee (IRC), a primary endpoint, was 52.9% (9/17 patients) in 17 patients who received 480 mg of Velexbru in the fasting which is the approved dosage and administration this time. Grade 3-4 neutropenia, leucopenia and hypertriglyceridemia each occurred in 11.8% (2/17) of patients. This approval represents that Velexbru is the first BTK inhibitor approved for the treatment of recurrent or refractory PCNSL in the world where no standard of care has been established. On November 27, 2019, ONO also submitted an application of Velexbru in Japan for the treatment of Waldenstrom macroglobulinemia (WM) and lymphoplasmacytic lymphoma (LPL).

About Primary Central Nervous System Lymphoma (PCNSL)

PCNSL is a malignant lymphoma in which the lesion is localized in the cerebrospinal cord (including the eyes) at the first onset. It is estimated that there are approximately 980 new cases with PCNSL *1, 2 per year in Japan . The signs and symptoms presented by patients with PCNSL vary depending on the site of the lesion, and include localized neuropathy, neuropsychiatric symptoms, symptoms associated with increased intracranial pressure, seizure, eye symptoms, headache, difficulty in movement, cranial neuropathy and radiculopathy. Currently, untreated PCNSL patients receive high-dose methotrexate-based treatment followed by whole-brain radiation therapy, by which a certain patient population shows long-term remissions, but many patients will relapse. There are also refractory patients who do not respond to the drug treatment. Standard treatment has not been established for patients with recurrent or refractory PCNSL, and treatment options are limited for them. Therefore, a new treatment drug is expected for *3 patients with recurrent or refractory PCNSL . *1 : Neurol Med Chir (Tokyo). 2017;57(Supplement 1):9-102. *2 : Jpn J Neurosurg VOL.24 NO.10 2015.10 *3 : Practical Guidelines for Neuro-Oncology 2019

About ONO-4059-02 Study

This study is a multi-center, open-label, single-arm Phase I/II study, evaluating the efficacy and safety of tirabrutinib hydrochloride monotherapy in patients with recurrent or refractory PCNSL. In this study, 44 patients were recruited and received tirabrutinib hydrochloride 320 mg (20 patients), 480 mg (7 patients) and 480 mg fasted (17 patients), once daily in either groups. Patients were treated until disease progression or unacceptable toxicity was observed.

About Velexbru

Velexbru (tirabrutinib hydrochloride), discovered and developed by ONO, is a highly selective, oral BTK inhibitor and has been developed for the treatment of patients with B-cell tumors and autoimmune diseases in Japan. B-cell receptor (BCR) signaling plays a core role in the survival, activation, proliferation, maturation and differentiation of B-cell lymphocyte. The BCR signaling pathway is known to be permanently activated, particularly in B-cell non-Hodgkin lymphoma (B-NHL) and chronic lymphocytic leukemia (CLL). Velexbru is expected to have a therapeutic effect because it inhibits BTK, a mediator located downstream of BCR. In December 2014, ONO out-licensed tirabrutinib hydrochloride to Gilead Sciences, Inc. (Gilead) to allow the right to develop and commercialize the product in all countries of the world, except Japan, South Korea, Taiwan, China and ASEAN countries where ONO retains the development and commercialization rights of the product.

Overview of Velexbru Tablet 80mg Product Name Velexbru Tablet 80mg Generic name (JAN) tirabrutinib hydrochloride Indication Relapsed or refractory primary central nervous system lymphoma Dosage and Usually, for adults, administer at 480 mg of tirabrutinib orally once a day administration in the fasting. The dose should be decreased based on patients’ condition. Manufacturer/distributor Ono Pharmaceutical Co., Ltd. Approval date March 25, 2020.

On March 25, 2020 Oasmia Pharmaceutical AB and US based Elevar Therapeutics Inc. reported that they have signed a global strategic partnership deal regarding the commercialization of Oasmia’s anticancer product Apealea (Press release, Oasmia, MAR 25, 2020, View Source [SID1234556577]). The agreement includes milestone payments with a potential of up to USD 678 million depending on Elevar’s achievement of future sales milestones, clinical development milestones and regulatory approval milestones. Elevar will also pay Oasmia double-digit royalties on sales of Apealea. Oasmia will also receive USD 20 million as an upfront payment.

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The worldwide partnership between Oasmia and Elevar grants Elevar the exclusive right to commercialize Apealea, a proprietary formulation of paclitaxel, in all global territories, with the exception of countries in which Oasmia and its partners are already present including Nordics, Baltics, Russia and some other CIS countries[1] in which Oasmia and its partners will continue to drive the commercialization of the product. The arrangement gives Elevar the right to sub-license Apealea to other strategic partners, including, for example, in Europe.

Elevar will be responsible for all regulatory application processes in its territory, including the submission of the approval application to the FDA in the US.

The collaboration between the two companies will include a joint steering committee and working teams in order to optimize the global development, launch and commercialization processes. The partnership will build upon Oasmia’s product development strategy for Apealea, and exploring possible new indications.

Oasmia will remain in sole control over, and will continue to develop, its proprietary technology platform XR17, for use with active pharmaceutical ingredients other than paclitaxel.

"This first major commercial partnership for Oasmia demonstrates both its clinical & regulatory capabilities in getting Apealea approved for the EU and its commercial capabilities by successfully negotiating a global partnership agreement with a US-based company for global exploitation of that product. This is a great achievement. Clinical studies have demonstrated that Apealea is an effective cancer treatment with strong benefits for cancer patients. We will capitalize on this partnership to move Oasmia to its next level of growth by continuing to develop the other compounds in our pipeline. We will also be looking to add new complementary assets. I believe by implementing this partnership, Oasmia has achieved a significant milestone and is on track to become a major oncology player, says Francois R Martelet, CEO at Oasmia.

"At Elevar, we are thrilled to take this exciting anti-cancer drug to the market. We see great potential to commercialize Apealea on a global scale and to develop it further, creating new tools for patients and health care professionals to fight cancer", says Alex Kim, CEO of Elevar.

"This is a great step forward for Oasmia on our way to truly capitalize on the proprietary XR17 technology that has been developed by our company. This deal, together with a lot of hard work the last year, has created a very good platform for the future growth of Oasmia. We have a lot more to achieve", says Jörgen Olsson, Chairman of the Board in Oasmia.

About Apealea
Apealea is a patented, water-soluble, intravenously injectable formulation of paclitaxel, developed using Oasmia’s proprietary technology platform – XR17 – which facilitates the solubility of paclitaxel. Paclitaxel is a chemotherapy medication used to treat a number of types of cancers. Apealea has been authorized for marketing by the European Commission. Apealea in combination with Carboplatin is approved for the treatment of adult patients with first relapse of platinum-sensitive epithelial ovarian cancer, primary peritoneal cancer and fallopian tube cancer.

On March 25, 2020 Amplia Therapeutics Limited (ASX: ATX) ("Amplia" or the "Company") reported that the United States Food and Drug Administration (FDA) has awarded Amplia’s Focal Adhesion Kinase inhibitor (FAKi) AMP945 Orphan-Drug Designation for the treatment of pancreatic cancer (Press release, Amplia Therapeutics, MAR 25, 2020, View Source [SID1234555975]).

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The designation means that Amplia will qualify for waived FDA fees, clinical trial protocol assistance and other incentives. Furthermore, if FDA ultimately approve AMP945 for the treatment of pancreatic cancer, Amplia would qualify for seven years’ market exclusivity in FDA-administered markets.

Amplia is aiming to start a Phase 1 clinical trial of AMP945 in healthy volunteers later this year to confirm that, like other FAKi, it is well tolerated. If this trial is successful, Amplia plans to initiate its first Phase 2 clinical study of AMP945 in cancer patients in 2021. AMP945 has multiple modes of action that make it an appealing candidate for incorporation into treatment regimes for hard-to-treat solid cancers such as pancreatic, ovarian, breast and lung.

Commenting on the Orphan Drug Designation, Professor Paul Timpson, a member of Amplia’s Scientific Advisory Board noted that "Pancreatic cancer is a seriously unmet medical need and FDA’s designation of AMP945 as an orphan-drug underlines global regulatory agencies’ interest in supporting novel treatments for this deadly disease".

This ASX announcement was approved and authorised for release by the Board of Amplia Therapeutics

On March 25, 2020 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a biotechnology company developing novel LAG-3-related immunotherapy treatments for cancer and autoimmune diseases, reported data from its Phase IIb AIPAC clinical trial in HER2-negative / Hormone Receptor positive (HR+) metastatic breast cancer (MBC) (Press release, Immutep, MAR 25, 2020, View Source [SID1234555945]).

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AIPAC is a multicentre, placebo-controlled, double-blind, randomised study evaluating the Company’s lead product candidate, eftilagimod alpha (efti, LAG-3Ig or IMP321) in combination with paclitaxel, a taxane standard of care chemotherapy, in 227 MBC patients to boost the T-cell immune responses against tumours.

63% of patients who received paclitaxel plus efti were progression-free at the 6-month landmark (at the end of the chemo-immunotherapy combination phase) and according to RECIST 1.1 based on blinded independent central readers (BICR). This compares favourably to 54% of patients who received paclitaxel plus placebo. The PFS data yielded an unadjusted hazard ratio (HR) of 0.93. The secondary endpoint of Overall Response Rate (ORR) increased to 48.3% in the efti group, from 38.4% in the placebo group.

Favourable results were reported in multiple predefined patient subgroups, e.g.:

patients with low monocytes count at baseline had a positive HR of 0.61 (median PFS of 5.45 vs. 7.29 months) favouring efti

patients with a more aggressive, more immunogenic luminal B type had a positive HR of 0.65 (median PFS of 5.45 vs. 7.29 months) favouring efti

patients with lower general performance status at baseline had a positive HR of 0.76 (median PFS of 6.67 vs. 7.13 months) favouring efti

The combination of efti and paclitaxel chemotherapy was overall safe and well tolerated, further building upon efti’s strong safety profile to date.

Immutep Limited, Level 12, 95 Pitt Street, Sydney NSW 2000

ABN: 90 009 237 889

These results, together with consideration of the Overall Survival (OS) and the immuno-monitoring data expected to be reported later this year, could allow the Company to build a platform to commence planning for a phase III clinical trial of efti in metastatic breast carcinoma. This will include the hypotheses for the primary and secondary endpoints and the appropriate stratification for different patient subgroups. Immutep will advance its discussions with the regulatory authorities accordingly.

Immutep CSO and CMO, Dr Frederic Triebel said: "The PFS results reported from the AIPAC study in a randomised clinical setting indicate an overall trend for clinical efficacy in HR-positive metastatic breast carcinoma, which is not a particularly immunogenic tumor and very interesting results in some meaningful subgroups. We would like to thank the patients and their families, along with the investigators for participating in this important study."

Immutep CEO, Marc Voigt stated: "We are pleased to see a clinical benefit for patients receiving efti in HR-positive metastatic breast carcinoma in multiple patient subgroups. This is just one of the four advanced solid tumor indications targeted in our efti clinical development program. We are pleased to see an efficacy trend and will carefully assess the data for further development of efti given our positive data in patient subgroups. More AIPAC results should become available in the coming months, including immuno-monitoring data and Overall Survival data."

"Another piece of evidence for efti efficacy in cancer patients comes from the interim results we are seeing from our ongoing phase II TACTI-002 trial of efti. In TACTI-002, 47% of first line non-small cell lung cancer patients responding to the combination treatment of efti with pembrolizumab, an anti-PD-1 therapy. These results are remarkable given that usually only 20% of patients respond to pembrolizumab monotherapy in this PDL-1 all comer trial indication."

AIPAC Principal Investigator, Hans Wildiers of University Hospitals Leuven, Leuven, Belgium, said: "I am pleased to see this innovative chemo-immunotherapy approach in AIPAC is well tolerated by patients, while showing a numerically (non-significant) higher progression free survival rate compared to chemotherapy alone. Hormone receptor positive breast tumours represent the majority of metastatic breast cancer patients and any active well tolerated adjunct to the weekly paclitaxel standard of care regimen, is desirable. This combination warrants further investigation in larger and more biomarker selected populations."

Further Reporting from AIPAC

The Company expects to present these clinical results in more detail at an upcoming conference. Patients are continuing in the follow up phase of the study enabling OS data to be collected. Immutep expects to report OS data in late-2020.

Webcast Details

Immutep will present this AIPAC data in a global webcast, details are as follows:

Immutep Limited, Level 12, 95 Pitt Street, Sydney NSW 2000

ABN: 90 009 237 889

Date & Time: Thursday, March 26th, 8am Australian Eastern Daylight Time / Wednesday, March 25th, 5pm US Eastern Daylight Time
Register: Interested parties can register via a link to the webcast on the Company’s website or via the following link: View Source
Questions: Investors are invited to submit questions in advance via [email protected].
A replay of the webcast will also be available at www.immutep.com from the day after the event.

About the AIPAC trial

Active Immunotherapy PAClitaxel (AIPAC) is a Phase IIb clinical trial in HER2-negative/ HR positive metastatic breast cancer. Based on Immutep’s LAG-3 technology, the study evaluates the combination of the Company’s lead product candidate, eftilagimod alpha (efti, LAG-3Ig or IMP321), and a taxane chemotherapy, called paclitaxel, as an immunotherapy. This combination is aimed at boosting the immune response against tumour cells compared to chemotherapy alone. In AIPAC, 227 hormone receptor positive metastatic breast cancer patients are randomised 1:1 to treatment A (paclitaxel chemotherapy plus placebo) or treatment B (paclitaxel chemotherapy plus eftilagimod alpha) for six months.

Patients receive weekly paclitaxel at Days 1, 8 and 15 with either efti of placebo injected subcutaneously, on Days 2 and 16 of each 4-week cycle, repeated for 6 cycles. Thereafter, patients pass over to the maintenance phase with efti alone.

The primary end point is to determine progression-free survival (PFS) and key secondary objectives include overall survival, safety, quality of life and objective response rate.

For more information regarding the AIPAC trial, visit clinicaltrials.gov (identifier NCT02614833) and View Source).

On March 25, 2020 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage biopharmaceutical company, reported that it will report financial results for the year ended December 31, 2019 and host a conference call on Friday, March 27, 2020 (Press release, Altimmune, MAR 25, 2020, View Source [SID1234555878]).

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Conference Call Details
Date: Friday, March 27
Time: 8:30 am Eastern Time
Domestic: 877-423-9813
International: 201-689-8573
Conference ID: 13701071
Webcast: View Source