On April 15, 2019 Trillium Therapeutics Inc. (Nasdaq/TSX: TRIL), a clinical-stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported the publication of data highlighting the role of TTI-621 in treating patients with Sézary syndrome (SS), a form of cutaneous T-cell lymphoma (CTCL) (Press release, Trillium Therapeutics, APR 15, 2019, View Source [SID1234535141]).

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The paper titled "Targeting CD47 in Sézary syndrome with SIRPαFc", published in the April 9th issue of Blood Advances, demonstrates that TTI-621 (SIRPα-IgG1 Fc) triggers macrophage-mediated phagocytosis of Sézary cells and reduces tumor load in SS patients following intravenous administration. Four of five heavily pre-treated SS patients had a decrease in the dominant malignant clone and other markers of tumor burden after a single infusion of TTI-621. "We continue to be excited by the monotherapy data emerging from our TTI-621 clinical studies", stated Dr. Niclas Stiernholm, president and CEO of Trillium Therapeutics. "The results in this manuscript provide further support for investigating the therapeutic potential of our novel CD47-blocking agent in CTCL patients".

On April 15, 2019 Aqilion reported that searches for early innovative pharmaceutical projects thatare based on solid research resting on a firm biological foundation and offering both clinical relevance and patient benefit (Press release, Aqilion, APR 15, 2019, View Source [SID1234568119]). Immunscape has developed innovative molecules that could potentially become new drugs for the treatment of cancer and autoimmune diseases. The project is currently in an early phase of development. The purpose of the new collaboration is to jointly assess and develop the commercial potential of the project.

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Aqilion can invest resources in interesting projects to further evaluate their potential and to ensure that they proceed to a full-scale project or project company. Both Aqilion and Immunscape are actively engaged in the assessment in a collaboration that entails an exclusive option for Aqilion to continue its involvement if the project proves to be promising.

"The pre-project phase is important to strengthen the common vision of the project and to assess together with Immunscape’s team whether Aqilion is the right partner to promote continued commercial development of the project. Immunscape has solid drug discovery experience and expertise and the company’s innovative molecule platform is extremely interesting for Aqilion. We look forward to working together on this exciting project," says Sarah Fredriksson, CEO of AQILION AB.

"We are extremely pleased to have initiated this collaboration with Aqilion, which will accelerate our drug discovery efforts," says Lars Öhman, CEO of Immunscape AB.

On April 15, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX) reported that the U.S. Food and Drug Administration (FDA) granted orphan drug designation to its phosphoinositide-3-kinase (PI3K) delta inhibitor, umbralisib (TGR-1202), for the treatment of patients with all three types of marginal zone lymphoma (MZL): nodal, extranodal, and splenic MZL (Press release, TG Therapeutics, APR 15, 2019, View Source [SID1234535144]).

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Umbralisib monotherapy is being evaluated in the UNITY-NHL Phase 2b registration directed clinical trial. The MZL cohort of the UNITY-NHL trial is currently evaluating the safety and efficacy of single agent umbralisib in patients with MZL who have received at least one prior anti-CD20 regimen, the same indication for which the FDA recently granted breakthrough therapy designation for umbralisib.

Michael S. Weiss, Executive Chairman and Chief Executive Officer of TG Therapeutics, stated "Receiving orphan drug designation for umbralisib in patients with MZL is another important milestone in our commitment to developing novel treatment options for patients with B-cell malignancies, including orphan diseases such as MZL." Mr. Weiss continued, "We are highly encouraged by the interim results presented thus far for the MZL cohort of the UNITY-NHL trial and we look forward to presenting final data from this cohort later this year and to discussing the results with the FDA with the goal of filing for accelerated approval of umbralisib by year-end."

ABOUT ORPHAN DRUG DESIGNATION

Orphan drug designation is granted by the U.S. FDA to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain incentives which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. If a product that has orphan drug designation subsequently receives the first FDA approval for the disease for which it has such designation, the product is entitled to orphan product exclusivity.

ABOUT MARGINAL ZONE LYMPHOMA

Marginal zone lymphoma (MZL) comprises a group of indolent (slow growing) B-cell non-Hodgkin lymphomas (NHLs) that begin forming in the marginal zone of lymphoid tissue. With an annual incidence of approximately 7,500 newly diagnosed patients, MZL is the third most common B-cell NHL accounting for approximately eight percent of all NHL cases. MZL consists of three different subtypes: extranodal MZL, nodal marginal zone lymphoma (NMZL), and splenic marginal zone lymphoma (SMZL).

ABOUT THE UNITY-NHL PHASE 2b STUDY—Marginal Zone Lymphoma Cohort

The multicenter, open-label, UNITY-NHL Phase 2b study – Marginal Zone Lymphoma cohort was designed to evaluate the safety and efficacy of single agent umbralisib in patients with MZL who have received at least one prior anti-CD20 regimen. The primary endpoint is overall response rate (ORR) as determined by Independent Review Committee (IRC) assessment. Secondary endpoints include safety, duration of response, and progression-free survival (PFS).

The MZL cohort completed enrollment in August 2018, and in February of 2019 the Company announced that the primary endpoint of ORR as determined by central IRC was met for all treated patients (n=69).

Earlier this month at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, interim data from the UNITY-NHL MZL cohort were presented on the first 42 patients enrolled, demonstrating an ORR of 52%, with a Complete Response (CR) rate of 19%, and a tolerable safety profile amongst all patients treated to date (n=69).

On April 12, 2019 Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) reported that the Company will report its financial results for the first quarter ended March 31, 2019 before the US financial markets open on April 25, 2019 (Press release, Alexion, APR 12, 2019, View Source [SID1234535112]). Following the release of the financial results, Alexion management will conduct a conference call and audio webcast at 8:00 a.m. Eastern Time (ET).

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To participate in this conference call, dial 866-762-3111 (USA) or 210-874-7712 (International), conference ID 1692605 shortly before 8:00 a.m. ET. The audio webcast can be accessed on the Investor page of View Source and an archived version will be available for a limited time following the presentation.

On April 12, 2019 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer and, through our license to Santen Pharmaceutical Co. Ltd., wet age-related macular degeneration, reported that its Phase 3 TAPPAS trial evaluating TRC105 in combination with Votrient (pazopanib) in patients with advanced or metastatic angiosarcoma was terminated for futility based on the recommendation of the Independent Data Monitoring Committee (IDMC) following its review of interim unblinded safety and efficacy data from more than 120 patients enrolled in the trial at the time of the analysis (Press release, Tracon Pharmaceuticals, APR 12, 2019, View Source [SID1234535113]).

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TRACON will work with investigators to appropriately conclude the study in a manner consistent with the best interests of each patient. Data from this study will be analyzed and submitted for presentation at an upcoming scientific congress.

"We are disappointed that TRC105 in combination with Votrient did not demonstrate clinically meaningful efficacy in patients with advanced or metastatic angiosarcoma," said Charles Theuer, M.D. Ph.D., President and CEO of TRACON. "Given these data, we will terminate further enrollment in company sponsored trials of TRC105 in oncology. We will continue to support our partner, Santen, in their development of DE-122 in wet AMD, where the anti-angiogenic and anti-fibrotic properties of endoglin inhibition may be more relevant than in oncology. We will also continue to develop our other drug candidates, including TRC253 in partnership with Janssen and TJ004309 (also known as TJ-D5) in partnership with I-Mab, and, intend to advance candidates within I-Mab’s broad bispecific pipeline into the clinic in the US as early as the beginning of next year. We will also continue our business development efforts to source additional innovative products to fortify our pipeline through risk share and cost share arrangements. On a financial note, as a result of the expected savings to be generated from terminating TRACON sponsored trials of TRC105, we anticipate our current cash runway will now extend into the third quarter of 2020."

Investor Conference Call

The Company will hold a conference call today at 8:30 a.m. EDT / 5:30 a.m. PDT to provide further details on the TAPPAS trial and TRACON’s clinical stage pipeline. The dial-in numbers are (877) 407‑9039 for domestic callers and (201) 689-8470 for international callers. Please use passcode 13689814. A live webcast of the conference call will be available at View Source

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About the TAPPAS trial in Advanced or Metastatic Angiosarcoma

The TAPPAS trial was designed to compare treatment with TRC105 and Votrient to treatment with single agent Votrient in up to 340 patients with advanced or metastatic angiosarcoma. Patients were randomized in equal numbers and the primary endpoint was progression free survival by RECIST 1.1. Key secondary endpoints included objective response rate, overall survival, safety and tolerability.

About TRC105 (carotuximab)

TRC105, the oncology formulation of carotuximab, is a novel, clinical stage antibody to endoglin, a protein overexpressed on proliferating endothelial cells that is essential for angiogenesis, the process of new blood vessel formation. TRC105 has received orphan designation for the treatment of soft tissue sarcoma in both the US and EU. The ophthalmic formulation of TRC105, DE-122, is currently being studied in the randomized Phase 2 AVANTE trial in patients with wet AMD. For more information about the clinical trials, please visit TRACON’s website at www.traconpharma.com/clinical_trials.php.

About DE-122 (carotuximab)

DE-122, a novel ophthalmic formulation of carotuximab, is active in preclinical choroidal neovascularization (CNV) models and expected to enhance the effect of approved VEGF inhibitors used to treat wet AMD. DE-122 is being investigated in the Phase 2 randomized AVANTE trial assessing the efficacy and safety of intravitreal injections in combination with Lucentis (ranibizumab) compared to Lucentis monotherapy in patients with wet AMD.