10-Q – Quarterly report [Sections 13 or 15(d)]

Aileron Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Aileron Therapeutics, 2017, AUG 10, 2017, View Source [SID1234522297]).

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10-Q – Quarterly report [Sections 13 or 15(d)]

Sophiris Bio has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Sophiris Bio, AUG 10, 2017, View Source [SID1234520159]).

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TG Therapeutics, Inc. Announces Successful Outcome from the First Pre-Planned Interim Analysis by Independent DSMB of the DLBCL Cohort in the UNITY-NHL Phase 2b Trial

On August 10, 2017 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that the independent Data Safety Monitoring Board (DSMB) of the UNITY-NHL Phase 2b registration directed trial has successfully completed the first pre-specified interim analysis to evaluate the Overall Response Rate (ORR) in the cohort enrolling patients with relapsed or refractory Diffuse Large B-cell Lymphoma (DLBCL) that are not eligible for high-dose chemotherapy or transplant (Press release, TG Therapeutics, AUG 10, 2017, View Source [SID1234520157]). Upon review of the available ORR data, based on pre-specified efficacy thresholds of ORR, the DSMB recommended the Company cease enrollment into the single agent TGR-1202 arm, while continuing enrollment into the TG-1101 + TGR-1202 arm which has demonstrated an acceptable level of efficacy to warrant continued evaluation. Per the UNITY-NHL protocol, the single agent TGR-1202 arm will be replaced by an arm evaluating the triple combination arm of TG-1101, TGR-1202, and bendamustine.

Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer stated, "The DLBCL cohort in our UNITY-NHL trial was designed to evaluate the contribution of TGR-1202 in our combination ‘U2’ regimen. We are extremely pleased that the DSMB has recommended continued enrollment in the U2 arm, while allowing us to proceed with replacing the single agent TGR-1202 arm with the triple combination of TG-1101, TGR-1202, and bendamustine (also referred to as U2 + Benda) as planned. As recently presented, the triplet combination of U2 + Benda was highly active, with a 50% ORR in patients with refractory DLBCL and a 100% ORR in relapsed DLBCL patients. We have long believed that patients with aggressive DLBCL in particular are best treated with combination therapy rather than single agents and are pleased to see our UNITY-NHL study advancing to the next level."

ABOUT UNITY-NHL PHASE 2b TRIAL

UNITY-NHL is a global Phase 2bclinical trial evaluating TG-1101 and TGR-1202, ("U2"), in patients with various types of B-cell lymphoma. The trial consists of three independent cohorts enrolling patients with relapsed or refractory Diffuse Large B-cell Lymphoma (DLBCL), Follicular Lymphoma (FL), Small Lymphocytic Lymphoma (SLL), and Marginal Zone Lymphoma (MZL). The study is evaluating the U2 combination and the combination of U2 + bendamustine in patients with DLBCL; TGR-1202 monotherapy and in the U2 combination in patients with FL and SLL; and TGR-1202 monotherapy in patients with MZL.

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Takeda and Shattuck Labs Announce Research Collaboration to Explore Agonist Redirected Checkpoint Fusion Proteins in Immuno-Oncology

On August 10, 2017 Takeda Pharmaceutical Company Limited (TSE: 4502) and Shattuck Labs, Inc. reported that they have entered into a research collaboration to explore and develop checkpoint fusion proteins that have the potential to become highly differentiated, next-generation immunotherapies (Press release, Shattuck Labs, AUG 10, 2017, View Source [SID1234520210]). Under the terms of the collaboration agreement, Takeda will hold options for exclusive global development and commercialization rights for up to four molecules resulting from the collaboration.

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Shattuck Lab’s Agonist Redirected Checkpoint (ARC) platform combines two binding domains to create fusion proteins that potentially restore and enhance immune system function in a single construct. The ARC molecules block checkpoint molecules while simultaneously stimulating TNF superfamily co-stimulatory receptors on T-cells and innate cells, which are targets controlling the immune system and often dysregulated in cancers, thereby enabling combination immunotherapy with a single product.

"Shattuck Labs has pioneered the unique ARC platform, and we are excited about the opportunity this collaboration presents to develop groundbreaking, next-generation immuno-oncology treatments," said Christopher Arendt, Ph.D., Head, Oncology DDU & Immunology Unit, Takeda. "Research partnerships are a key aspect of our continued dedication to oncology innovation, and this collaboration will bring us closer to our goal of discovering, developing and delivering breakthrough oncology therapies."

The collaboration will include two pre-clinical and four discovery stage programs. Takeda will provide funding for pre-clinical and clinical development and will have the option to take an exclusive license to further develop and commercialize up to four ARC molecules resulting from the collaboration. Additional terms of the collaboration are not disclosed.

"Takeda shares our passion and mission to develop and advance novel therapies in oncology, with the goal of achieving better clinical outcomes in patients," said Josiah Hornblower, Chairman and CEO of Shattuck Labs. "We are very excited to work with Takeda, a proven leader in oncology, to continue to validate the ARC technology."

Takeda signed the collaboration agreement through its wholly-owned subsidiary, Millennium Pharmaceuticals, Inc.

About Takeda Pharmaceutical Company

Takeda Pharmaceutical Company Limited is a global, research and development-driven pharmaceutical company committed to bringing better health and a brighter future to patients by translating science into life-changing medicines. Takeda focuses its R&D efforts on oncology, gastroenterology and central nervous system therapeutic areas plus vaccines. Takeda conducts R&D both internally and with partners to stay at the leading edge of innovation. New innovative products, especially in oncology and gastroenterology, as well as Takeda’s presence in Emerging Markets, are currently fueling the growth of Takeda. More than 30,000 Takeda employees are committed to improving quality of life for patients, working with Takeda’s partners in health care in more than 70 countries. For more information, visit www.takeda.com/news.

About Shattuck Labs

Headquartered in Austin, Texas, Shattuck Labs, Inc. is a biopharmaceutical company committed to developing biologic medicines for oncology and inflammatory disease. Shattuck Labs developed the Agonist Redirected Checkpoint (ARC) platform to enable immuno-oncology therapeutics capable of simultaneously blocking immune checkpoints and stimulating TNF superfamily receptors. Shattuck Lab’s state-of-the-art research and development organization is currently advancing multiple lead products toward clinical trials.

Syndax Pharmaceuticals Reports Second Quarter 2017 Financial Results and Provides Clinical and Business Update

On August 10, 2017 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq:SNDX), a clinical stage biopharmaceutical company developing entinostat and SNDX-6352 in multiple cancer indications, reported its financial results for the second quarter ended June 30, 2017 (Filing, Q2, Syndax, 2017, AUG 10, 2017, View Source [SID1234520162]). In addition, the Company provided a clinical and business update. As of June 30, 2017, Syndax had $130.0 million in cash, cash equivalents and short-term investments.

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Enrollment in all cohorts of ENCORE 601 have proceeded rapidly. The second stage of the ENCORE 601 cohort enrolling PD-1 (programmed death receptor-1) refractory melanoma patients has reached its accrual target. Enrollment in the PD-1 refractory non-small cell lung cancer (NSCLC) cohort remains on track, and is expected to be complete in the fourth quarter. The Company also announced that the first patient has been dosed in the ENCORE 601 cohort enrolling microsatellite stable colorectal cancer (CRC) patients.
"The second quarter was marked by continued progress across our clinical pipeline. The initial results from ENCORE 601 provide the first clinical evidence that entinostat may reverse resistance to immune checkpoint therapies," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "We had a productive Type B meeting with the FDA in June to discuss potential registration paths for entinostat when used in combination with a PD-1 inhibitor for the treatment of melanoma patients who have progressed on checkpoint therapy. We are now evaluating the options discussed with the FDA and will provide an update by the end of the year.
Pipeline Updates

• ECOG-ACRIN Cancer Research Group, sponsor of E2112, the Phase 3 registration trial of entinostat plus exemestane in advanced HR+, HER2- breast cancer, expects the progression free survival analysis to be available in the first half of 2018 directly following completion of enrollment. As of the end of July, the trial was 78% enrolled.


A number of patients enrolled in the ENCORE 601 cohort of NSCLC patients naïve to PD-(L)1 therapy remain on drug, with a determination on whether the cohort has satisfied the pre-specified efficacy criteria for advancement to the second stage of the trial expected by year end. The pre-specified objective response threshold to advance this cohort into the second stage of the Phase 2 trial has been revised to ³ 4 responses out of 17 (versus the previous benchmark of ³ 3 responses out of 13) in order to incorporate Phase 1b patients dosed at the recommended 5 mg dose of entinostat. Similarly, the cohort of PD-(L)1 refractory NSCLC patients, announced in May to have met the pre-specified objective response threshold to advance into the second stage of

the Phase 2 trial, used revised criteria requiring ³3 responses out of 31 (versus the previous benchmark of >2 responses out of 20) in order to incorporate Phase 1b patients dosed at entinostat 5 mg.

• Enrollment in the second stage of the PD-(L)1 refractory melanoma cohort of ENCORE 601 has completed, and the second stage of the PD-(L)1 refractory NSCLC cohort of ENCORE 601 is expected to complete enrollment in the fourth quarter. Syndax expects to present data from stage one of the NSCLC cohorts, as well as biomarker data from the melanoma cohort, at the Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in the fall, and the full trial data from both the refractory NSCLC and melanoma cohorts at a medical congress in the first half of 2018.

• Dosing has been initiated in the ENCORE 601 cohort of patients with microsatellite stable CRC. This new cohort, announced in April 2017, employs a Simon two-stage design enrolling 13 patients in the first stage, and requires ³ 2 confirmed objective responses to proceed to the second stage. A decision on whether to advance to the second stage of the CRC cohort is expected in the first half of 2018.

• Enrollment is also progressing in the two additional trials aimed at exploring the ability of entinostat to enhance the efficacy of PD-L1 inhibitor therapies, ENCORE 602 and ENCORE 603. ENCORE 602, the Phase 1b/2 clinical trial evaluating the combination of entinostat plus Genentech’s PD-L1 inhibitor, TECENTRIQ, in patients with triple negative breast cancer, is expected to complete enrollment in the Phase 2 portion by the end of the fourth quarter. ENCORE 603, the Phase 1b/2 clinical trial evaluating entinostat in combination with Pfizer/Merck KGaA’s BAVENCIO in patients with ovarian cancer, recently began enrolling patients into the Phase 2 portion and is expected to complete enrollment in the first half of 2018.

• Enrollment is now complete in the Phase 1 single ascending dose (SAD) clinical trial of SNDX-6352 in healthy volunteers to determine the safety, pharmacokinetics and pharmacodynamics of the anti-CSF-1R monoclonal antibody. The Company expects to present data from this study at a scientific congress in the fourth quarter of 2017. Syndax also plans to initiate a multiple ascending dose (MAD) trial in cancer patients in the third quarter of 2017.
Syndax Expects to Participate in the Following Upcoming Investor Conferences

• Citi 12th Annual Biotech Conference, September 6-7, 2017 in Boston

• Morgan Stanley Healthcare Conference, September 11-13, 2017 in New York

• Cantor Fitzgerald 2017 Global Healthcare Conference, September 25-27, 2017 in New York
Second Quarter 2017 Financial Results
As of June 30, 2017, Syndax had cash, cash equivalents and short-term investments of $130.0 million and 22,219,843 shares issued and outstanding. This includes aggregate net proceeds of $48.7 million, raised in a follow-on offering in May 2017 whereby 3,950,190 shares of common stock were sold at an offering price of $13.25 per share.
Second quarter 2017 research and development expenses increased to $9.9 million from $6.1 million for the comparable period in the prior year due to increases in clinical trial activities of $2.3 million, employee compensation expense of $0.8 million, and legal and consultant expenses of $0.6 million. The increase in clinical trial activities was primarily due to increases in spending related to the additional cohorts added to ENCORE 601, increased activities in ENCORE 602 and ENCORE 603, start-up costs related to SNDX-6352, our Phase 1 clinical pharmacology trials and CMC activities. The increase in employee compensation costs was primarily due to increased headcount.
General and administrative expenses totaled $4.3 million during the second quarter of 2017 compared with $2.8 million in the comparable period in the prior year. The increase in general and administrative expenses was primarily due to an increase in employee compensation of $1.0 million combined with increases in consulting expenses of $0.4 million. The increase in employee compensation of $1.0 million was primarily due to increases in non-cash stock-based compensation of $0.6 million, combined with an increase in salary expense of $0.4 million due to increased headcount.
For the three months ended June 30, 2017, Syndax reported a net loss attributable to common stockholders of $13.6 million, or $0.70 per share, compared to $8.4 million, or $0.47 per share, for the comparable prior year period.
Financial Guidance
Today the Company provided operating expense guidance for the third quarter and full year 2017. For the third quarter and full year 2017, research and development expenses are expected to be $12.0 to $14.0 million and $46.0 to $51.0 million, respectively, and total operating expenses are expected to be $16.0 to $18.0 million and $63.0 to $68.0 million, respectively.