On October 30, 2019 vTv Therapeutics Inc. (Nasdaq:VTVT) reported financial results for the third quarter that ended September 30, 2019, and provided an update on recent achievements, events, and its ongoing clinical trials in type 1 diabetes and Alzheimer’s disease (Press release, vTv Therapeutics, OCT 30, 2019, View Source [SID1234550092]).

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"We hosted an informative type 1 diabetes key opinion leader event with JDRF this quarter where two prominent KOLs, Drs. John Buse and Kevan Herold, shared important information including that, despite the increasingly widespread use of improved technologies like continuous glucose monitoring, nearly 80% of individuals with type 1 diabetes fail to achieve the American Diabetes Association’s target goals for HbA1c," said Steve Holcombe, chief executive officer, vTv Therapeutics. "Based on the data we’ve seen to date in our trial of TTP399 in patients with type 1 diabetes, we believe our glucokinase activator could eventually provide a meaningful treatment option to patients with type 1 diabetes that would lower A1c, improve glycemic control and reduce daily insulin use, without increasing hypoglycemic events."

"We are also pleased that we are continuing to make progress with the Elevage Study of azeliragon in patients with mild Alzheimer’s disease with type 2 diabetes that is expected to read-out in late 2020 or early 2021."

Recent Achievements and Outlook

Type 1 Diabetes Study

Completed enrollment for Part 2 of the Simplici-T1 Study. In September 2019, we completed enrollment of patients in part 2 of the phase 2 Simplici-T1 Study, a 12-week study to evaluate TTP399 as an adjunct to insulin therapy for patients with type 1 diabetes. We expect to report results in the latter part of the first quarter of 2020.

Presented Additional Positive Data from Part 1 of the Simplici-T1 Study. In September 2019, we presented positive continuous glucose monitor (CGM) and insulin dose data from the patients with complete CGM profiles from part 1 of the Simplici-T1 Study at the 55th Annual Meeting of the European Association for the Study of Diabetes. In addition to the previously presented topline improvement in HbA1c of 0.7% relative to placebo, key results from this presentation included:

Patients taking TTP399 treatment (n=6) had increased "Time in Range" from baseline to end of treatment by 11% (2.7 hours) (p=0.055) per day (24 hours), and by 12% (1.7 hours) (p=0.04) during the critical waking hours (7am-9pm) relative to placebo (n=9).

Patients taking TTP399 treatment reduced the total daily mealtime bolus insulin dose by 23% compared to 4% for placebo while significantly improving glycemic control.

Patients in the TTP399 treatment group experienced fewer Level 1 (≥54-70 mg/dl) and Level 2 (<54 mg/dl) hypoglycemic events than patients in the placebo group.

Alzheimer’s Disease Study

Enrollment continues for Phase 2/3 Elevage Study of azeliragon. We began enrolling patients in the phase 2 part of the Elevage Study to evaluate the efficacy and safety of azeliragon in patients with mild Alzheimer’s disease (AD) and type 2 diabetes.

The Elevage Study is designed to confirm the positive post-hoc subgroup results from the previous phase 3 study of azeliragon in patients with mild Alzheimer’s disease in which the subgroup of AD patients with type 2 diabetes (defined by an HbA1c of greater than 6.5% at any time in the study) treated with azeliragon demonstrated a potential benefit in both cognition and function relative to those treated with placebo.

The phase 2 part of the Elevage Study will evaluate the efficacy and safety of azeliragon in patients with mild Alzheimer’s disease (screening MMSE 21 to 26, baseline MMSE 19 to 27; and ADAS-cog14 score ≥10) and type 2 diabetes (screening HbA1c 6.5% to 9.5%, inclusive). We expect to report topline results from the phase 2 proof of concept study by late 2020 or early 2021.

Recent Events

Hosted Key Opinion Leader (KOL) Event to Discuss the Type 1 Diabetes Landscape and Emerging Therapies as Adjuncts to Insulin. In October, vTv hosted a KOL event to provide an opportunity to learn more about type 1 diabetes. Dr. John Buse, UNC-CH; Dr. Kevan Herold, Yale University; Dr. Esther Latres, JDRF International; and Dr. Carmen Valcarce, vTv Therapeutics, presented to a diverse audience of analysts, investors, and members of the type 1 diabetes community. The KOLs reported that patients with type 1 diabetes have a serious, unmet need for an adjunct to insulin treatment.

An archived version of the event can be found here.

Second Quarter 2019 Financial Results

Cash Position: Cash and cash equivalents as of September 30, 2019, were $2.4 million compared to $1.5 million as of June 30, 2019.

Revenue: Revenues were not material for the third quarter of 2019 versus $1.8 million in the second quarter of 2019. Revenue recognized in the second quarter of 2019 was due to a milestone recognized under our license agreement with Newsoara and the continued amortization of revenue from our license agreement with Reneo over the transfer services period.

R&D Expenses: Research and development expenses were $3.7 million in the third quarter of 2019 and $4.2 million in the second quarter of 2019. The decrease in these costs

was driven primarily by the decreases in costs for the Elevage Study. Such costs were higher in the second quarter of 2019 as we were performing startup activities related to the sites and initiation of patient enrollment.

G&A Expenses: General and administrative expenses were $1.8 million and $2.4 million in each of the third and second quarters of 2019, respectively. The decrease in these costs is related to the recognition of a gain on the sale of laboratory equipment in the third quarter of 2019 as well as overall lower spending in the quarter.

Net Loss Before Non-Controlling Interest: Net loss before non-controlling interest was $6.0 million for the third quarter of 2019 compared to net loss before non-controlling interest of $5.1 million for the second quarter of 2019.

Net Loss Per Share: GAAP net loss per share was $0.13 and $0.10 for the three months ended September 30, 2019 and June 30, 2019, respectively, based on weighted-average shares of 32.1 million and 28.0 million for the three month periods ended September 30, 2019 and June 30, 2019, respectively. Non-GAAP net loss per fully exchanged share was $0.10 and $0.09 for the three months ended September 30, 2019 and June 30, 2019, respectively, based on non-GAAP fully exchanged weighted-average shares of 55.2 million and 51.1 million for the three months ended September 30, 2019 and June 30, 2019, respectively.

On October 30, 2019 Athenex, Inc. (Nasdaq: ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported that it has received Orphan Designations from the European Commission (EC) for paclitaxel and encequidar ("Oral Paclitaxel") for the treatment of soft tissue sarcoma, following a positive opinion from the European Medicines Agency (EMA) (Press release, Athenex, OCT 30, 2019, View Source [SID1234573893]).

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Dr. Rudolf Kwan, Chief Medical Officer of Athenex, commented, "We are very pleased to receive Orphan Designations from the European Commission, as this further recognizes the potential benefits of Oral Paclitaxel. Earlier this year, we shared encouraging early results from a clinical study in angiosarcoma, a form of soft tissue sarcoma, that showed rapid responses in a difficult to treat patient population. We will soon be extending this study to include sites in Europe."

The EC grants Orphan Designation status to support development of medicines for the treatment of diseases that affect fewer than 5 in 10,000 people in the European Union and provide a significant benefit to those affected by the condition. Orphan Designation may provide certain benefits, including protocol assistance, fee reductions, and a ten-year period of market exclusivity if the drug is approved.

The U.S. FDA has granted oral paclitaxel and encequidar an Orphan Drug Designation in the treatment of angiosarcoma.

The Orascovery platform was initially developed by Hanmi Pharmaceuticals and licensed exclusively to Athenex for all major worldwide territories except Korea, which is retained by Hanmi.

On October 20, 2019 NexImmune, a clinical stage immunotherapy company developing novel T cell therapies, reported that it received Investigational New Drug (IND) clearance for the company’s first cellular therapy product (Press release, NexImmune, OCT 30, 2019, View Source [SID1234554876]). NEXI-001 is being developed for the treatment of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) patients with relapsed disease after an allogeneic hematopoietic cellular transplant (allo-HCT). Scott Carmer, NexImmune’s President and CEO, commented "we are excited to initiate clinical trials with NEXI-001, and to provide these patients with a promising new treatment option. NEXI-001 is a product that is meaningfully differentiated from other cellular therapies in that it contains multiple populations of antigen specific endogenous T cells with enhanced anti-tumor properties. Because of this, we are confident NEXI-001 could address key limitations observed with other cellular immunotherapies; specifically, tumor escape through single target down-regulation and tumor relapse due to diminished T cell persistence."

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The Phase 1/2 trial of NEXI-001 will begin enrolling patients at clinical sites across the United States, including Memorial Sloan Kettering, MD Anderson, The City of Hope, The Dana Farber Cancer Institute, and AdventHealth. The trial is a multi-center, dose-escalating, open-label, single-arm study evaluating the safety, tolerability and initial efficacy of adoptively-transferred donor-derived T-cells as a treatment for AML or MDS patients with relapsed disease after allo-HCT. The trial will evaluate two increasing dose levels, with three patients enrolled in each dose escalation cohort. After the initial safety evaluation phase, up to 20 patients will be enrolled in the dose expansion phase. All patients will be followed for at least one year.

Miguel Perales, MD, Deputy Chief of Bone Marrow Transplant Services at Memorial Sloan Kettering Cancer Center and the trial’s lead investigator, stated "for patients receiving T cell therapy after allo-HCT, the primary treatment goal is to separate the efficacy benefits of Graft-versus-Leukemia (GVL) from the toxicities associated with Graft-versus-Host Disease (GVHD). This requires the ability to deliver a cell therapy product that contains a significant proportion of leukemia antigen specific memory T cells, and few-to-no T cell subtypes with allo-reactive potential. We believe NEXI-001 represents a T cell product with potential to deliver against these key treatment goals. Our center is excited to participate in this clinical trial, and we are enthusiastic about the promise NEXI-001 represents for our post-transplant AML/MDS patients with relapsed disease."

Lee Greenberger, MD, Chief Scientific Officer at The Leukemia and Lymphoma Society, commented further, "patients who relapse after allo-HCT are left with a dismal prognosis and limited treatment options, and are in need of new treatments that offer significant benefit. LLS is proud to partner with NexImmune on the development of products like NEXI-001 through our Therapy Acceleration Program (TAP). We believe this unique technology has the potential to meaningfully benefit patients who are in urgent need of novel approaches to treating this disease."

About AML / MDS relapse after Allogeneic Hematopoietic Cellular Transplant
Among patients with Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS), allogeneic hematopoietic cellular transplantation (HCT) used as post-remission therapy for those with high-risk genetic profiles or as salvage therapy for those with disease refractory to chemotherapy offers the highest potential for long-term survival. However, relapse after allogenic HCT remains the major cause of treatment failure, is associated with an extremely poor prognosis, and represents a major therapeutic challenge. In this situation, no standard therapy is defined, but treatment options generally aim to reduce disease burden and to enforce a graft-versus-leukemia (GvL) effect. Commonly used treatment options for these patients are chemotherapy combined with cellular-based approaches, such as donor lymphocyte infusion (DLI)or second transplantation.

About NEXI-001
NEXI-001 is an endogenous cellular therapy and includes populations of primed antigen specific CD8+ cells directed at multiple leukemia relevant antigen targets. Generating T cells against multiple tumor targets minimizes the potential for tumor escape. In addition, NEXI-001 contains T cell subtypes that are predominantly memory phenotypes, with the majority being characterized as stem cell memory and central memory T cells. Importantly, each NEXI-001 product contains minimal populations of naïve T cells (average <5%). Minimizing the naïve T cell compartment reduces alloreactivity and Graft-versus-Host Disease (GvHD) in patients after an allo-HCT, while T cell products that contain high proportions of memory cells, particularly central memory and stem cell memory, have been associated with potent anti-tumor activity, long-term T cell persistence and durable anti-tumor response.

On October 30, 2019 ArQule, Inc. (Nasdaq: ARQL) reported its financial results for the third quarter of 2019 (Press release, ArQule, OCT 30, 2019, View Source [SID1234550016]).

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For the quarter ended September 30, 2019, the Company reported a net loss of $10.7 million, or $0.09 per basic share, compared with net loss of $5.6 million, or $0.05 per basic share, for the quarter ended September 30, 2018.

As of September 30, 2019, the Company had a total of approximately $174.1 million in cash, cash equivalents, and marketable securities.

Key Highlights from Q3, 2019

ARQ 531, our potent and reversible dual inhibitor of both wild-type and C481-mutant BTK:
Completed recruitment of the phase 1 dose escalation trial and determined a recommended phase 2 dose (RP2D) of 65 mg once a day
Submitted American Society of Hematology (ASH) (Free ASH Whitepaper) abstract in July, which is scheduled to be made public at 9:00 a.m. ET, November 6, 2019
Began dosing phase 2 expansion cohorts in multiple B cell malignancies at the RP2D
Requested and received a meeting date with the FDA to discuss registrational trial design
Miransertib, our potent and selective first-generation AKT inhibitor:
Initiated multiple additional sites in our previously described registrational trial, MOSAIC (Miransertib in Overgrowth Syndromes in Adults and Children), for both Proteus syndrome (PS) and PIK3CA-related Overgrowth Spectrum (PROS)
Recently announced the first patient dosed under the MOSAIC protocol at Texas Children’s Hospital (TCH) Vascular Anomalies Center
ARQ 751, our highly potent and selective next-generation AKT inhibitor:
Continued the signal generation work in genetically defined solid tumors
Derazantinib, our FGFR inhibitor, partnered with Basilea and Sinovant, in a registrational trial for intrahepatic cholangiocarcinoma:
Continued to interact with our partners, Basilea and Sinovant
Paolo Pucci, Chief Executive Officer of ArQule, commented, "We are very excited to present the final phase 1 data set for ARQ 531 at ASH (Free ASH Whitepaper) in December. These data will include meaningful updates on clinical activity and durability that provide a more complete picture of the potential of ARQ 531 and will reinforce its position as the leading reversible BTK inhibitor."

"The tremendous progress ARQ 531 has made in such a short period has allowed us to provide first ever proof of concept with a reversible BTK inhibitor in the emerging unmet medical need of C481-mutant CLL patients. We are now actively recruiting patients in the multi-arm phase 2 trial," commented Dr. Brian Schwartz, Chief Medical Officer of ArQule. "It is also very gratifying to have begun patient enrollment in our registrational MOSAIC trial in Proteus syndrome and PROS."

Revenues and Expenses

Revenues for the third quarter, 2019, were $0.2 million compared with revenues of $5.0 million for the third quarter, 2018.

Research and development expenses in the third quarter, 2019 were $8.3 million compared with $7.3 million for the third quarter, 2018.

General and administrative expenses in the third quarter, 2019 were $3.2 million compared with $3.4 million for the third quarter, 2018.

2019 Financial Guidance

For 2019, ArQule expects revenue to range between $2 and $5 million. Net loss is expected to range between $40 and $43 million, and net loss per share to range between $(0.35) and $(0.37) for the year. ArQule expects to end 2019 with approximately $160 million in cash and marketable securities which will support the current business plan into 2022.

Conference Call and Webcast

ArQule will hold its second quarter financial results call today, October 30, 2019 at 9:00 a.m. ET. The live webcast can be accessed in the "Investors and Media" section of our website, www.arqule.com, under "Events & Presentations." You may also listen to the call by dialing (877) 868-1831 within the U.S. or (914) 495-8595 outside the U.S. and entering the conference ID: 4289763. A replay will be available two hours after the completion of the call and can be accessed in the "Investors & Media" section of our website, www.arqule.com, under "Events and Presentations."

On October 30, 2019 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its third quarter 2019 financial results and provided an update on the company, including its progress with IPI-549, a first-in-class oral immuno-oncology product candidate targeting immune-suppressive tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition (Press release, Infinity Pharmaceuticals, OCT 30, 2019, View Source [SID1234550039]).

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"It is an exciting and important time for Infinity as we anticipate completing enrollment in MARIO-1 by the end of this year and presenting data from our MARIO-1 and MARIO-3 trials in 2020. During 2020 we also expect to complete enrollment in MARIO-275, our randomized, controlled Phase 2 trial in patients with bladder cancer," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "As we advance multiple studies designed to produce compelling data across a wide range of indications in innovative combinations, we are witnessing surging recognition of the importance of tumor macrophage-directed immuno-oncology therapies among the medical and scientific communities. With additional clinical and translational data expected from IPI-549 studies starting in 2020, Infinity is positioned to become the leader in macrophage-targeted immunotherapy."

Updated IPI-549 Clinical Program Guidance

MARIO-1 enrollment completion expected by year end with data presentation expected in 2020. MARIO-1 is the company’s ongoing Phase 1/1b study of IPI-549 as a monotherapy and in combination with Opdivo in patients with advanced solid tumors.
MARIO-3 enrollment completion and data presentation expected in 2020. MARIO-3 is the company’s ongoing Phase 2 study in collaboration with Roche/Genentech to evaluate IPI-549 in novel triple combination front-line therapies with Tecentriq and Abraxane in triple negative breast cancer (TNBC) and with Tecentriq and Avastin in renal cell cancer (RCC).
MARIO-275 enrollment completion expected during 2020. MARIO-275 is the company’s global, randomized Phase 2 study in collaboration with Bristol-Myers Squibb, to evaluate IPI-549 in combination with Opdivo in platinum-refractory, I/O naïve patients with advanced urothelial (bladder) cancer.
Arcus Biosciences Collaboration Study Initiated: This Phase 1 trial, being conducted by Arcus, is evaluating a checkpoint-inhibitor free, novel triple-combination regimen of IPI-549 + AB928 (dual adenosine receptor antagonist) + Doxil in advanced TNBC patients.
Third Quarter 2019 Financial Results

At September 30, 2019, Infinity had total cash, cash equivalents and available-for-sale securities of $52.0 million, compared to $63.0 million at June 30, 2019.
Research and development expense for the third quarter of 2019 was $7.1 million, compared to $5.4 million for the same period in 2018. The increase in R&D expense was primarily due to an increase in clinical and development activities for IPI-549.
General and administrative expense was $3.6 million for the third quarter of 2019, compared to $3.4 million for the same period in 2018.
Net loss for the third quarter of 2019 was $11.4 million, or a basic and diluted loss per common share of $0.20, compared to net income of $13.4 million, or a basic and diluted earnings per common share of $0.23 for the same period in 2018. In the third quarter of 2018, we recognized $22.0 million in revenue related to a one-time payment due from Verastem for the approval by the U.S. Food and Drug Administration of Copiktra, which Infinity licensed to Verastem in 2016.
2019 Financial Guidance

Net Loss: Infinity expects net loss for 2019 to range from $40 million to $50 million.
Cash and Investments: Infinity expects to end 2019 with a year-end cash, cash equivalents and available-for-sale securities balance ranging from $40 million to $50 million.
Cash Runway: Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities will be adequate to satisfy the company’s capital needs for at least the next 12 months. Infinity’s financial guidance excludes additional funding or business development activities.
Conference Call Information

Infinity will host a conference call today, October 30, 2019, at 4:30 p.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial 1-877-316-5293 (domestic) and 1-631-291-4526 (international) five minutes prior to start time. The conference ID number is 2612978. An archived version of the webcast will be available on Infinity’s website for 30 days.