On August 9, 2018 Aurinia Pharmaceuticals Inc. (NASDAQ:AUPH / TSX:AUP) ("Aurinia" or the "Company")reported that it has released its financial results for the second quarter ended June 30, 2018 (Press release, Aurinia Pharmaceuticals, AUG 9, 2018, View Source [SID1234528597]). Amounts, unless specified otherwise, are expressed in U.S. dollars.

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"We are excited to announce that the AURORA Phase III trial in lupus nephritis is running ahead of schedule and we now anticipate completing enrollment in early Q4 2018. We are extremely pleased with the trial’s progress thus far and having patients roll over into the AURORA 2 extension study reinforces our confidence in the program", said Richard Glickman, Aurinia’s CEO and Chairman of the Board. "Our clinical team continues to deliver on our important milestones with the Phase II trials in FSGS and Dry Eye now initiated. We are well-capitalized into 2020 and look forward to an eventful second half of the year."

Highlights

Our Phase III clinical trial ("AURORA") to evaluate voclosporin for the treatment of lupus nephritis ("LN"), which we initiated in May of 2017, is now expected to complete enrollment in early Q4 2018. We have over 225 clinical trial sites activated and able to enroll patients in 29 countries around the globe.
The first patients have rolled over into the AURORA 2 blinded extension study from the AURORA Phase III clinical trial. The purpose of AURORA 2 is to assess the long-term safety and tolerability of voclosporin in patients with LN; however, this study is not a requirement for potential regulatory approval for voclosporin.
We initiated a Phase II proof-of-concept study in focal segmental glomerulosclerosis ("FSGS") in June 2018. This is an open-label study of 20 treatment naïve patients. We submitted our Investigational New Drug application ("IND") to the FDA in Q1 2018 and received agreement from the FDA with regards to the guidance we provided on this study.
We also initiated a Phase II head-to-head tolerability study of voclosporin ophthalmic solution ("VOS") versus Restasis (cyclosporine ophthalmic emulsion) 0.05% for the treatment of Dry Eye Syndrome ("DES") in July 2018. Depending on the pace of recruitment, data could be available as early as the end of this year or early 2019. This four-week study of approximately 90 patients is expected to be completed by the end of 2018. We believe calcineurin inhibitors ("CNIs") are a mainstay of treatment for DES, and the goal of this program is to develop a best-in-class treatment option, and upon completion, we will look to evaluate strategic alternatives for this asset.
Financial Liquidity at June 30, 2018

At June 30, 2018, we had cash, cash equivalents and short term investments of $150.2 million compared to $159.1 million at March 31, 2018 and $173.5 million at December 31, 2017. Net cash used in operating activities was $12.3 million for the second quarter ended June 30, 2018 compared to $14.0 million for the second quarter ended June 30, 2017.

We believe, based on our current plans, that we have sufficient financial resources to fund our existing LN program, including the AURORA trial and the NDA submission to the FDA, conduct the Phase II trials for FSGS and DES, and fund operations into 2020.

Financial Results for the Three and Six Months Ended June 30, 2018

We reported a consolidated net loss of $15.7 million or $0.19 per common share for the three months ended June 30, 2018, as compared to a consolidated net loss of $2.4 million or $0.03 per common share for the three months ended June 30, 2017.

The increase in the loss for the three months ended June 30, 2018 compared to the same period in 2017 was primarily due to the non-cash change in the estimated fair value of derivative warrant liabilities of $9.4 million. The three months ended June 30, 2018 reflected a $1.9 million increase in the estimated fair value of derivative warrant liabilities compared to a reduction of $7.5 million in the estimated fair value of derivative warrant liabilities for the three months ended June 30, 2017. The change in the revaluation of the derivative warrant liabilities is primarily driven by the change in our share price at each period end. An increase in our share price results in an increase in the estimated fair value of derivative warrant liabilities and vice versa. The derivative warrant liabilities will ultimately be eliminated on the exercise or forfeiture of the warrants and will not result in any cash outlay by the Company.

The net loss before the non-cash change in estimated fair value of derivative warrant liabilities was $13.8 million for the three months ended June 30, 2018 compared to $9.9 million for the same period in 2017 with the increased loss amount primarily reflecting higher research and development expenses.

For the six months ended June 30, 2018, the consolidated net loss was $31.2 million or $0.37 per common share compared to a consolidated net loss of $54.3 million or $0.78 per common share for the comparable period in 2017. For the six months ended June 30, 2018 we recorded an increase of $4.6 million in the estimated fair value of derivative warrant liabilities compared to $33.3 million for the comparable period in 2017.

The net loss before the non-cash change in estimated fair value of derivative warrant liabilities was $26.6 million for the six months ended June 30, 2018 compared to $21.1 million for the same period in 2017. The increased loss reflected higher research and development expenses.

Research and development expenses increased to $10.5 million for the three months ended June 30, 2018, compared to $7.1 million for the three months ended June 30, 2017. We incurred research and development expenses of $19.4 million for the six months ended June 30, 2018, as compared to $14.4 million for the same period in 2017. The increased research and development expenses reflected higher AURORA clinical and drug supply costs as well as startup costs for the AURORA 2 extension study, and the FSGS and DES studies.

Corporate, administration and business development expenses increased to $3.5 million for the three months ended June 30, 2018, compared to $2.9 million for the same period in 2017. We incurred corporate, administration and business development expenses of $7.3 million for the six months ended June 30, 2018 compared to $6.3 million for the comparable period in 2017. The increase was primarily due to higher non-cash stock compensation expense in 2018 compared to the same periods in 2017.

On August 9, 2018 CymaBay Therapeutics, Inc. (NASDAQ: CBAY) a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported financial results and a corporate update for the quarter and six months ended June 30, 2018 (Press release, CymaBay Therapeutics, AUG 9, 2018, View Source [SID1234528710]).

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"Having completed a significant capital raise to start the year, we are now laser focused on high quality execution as we continue to advance novel treatment alternatives to patients suffering from liver diseases with significant unmet needs," said Sujal Shah, President and Chief Executive Officer of CymaBay. "We have collected feedback from regulatory agencies and finalized our design for our planned Phase 3 study of seladelpar in primary biliary cholangitis (PBC) and we are on track to initiate this study in the second half of the year. Data from the ongoing Phase 2 study of seladelpar in patients with PBC were presented in a late-breaking presentation at The International Liver CongressTM in April, and we believe these data support the potential of seladelpar to provide improved efficacy and better tolerability over existing second-line therapy. We are also excited to expand development of seladelpar into non-alcoholic steatohepatitis (NASH) with the recent initiation of a Phase 2b proof-of-concept study. We believe seladelpar’s PPAR-delta mechanism of action may be particularly well suited to treat NASH given its beneficial impacts on lipid, glucose, and sterol metabolism, as well as its effects on inflammation and fibrogenesis."

Second Quarter 2018 Business Highlights

Announced plans to proceed with a double-blind, placebo-controlled Phase 3 pivotal study of seladelpar in PBC. The study intends to enroll approximately 240 patients randomized to receive either 5 mg or 10 mg seladelpar, or placebo. Patients who have an inadequate response on the 5 mg dose will have the potential to increase to 10 mg after 6 months.
Presented new 12-week and 26-week results from the ongoing Phase 2 study of seladelpar in primary biliary cholangitis (PBC) at The International Liver CongressTM in April.
• The results showed potent anti-cholestatic and anti-inflammatory activities, with no drug-induced pruritus, through 26 weeks of treatment.
• 52-week data from this study are expected to be announced in the fourth quarter of 2018.
Initiated a Phase 2b proof-of-concept study of seladelpar for the treatment of NASH. This randomized, placebo-controlled, parallel, dose-ranging study is intended to enroll approximately 175 patients with liver biopsy proven NASH. The primary efficacy outcome is change in liver fat content from baseline to 12 weeks as measured by magnetic resonance imaging. The secondary analysis includes evaluation of histological improvement in NASH and fibrosis as assessed by comparing liver biopsy samples taken at baseline and 52 weeks.
Added CBAY to the Russell 3000 and the Russell 2000 Indexes at the conclusion of the Russell US Indexes annual reconstitution.
Expanded workforce with clinical, regulatory, scientific and administrative personnel necessary to support expansion of clinical programs, notably the Phase 3 PBC registration study, and business operations.
Amended the existing corporate office lease to extend it for an additional 5-year term and relocate to a larger facility within current corporate campus location.
Second Quarter 2018 Financial Results

Cash, cash equivalents and marketable securities totaled $212.1 million at June 30, 2018. Based on current projections, existing cash is expected to fund the current operating plan into 2021.
Term loan facility repaid in full resulting in a debt-free balance sheet at June 30, 2018.
No collaboration revenue was recognized in the second quarter of 2018.
Research and development expenses were $14.4 million in the second quarter of 2018 as compared to $4.0 million in the same period of 2017 and consisted primarily of higher clinical trial expenses related to ongoing PBC Phase 2 and extension studies, start-up activities for the planned PBC Phase 3 study, and enrollment activities associated with the recently initiated NASH Phase 2b study. Additionally, higher seladelpar drug manufacturing expenses were incurred to provide clinical supplies to these studies.
General and administrative expenses were unchanged at $3.6 million in the second quarter of 2018 and 2017.
Net loss was $17.5 million, or ($0.30) per share in the second quarter of 2018, as compared to $8.9 million, or ($0.31) per share in the second quarter of 2017. Net loss was higher primarily due to increased research and development expenses incurred to support expanding clinical studies.
First Half 2018 Financial Results

No collaboration revenue was recognized in the first half of 2018 as compared to $4.8 million in the same period of 2017. Revenue associated with the collaboration arrangement with Kowa Pharmaceuticals America was recognized in 2017 upon transfer of a license and know how to Kowa.
Research and development expenses were $23.9 million in the first half of 2018 as compared to $8.1 million in the same period of 2017 and consisted primarily of higher clinical trial expenses related to ongoing PBC Phase 2 and extension studies, start-up activities for the planned PBC Phase 3 study, and enrollment activities associated with the recently initiated NASH Phase 2b study. Additionally, higher seladelpar drug manufacturing expenses were incurred to provide clinical supplies to these studies.
General and administrative expenses were $6.9 million in the first half of 2018, as compared to $7.3 million in the first half of 2017. Expenses were higher in 2017 primarily due to severance expenses associated with the retirement of CymaBay’s former CEO.
Net loss was $34.5 million, or ($0.61) per share in the first half of 2018, as compared to $14.3 million, or ($0.52) per share in the second quarter of 2017. Net loss was higher primarily due to increased research and development expenses incurred to support the expanding clinical studies and lower collaboration revenue.
Conference Call Details
CymaBay management will host a conference call today at 4:30 p.m. ET to discuss second quarter 2018 financial results and provide a business update. To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID# 13680965. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

On August 9, 2018 Corcept Therapeutics Incorporated (NASDAQ: CORT), a company engaged in the discovery, development and commercialization of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the stress hormone cortisol, reported its results for the quarter ended June 30, 2018 (Press release, Corcept Therapeutics, AUG 9, 2018, View Source [SID1234528784]).

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Financial Highlights

Revenue of $62.3 million, a 75 percent increase from second quarter 2017 GAAP net income of $0.14 per share, compared to $0.10 per share in second quarter 2017 Non-GAAP net income of $0.20 per share, compared to $0.13 per share in second quarter 2017 Cash and investments of $159.9 million, a $19.6 million increase from first quarter 2018 2018 revenue guidance revised to $250 – 270 million, from $275 – 300 million Company announces $100 million stock repurchase program Relacorilant Data

The final 18 patients enrolled in the trial (the "High-Dose" cohort) receive 250 mg/day of relacorilant for four weeks, with dose being increased, as tolerability permits, to 300 mg/day for four weeks, then 350 mg/day for four weeks, then 400 mg/day for four weeks; data are available for the 250 mg/day and 300 mg/day dose levels.

Based on FDA feedback, Corcept has developed response criteria for relacorilant’s Phase 3 trial. Applying these endpoints to the High-Dose cohort at eight weeks of treatment (conclusion of the 300 mg dose level) produces the following results:

Fifty-eight percent of patients with hyperglycemia achieved improved glucose control, as shown by a
(i) 0.5 percent or greater reduction in HbA1c or (ii) 50 mg/dl or greater reduction (or normalization) in 2-hour glucose as measured in the oral glucose tolerance test or (iii) 25 percent or greater decrease in antidiabetic medications

Fifty-five percent of patients with uncontrolled hypertension achieved a five millimeter or greater drop in either systolic or diastolic blood pressure, as measured by 24-hour ambulatory monitoring No evidence of progesterone receptor affinity; no instances of hypokalemia Testing of higher doses is ongoing; Phase 3 trial planned to start this year Oncologic and Metabolic Disorders

Placebo-controlled, Phase 2 trial of relacorilant plus Abraxane (nab-paclitaxel) in metastatic ovarian cancer planned to start by year-end Results expected by year-end in study of relacorilant plus Abraxane in patients with metastatic pancreatic cancer Dosing continues in Phase 1/2 trial of CORT125281 plus Xtandi (enzalutamide) in patients with metastatic castration-resistant prostate cancer Planning underway for placebo-controlled, Phase 2 trials of CORT118335 in patients with antipsychotic-induced weight gain and non-alcoholic steatohepatitis ("NASH"); both trials planned to start by year-end Financial Results

Corcept reported quarterly revenue of $62.3 million, compared to $35.6 million in the second quarter of 2017. Second quarter GAAP net income was $18.2 million, compared to $12.6 million in the same period last year. Excluding non-cash expenses related to stock-based compensation, utilization of deferred tax assets, accreted interest on the company’s retired royalty financing obligation and related income tax effects, non-GAAP net income in the second quarter was $25.4 million, compared to $16.0 million in the second quarter of 2017. (A reconciliation of GAAP to non-GAAP net income is set forth below.) The company reduced its 2018 guidance to $250 – 270 million.

Second quarter operating expenses were $41.7 million, compared to $22.8 million in the second quarter of 2017, primarily due to increased spending to advance relacorilant, CORT118335 and CORT125281 and costs from increased sales volume.

Cash and investments were $159.9 million at June 30, 2018, an increase of $19.6 million from first quarter 2018.

The company announced a program to repurchase up to $100 million of its common stock, which it intends to fund using cash and investments. Details of the program are provided below.

"Our Cushing’s syndrome franchise continues its significant growth, driven by physicians’ increasing realization that hypercortisolism is a serious disorder and that cortisol modulation is the best medical therapy for many patients," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "We are confident this shift in medical practice will continue."

Relacorilant’s Phase 2 Trial

"Interim data from our Phase 2 trial’s High-Dose cohort showed that relacorilant provided clinically meaningful benefit without the two off-target effects – progesterone receptor affinity and increased cortisol levels – that cause Korlym’s most common and serious adverse events – termination of pregnancy, endometrial thickening, vaginal bleeding and low potassium (hypokalemia)," said Robert S. Fishman, MD, Corcept’s Chief Medical Officer.

"That relacorilant did not cause hypokalemia in these patients is surprising – and important," he added. "Forty-four percent of the patients in Korlym’s pivotal trial experienced hypokalemia, which can be life-threatening. It is one of the most common adverse events in patients taking Korlym today.

"Interim efficacy data have also been impressive. Based on our planned Phase 3 endpoints, 58 percent of the patients with hyperglycemia achieved improved glucose control. Applying the same endpoints, this figure was 48 percent at the comparable time in Korlym’s pivotal trial ("SEISMIC") and 23 percent at the conclusion of treatment in the Low-Dose cohort. (See Figure 1) For patients with hypertension, 55 percent responded in the High-Dose cohort, compared to 44 percent in SEISMIC and 45 percent of the Low-Dose cohort. (See Figure 2)

"Relacorilant was well-tolerated," he concluded. "We observed one serious adverse event, a pilonidal abscess, which resolved without discontinuing relacorilant. One patient discontinued due to musculoskeletal pain and fatigue – a relatively common adverse event seen as cortisol modulation decreases cortisol activity."

Oncology

"At ASCO (Free ASCO Whitepaper)’s annual meeting this May, we reported positive data from the dose-finding portion of our Phase 1/2 study of relacorilant plus Abraxane to treat patients with solid tumors," added Dr. Fishman. "At the minimum therapeutic dose, four of nine patients with metastatic pancreatic cancer and four of seven patients with metastatic ovarian cancer demonstrated durable disease control. These results are especially notable in patients with such dire disease, all of whom had progressed on one or more prior taxane-based treatments. Recently, another patient with pancreatic cancer has achieved a partial response.

"These results justify significantly expanding our oncology program. By year-end, we plan to open a placebo-controlled, Phase 2 trial of relacorilant plus Abraxane in metastatic ovarian cancer. We also expect to have enough data by year-end in patients with metastatic pancreatic cancer to determine if a definitive trial is warranted."

Conference Call

Corcept will hold a conference call August 9, 2018, at 5:00 pm Eastern Time (2:00 pm Pacific Time). To participate, dial 1-888-394-8218 from the United States or 1-323-794-2588 internationally ten minutes before the start of the call (passcode: 6703650). A replay will be available through August 23, 2018 at 888-203-1112 from the United States and 719-457-0820 internationally (passcode: 6703650).

On August 8, 2018 German biopharma company MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; NASDAQ: MOR) and I-Mab Biopharma ("I-Mab"), a Shanghai-based biotech company focused on innovative biologics in oncology and autoimmune diseases, reported that I-Mab has submitted an investigational new drug (IND) application to China National Drug Administration (CNDA) for TJ202/MOR202, a human monoclonal antibody directed against CD38 for the treatment of multiple myeloma (Press release, MorphoSys, AUG 8, 2018, View Source [SID1234528538]).

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Multiple myeloma is the second most common blood cancer worldwide. The patient number has gradually increased in China in recent years due to an increasingly aging population. Yet there are no effective biologics approved in China for this indication and current therapies have been associated with serious side effects and limited treatment efficacy.

TJ202/MOR202 is a monoclonal antibody derived from MorphoSys’s HuCAL antibody technology. The antibody is directed against CD38 on the surface of multiple myeloma cells, and, according to its suggested mode of action, recruits cells of the body’s immune system to kill the tumor. Scientific research suggest that an anti-CD38 antibody may have therapeutic potential also in other cancers, as well as autoimmune diseases.

China recently issued a new round of reform initiatives to accelerate clinical trial approval for new drugs, especially in oncology. "The IND submission was done after a successful pre-submission consultation meeting with Center for Drug Evaluation (CDE) of CNDA, which is required under China’s new drug regulation, unless waived," said Dr. Joan Shen, Head of R&D at I-Mab.

"CNDA has endorsed the overall clinical and regulatory strategy, as well as the study designs, which should lead to the biologics license application (BLA)," said Dr. Joan Shen.

Through a licensing agreement with MorphoSys AG in November 2017, I-Mab gained exclusive rights to develop and commercialize TJ202/MOR202 in Greater China territory, including mainland China, Hong Kong, Macau and Taiwan.

After observing patient responses in an interim analysis from an ongoing Phase 1/2a trial in patients with relapsed/refractory multiple myeloma in Germany and Austria, MorphoSys decided to support I-Mab to lead clinical development of TJ202/MOR202 in Greater China. MorphoSys will continue to evaluate additional other suitable indications for further global development of TJ202/MOR202.

Dr. Malte Peters, Chief Development Officer of MorphoSys AG commented: "We are delighted that our partner I-Mab has taken this important step in advancing TJ202/MOR202 into clinical development in China. We look forward to supporting I-Mab in developing this investigational compound with the goal of helping Chinese patients in multiple myeloma, an indication with a high unmet medical need."

"With a fast-to-market strategy under the new drug regulation, we hope to bring this innovative treatment to patients as soon as possible," Dr. Shen commented. "MorphoSys and I-Mab plan to continuously evaluate the potential and further development of TJ202 in other indications."

On August 8, 2018 Verastem, Inc. (Nasdaq: VSTM) (Verastem Oncology or the Company), focused on developing and commercializing medicines to improve the survival and quality of life of cancer patients, reported financial results for the quarter ended June 30, 2018 and provided an overview of certain corporate developments (Press release, Verastem, AUG 8, 2018, View Source [SID1234528554]).

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"During the second quarter of 2018, we’ve been actively preparing for the commercialization of duvelisib, our first-in-class, oral dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma for the treatment of patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) or follicular lymphoma (FL)," said Robert Forrester, President and Chief Executive Officer of Verastem Oncology. "In advance of our target action date of October 5, 2018, we have been building our U.S. sales force and commercial capabilities in preparation for a potential product launch of duvelisib in the U.S. in 2018. On the financial front, we have significantly strengthened our balance sheet, ending June 30, 2018 with $168.7 million in cash and cash equivalents."

Second Quarter 2018 and Recent Highlights:

Corporate and Financial

Duvelisib NDA accepted by FDA with priority review – In April 2018, Verastem Oncology announced that the U.S. Food and Drug Administration (FDA) accepted the duvelisib New Drug Application (NDA) for filing with Priority Review, with a target action date of October 5, 2018. In the accepted NDA, the Company is seeking full approval for duvelisib, its first-in-class investigational oral dual inhibitor of PI3K-delta and PI3K-gamma, for the treatment of relapsed or refractory CLL/SLL and accelerated approval for the treatment of relapsed or refractory FL. The duvelisib NDA is supported by clinical data from the randomized Phase 3 DUO study evaluating duvelisib as a monotherapy in patients with relapsed or refractory CLL/SLL, as well as the Phase 2 DYNAMO study evaluating patients with iNHL that are double-refractory to both rituximab and chemotherapy or radioimmunotherapy. Both DUO and DYNAMO achieved their primary endpoints.
Hosted Analyst and Investor Day highlighting commercial potential of duvelisib – In early May 2018, Verastem Oncology hosted an Analyst and Investor Day in New York City titled, "Duvelisib: Harnessing the Power of Dual PI3K Inhibition." Key opinion leaders in the hematologic oncology field including Dr. Lori Kunkel, Former Chief Medical Officer, Pharmacyclics, Dr. Jennifer Brown, Dana-Farber Cancer Institute, Dr. Ian Flinn, Sarah Cannon Research Institute, Dr. Steven Horwitz, Memorial Sloan Kettering Cancer Center as well as Dr. Brian Koffman, Founder & Medical Director of the Chronic Lymphocytic Leukemia (CLL) Society, and a CLL patient, joined the Verastem Oncology executive leadership team for an in-depth discussion regarding the unmet need among CLL/SLL and FL patients, where PI3K-delta and PI3K-gamma inhibitors fit into the treatment paradigm, and the growing opportunity for duvelisib in CLL/SLL and FL, and beyond. The Company also provided an overview of its duvelisib commercial strategy and initiatives. The webcast is available within the "Media" section of the Company’s website at www.verastem.com.
Signed exclusive license agreement with Yakult Honsha Co., Ltd. (Yakult) to develop and commercialize duvelisib in Japan – In June 2018, Verastem Oncology announced its entry into an exclusive license and collaboration agreement with Yakult to develop and commercialize duvelisib for the treatment, prevention or diagnosis of all oncology indications in Japan. The transaction, which carries a total deal value of up to $100.0 million, includes a one-time upfront payment of $10.0 million and up to an additional $90.0 million if certain future pre-specified development, regulatory and commercial milestones are successfully achieved by Yakult. In addition, Verastem Oncology is also eligible to receive double-digit royalties based on future net sales of duvelisib in Japan. Pursuant to the agreement, Yakult has the right to develop and commercialize duvelisib in Japan at its own cost and expense. In addition, Yakult may fund certain global development costs on a pro-rata basis. Verastem Oncology retains all rights to duvelisib outside of Japan.
Strengthened the balance sheet through the sale of equity for net proceeds of approximately $105 Million – In May 2018, Verastem Oncology completed an underwritten registered offering of 8,944,444 shares of its common stock at a price to the public of $4.50 per share. The net proceeds to Verastem from the offering were approximately $38.3 million. In June 2018, Verastem Oncology completed a registered offering of 7,166,666 shares of its common stock at a price of $6.00 per share to funds managed by Consonance Capital. The net proceeds to Verastem Oncology from this offering were approximately $42.8 million. The Company also sold 6,314,410 shares of common stock under its at-the-market equity offering program for net proceeds of approximately $23.7 million.
Joined the Russell 3000 Index – In June 2018, the Company joined the broad-market Russell 3000 Index as part of the Russell US Indexes annual reconstitution.

Scientific Presentations at Major Medical Meetings

Duvelisib

The efficacy of duvelisib monotherapy following disease progression on ofatumumab monotherapy in patients with relapsed/refractory CLL or SLL in the DUO crossover extension study – In June 2018, at both the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2018 Annual Meeting (ASCO 2018) and the European Hematology Association (EHA) (Free EHA Whitepaper) 2018 Annual Meeting (EHA 2018), Dr. Byrone Kuss, Flinders Medical Centre, and Dr. Peter Hillman, St. James University Hospital, respectively, presented additional data from the open-label, DUO crossover extension study where patients with radiologically confirmed progressive disease (PD) following treatment with ofatumumab in DUO were given the option to receive treatment with duvelisib. Among the 89 evaluable patients (median 3 prior therapies; range 2-8), duvelisib as a monotherapy achieved a 73% overall response rate (ORR) per investigators assessment in the extension study (95% confidence interval CI: 64,82); 5% complete response with incomplete marrow recovery (CRi), and 68% partial response (PR). The median progression-free survival (mPFS) for duvelisib in the DUO crossover extension study was 15 months (95% CI: 10,17). Notably, 83% of patients in the duvelisib arm post-crossover had >50% reductions in the size of their target nodal lesions. The safety profile of duvelisib as a monotherapy was manageable and consistent with what was observed in the Phase 3 DUO study. These data build upon the previously reported positive DUO results and further support duvelisib as an effective oral monotherapy treatment option for patients with relapsed or refractory CLL/SLL.
A Phase IB/II study of duvelisib in combination with Fludarabine (F), Cyclophosphamide (C), and Rituximab (R) (dFCR) for frontline therapy of younger CLL patients – At EHA (Free EHA Whitepaper) 2018, Dr. Matthew Davids, Dana-Farber Cancer Institute, presented data on the 31 patients evaluable for post-dFCR response. The ORR was 94%, with 26% (n=8) of patients achieving a complete response (CR) or CRi, and 68% achieving a PR. The best rate of minimum residual disease (MRD) negativity in the bone marrow (BM) in patients with at least one evaluation was 76% (22 of 29 patients). All patients who achieved CR/CRi at the primary endpoint also had BM-MRD negativity (26%). Among survivors, the median follow-up is 24.5 months (range 6.9-46 months). The two-year progression-free survival and overall survival rates for patients in the study were both 97%. Eight patients have now completed two years of duvelisib maintenance therapy. Based on these results the recommended Phase 2 dose of duvelisib in combination with FCR was 25mg twice daily. The most common all grade non-hematologic adverse events (AEs) were nausea (72%, all Grade 1/2), fatigue (69%, 3% Grade 3), fever (53%, all Grade 1/2), diarrhea (47%, 3% Grade 3), transaminitis (34%, 28% Grade 3/4), anorexia (34%, all Grade 1/2), vomiting (28%, all Grade 1/2), pruritus (16%, 3% Grade 3), arthritis (9%, all Grade 2) and Cytomegalovirus (CMV) reactivation (6%, both Grade 2). The most common all grade hematologic adverse events were thrombocytopenia (65%; 34% Grade 3-4), neutropenia (59%; 50% Grade 3-4), and anemia (38%, 16% Grade 3). Serious AEs included transaminitis (Grade ≥3), febrile neutropenia (n=6, all Grade 3), pneumonia (n=6, including 3 cases of PJP despite planned prophylaxis), and colitis (n=1 Grade 2, n=1 Grade 3). These results suggest that duvelisib in combination with FCR is an effective regimen for the initial therapy of younger, fit CLL patients and results in a high rate of BM-MRD negativity (76%), significantly higher than historical data with FCR.
The effect of duvelisib, a dual inhibitor of PI3K-δ,γ, on components of the tumor microenvironment in previously untreated follicular lymphoma patients – At both ASCO (Free ASCO Whitepaper) 2018 and EHA (Free EHA Whitepaper) 2018, Dr. Carla Casulo, University of Rochester, Wilmot Cancer Center, presented data from blood samples from healthy volunteers and FL patients treated in the CONTEMPO study. Samples collected both pre- and post-duvelisib treatment were analyzed. Ex vivo and in vitro PI3K-delta assays and PI3K-gamma assays, with PI3K-gamma-selective (idelalisib, TGR-1202, IPI-3063) and PI3K-delta-selective (IPI-549) inhibitors were compared. Collectively, the results of this analysis support the thesis that duvelisib disrupts PI3K-delta and PI3K-gamma function in FL patients, inhibiting the tumor microenvironment (TME) cancer-supportive macrophages and T-cells.
Duvelisib inhibition of chemokines in patients with CLL (DUO) and iNHL (DYNAMO) – At both ASCO (Free ASCO Whitepaper) 2018 and EHA (Free EHA Whitepaper) 2018, Dr. David Weaver, Verastem Oncology’s Vice President, Translational Medicine, presented data showing that PI3K-delta inhibition directly targets proliferation and survival of malignant leukemia and lymphoma cells, while PI3K-gamma inhibition modulates the TME through key support cells, including tumor-associated macrophages, nurse-like stroma and T-cells, and via soluble factors stimulating tumor growth, survival and migration. Serum samples from patients in the Phase 3 DUO study in relapsed/refractory CLL/SLL and the Phase 2 DYNAMO study in relapsed/refractory indolent non-Hodgkin lymphoma (iNHL) were collected at baseline and at infusion cycle date C2D1 and used for correlative studies of 24 chemokines, cytokines and serum factors. These data support the hypothesis that treatment with duvelisib results in significant reduction of chemokines potentially derived from the tumor cells and TME and that further investigation of the effects of duvelisib on TME pharmacodynamic markers is warranted.
Presented scientific data supporting immuno-oncology applications of duvelisib at the 3rd annual Advances in Immuno-Oncology Congress – In May 2018, Jonathan Pachter, Ph.D., Verastem Oncology’s Chief Scientific Officer, gave an oral presentation highlighting the unique potential of duvelisib, as a dual inhibitor of PI3K-delta and PI3K-gamma, to enhance the efficacy of immune checkpoint and co-stimulatory antibodies in preclinical models of both hematological malignancies and solid tumors. Dr. Pachter also moderated a round table discussion regarding novel checkpoint pathways and emerging strategies for combined modality treatment.
Defactinib

Presented preliminary Phase 1 results from combination trial with defactinib, pembrolizumab and gemcitabine in advanced cancer – At ASCO (Free ASCO Whitepaper) 2018, Dr. Andrea Wang-Gillam presented a poster describing results from the ongoing Phase 1 study evaluating defactinib in combination with pembrolizumab and gemcitabine in patients with advanced cancer, including pancreatic cancer. The combination treatment appears to be well tolerated, the recommended Phase 2 dose was established, and the expansion phase of the study is now ongoing. Encouraging signs of clinical activity were observed in three pancreatic ductal adenocarcinoma (PDAC) patients treated beyond 250 days, including one patient with confirmed PR and two patients with stable disease. Meaningful reductions (57-96%) in the pancreatic cancer marker CA19-9 were also observed in all three patients. In addition, analysis of paired biopsies showed that the combination treatment induced desirable biomarker changes including increased proliferating CD8+ T-cells and reduced immunosuppressive Tregs and macrophages.
Presented scientific data supporting immuno-oncology applications of defactinib at the 3rd Annual Advances in Immuno-Oncology Congress – During Dr. Pachter’s oral presentation, he also provided an update on the scientific rationale and clinical progress of Verastem Oncology’s lead focal adhesion kinase (FAK) inhibitor, defactinib, in combination with PD-1 and PD-L1 inhibitors in solid tumors.
All posters and presentations are available within the "Media" section of the Company’s website at www.verastem.com.

Second Quarter 2018 Financial Results

Net loss for the three months ended June 30, 2018 (2018 Quarter) was $18.4 million, or $0.30 per share, as compared to a net loss of $13.4 million, or $0.36 per share, for the three months ended June 30, 2017 (2017 Quarter). Net loss for the 2018 Quarter includes license revenue of $10.0 million, related to the upfront payment received in connection with the license and collaboration agreement with Yakult in June 2018. Cash used in operating activities, excluding the upfront payment from Yakult, was $20.3 million for the 2018 Quarter.

Research and development expense for the 2018 Quarter was $12.4 million compared to $9.0 million for the 2017 Quarter. The $3.4 million increase from the 2017 Quarter to the 2018 Quarter was primarily related to an increase of $1.6 million in contract research organization expense for outsourced biology, development and clinical services, which includes the Company’s clinical trial costs, an increase of $1.0 million in personnel related costs, and an increase of $0.4 million in stock-based compensation expense.

General and administrative expense for the 2018 Quarter was $15.8 million compared to $4.4 million for the 2017 Quarter. The increase of $11.4 million from the 2017 Quarter to the 2018 Quarter primarily resulted from increases in consulting and professional fees of $5.2 million, including $3.6 million related to commercial launch preparation activities, and an increase in personnel related costs of $4.4 million.

As of June 30, 2018, Verastem Oncology had cash and cash equivalents of $168.7 million compared to $86.7 million of cash, cash equivalents and investments as of December 31, 2017.

The number of outstanding common shares as of June 30, 2018 was 73,579,699.

Financial Guidance

Based on the Company’s current operating plans, assuming a favorable regulatory decision and estimated revenue, it expects to have sufficient cash and cash equivalents to fund operations into 2020.

About Duvelisib

Duvelisib is a first-in-class investigational oral, dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells and T-cells. PI3K signaling may lead to the proliferation of malignant B- and T-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib was evaluated in late- and mid-stage extension trials, including DUO, a randomized, Phase 3 monotherapy study in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL),4 and DYNAMO, a single-arm, Phase 2 monotherapy study in patients with refractory indolent non-Hodgkin lymphoma (iNHL).5 Both DUO and DYNAMO achieved their primary endpoints. Verastem Oncology’s New Drug Application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL, and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma (FL) was accepted for filing by the U.S. Food and Drug Administration (FDA), granted Priority Review and assigned a target action date of October 5, 2018. Duvelisib is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), and is being investigated in combination with other agents through investigator-sponsored studies.6 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

About Defactinib

Defactinib is an investigational inhibitor of focal adhesion kinase (FAK), a non-receptor tyrosine kinase that mediates oncogenic signaling in response to cellular adhesion and growth factors.7 Based on the multi-faceted roles of FAK, defactinib is used to treat cancer through modulation of the tumor microenvironment and enhancement of anti-tumor immunity.8,9 Defactinib is currently being evaluated in three separate clinical collaborations in combination with immunotherapeutic agents for the treatment of several different cancer types including pancreatic cancer, ovarian cancer, non-small cell lung cancer (NSCLC), and mesothelioma. These studies are combination clinical trials with pembrolizumab and avelumab from Merck & Co. and Pfizer/Merck KGaA, respectively.10,11,12 Information about these and additional clinical trials evaluating the safety and efficacy of defactinib can be found on www.clinicaltrials.gov.