Syndax Pharmaceuticals Reports Second Quarter 2019 Financial Results and Provides Clinical and Business Update

On August 7, 2019 Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the second quarter ended June 30, 2019 (Press release, Syndax, AUG 7, 2019, View Source [SID1234538298]). In addition, the Company provided a clinical and business update. As of June 30, 2019, Syndax had $80.5 million in cash, cash equivalents and short-term investments.

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"The FDA’s recent clearance of our IND application for SNDX-5613, a potent, highly selective, oral Menin inhibitor, marks an important milestone not only for Syndax, but also for patients suffering with acute leukemias," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "The Menin-MLL interaction has been strongly implicated in the development of MLL-r and NPM1 mutant leukemias. We look forward to initiating the clinical program and anticipate establishing a safe dose that provides appropriate target coverage in patients."

Dr. Morrison added, "We look forward to the near-term completion of E2112, the Phase 3 registration trial of entinostat plus exemestane in HR+, HER2- breast cancer, which we anticipate will occur either in the fourth quarter of 2019 or the first half of 2020. A positive survival benefit at either assessment will enable us to file an NDA with the FDA and take us one step closer to improving outcomes for patients with this difficult to treat disease."

Pipeline Updates

Entinostat

In May 2019, Syndax announced that the E2112 trial passed its fourth interim overall survival (OS) analysis and will continue as planned until either an OS benefit is observed, or the final target number of events occur. E2112 is Syndax’s NCI-sponsored, ECOG-ACRIN led Phase 3 registration trial of entinostat, a Class I selective HDAC inhibitor, plus exemestane in advanced hormone receptor positive, human epidermal growth factor receptor 2 negative (HR+, HER2-) breast cancer.

The Company continues to anticipate the final E2112 interim OS analysis in 4Q19. If necessary, the final OS assessment, which will be triggered when the trial reaches a total of 410 death events, is expected to be conducted in 2Q20. A positive OS assessment at any point would enable the Company to file for full regulatory approval. The E2112 trial design was informed by the Phase 2b ENCORE 301 trial, the results of which led to entinostat’s Breakthrough Therapy designation in HR+, HER2- breast cancer, in which patients receiving the entinostat/exemestane combination demonstrated a strong OS benefit.

SNDX-5613

In July 2019, Syndax announced that the U.S. Food and Drug Administration (FDA) cleared the Company’s Investigational New Drug (IND) application to begin a Phase 1/2 trial for SNDX-5613, a highly selective Menin inhibitor. The Company will refer to the clinical development of SNDX-5613 as the AUGMENT Program. The Phase 1/2 open-label trial will assess orally administered SNDX-5613 in adults with relapsed/refractory (R/R) acute leukemias. The Phase 1 dose escalation portion of the study will evaluate the safety, tolerability and pharmacokinetics of SNDX-5613, and will seek to establish a recommended Phase 2 dose. The Phase 2 portion will evaluate efficacy, as defined by Complete Response rate (per International Working Group response criteria), across three expansion cohorts: MLL-rearranged (MLL-r) acute lymphoblastic leukemia (ALL), MLL-r acute myeloid leukemia (AML), and NPM1 mutant AML. The Company expects to report initial clinical data from the trial in 2020.

SNDX-6352

Enrollment continues in the Phase 1 dose escalation trial of SNDX-6352, Syndax’s anti-CSF-1R monoclonal antibody, in patients with chronic graft versus host disease (cGVHD). The Company now anticipates results from this trial in the second half of 2020. The objectives of this trial are to evaluate the safety and preliminary efficacy of SNDX-6352 in cGVHD and to identify a recommended Phase 2 dose and schedule.

Corporate Updates

Syndax announced the appointment of Michael A. Metzger to its Board of Directors. Mr. Metzger has served as Syndax’s President and Chief Operating Officer since May 2015.

Second Quarter 2019 Financial Results

As of June 30, 2019, Syndax had cash, cash equivalents and short-term investments of $80.5 million and 31.6 million shares and share equivalents issued and outstanding.

Second quarter 2019 research and development expenses decreased to $12.3 million from $14.9 million. The second quarter decrease was primarily due to decreased development activities primarily in the ENCORE programs, and decreased CMC activities associated with SNDX-6352, partially offset by accrued milestone expenses associated with the development of SNDX-5613.

General and administrative expenses for the second quarter 2019 decreased to $3.5 million from $4.5 million. The decrease was primarily due to decreased professional fees and employee compensation expenses.

For the three months ended June 30, 2019, Syndax reported a net loss attributable to common stockholders of $14.9 million or $0.47 per share compared to $18.4 million or $0.74 per share for the prior year period.

Financial Guidance

Today the Company provided operating expense guidance for the third quarter and full year 2019. For the third quarter and full year 2019, research and development expenses are expected to be $11 to $12 million and $45 to $46 million, respectively, and total operating expenses are expected to be $15 to $16 million and $60 to $63 million, respectively.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Wednesday, August 7, 2019.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 8959885
Domestic Dial-in Number: 855-251-6663
International Dial-in Number: 281-542-4259
Live Webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.

Lipocine Announces Second Quarter 2019 Financial and Operational Results

On August 7, 2019 Lipocine Inc. (NASDAQ: LPCN), a clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, reported financial results for the second quarter and six months ended June 30, 2019, and provided a corporate update (Press release, Lipocine, AUG 7, 2019, View Source [SID1234538314]).

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Second Quarter and Recent Corporate Highlights

·Filed a New Drug Application ("NDA") for TLANDO as testosterone replacement therapy ("TRT") in adult males for conditions associated with a deficiency of endogenous testosterone, also known as hypogonadism.
oFDA has assigned November 9, 2019 as the Prescription Drug User Fee Act ("PDUFA") goal date.
·Received clearance from the U.S. Food and Drug Administration ("FDA") to clinically investigate LPCN 1144 in an expanded target population of adult male non-alcoholic steatohepatitis ("NASH") patients.
oFDA has waived the limitation of only testing in NASH subjects with total testosterone levels below 300 ng/dL (threshold for hypogonadism).
·Initiated the LiFT ("Liver Fat intervention with oral Testosterone") LPCN 1144 Phase 2 clinical study, a paired-biopsy study in confirmed pre-cirrhotic NASH subjects.
·Filed suit against Clarus Therapeutics, Inc. ("Clarus") in the United States District Court ("Delaware") alleging that Clarus’s JATENZO product infringes six of Lipocine’s U.S. patents.
·Joined the Russell Microcap Index effective July 1, 2019 as a result of the Russell indexes annual reconstitution.

"During the second quarter, we continued to advance our pipeline, with important milestones achieved for both TLANDO and LPCN 1144," said Dr. Mahesh Patel, Chairman, President and Chief Executive Officer of Lipocine. "The acceptance of the NDA for TLANDO for treating hypogonadism in males puts us on track for potential approval of this product in the fourth quarter of 2019. TLANDO is designed as a fixed dose oral TRT. For LPCN 1144, we were excited to initiate the LiFT study in biopsy confirmed NASH subjects, and based on our observed meaningful liver fat reduction in hypogonadal males, receive FDA clearance to expand our target patient population in adult NASH males regardless of their hypogonadal status. We believe this increases LPCN 1144’s potential utility in a broader NASH population who reportedly have low testosterone."

Second Quarter Ended June 30, 2019 Financial Results

Lipocine reported a net loss of $3.4 million, or ($0.14) per diluted share, for the quarter ended June 30, 2019, compared with a net loss of $3.3 million, or ($0.15) per diluted share, in the quarter ended June 30, 2018.

Research and development expenses were $2.0 million for the quarter ended June 30, 2019, compared with $1.5 million for the quarter ended June 30, 2018. The increase in research and development expenses was primarily due to increases in outside service costs related to the second quarter initiation of the LiFT LPCN 1144 Phase 2 clinical study in confirmed pre-cirrhotic NASH subjects and increased contract research organization and outside consulting costs for TLANDO in connection with completion of the ABPM study and the filing of the NDA.

General and administrative expenses were $1.4 million for the quarter ended June 30, 2019, compared with $1.7 million for the quarter ended June 30, 2018. The decrease in general and administrative was primarily due to decreased personnel costs, a decrease in severance compensation, a decrease in stock compensation expense and a decrease in bonus expense. The decreases were offset by increases in other general and administrative expenses including an increase in professional fees, an increase in corporate insurance and an increase in marketing expenses.

As of June 30, 2019, the Company had total cash, cash equivalents and marketable securities aggregating $19.5 million, compared to $20.3 million at December 31, 2018. Of the total cash, cash equivalents and marketable securities balances, $5.0 million is restricted under our agreement with Silicon Valley Bank until TLANDO receives approval.

Six Months Ended June 30, 2019 Financial Results

Lipocine reported a net loss of $6.7 million, or ($0.28) per diluted share, for the six months ended June 30, 2019, compared with a net loss of $6.0 million, or ($0.28) per diluted share, in the six months ended June 30, 2018.

Research and development expenses were $3.9 million for the six months ended June 30, 2019, compared with $2.9 million for the six months ended June 30, 2018. The increase/decrease in research and development expenses was primarily due to increased contract research organization and outside consulting costs for TLANDO in connection with completion of the ABPM study and the filing of the NDA, increased manufacturing costs for TLANDO, increased outside service costs related to the second quarter initiation of the LiFT LPCN 1144 Phase 2 clinical study in confirmed pre-cirrhotic NASH subjects, increased outside contract research organization costs and sample storage costs related to TLANDO XR (LPCN 1111) offset by decreased contract manufacturing costs for LPCN 1107 and decreased personnel costs.

General and administrative expenses were $2.6 million for the six months ended June 30, 2019, compared with $3.4 million for the six months ended June 30, 2018. The decrease in general and administrative was primarily due to decreased personnel costs, which includes a decrease in salaries and related benefits mainly from the elimination of our commercial sales and marketing team in 2018, a decrease in severance compensation, a decrease in stock compensation expense and a decrease in bonus expense of $12,000. The decreases were offset by increases in other general and administrative expenses including an increase in professional fees and an increase in corporate insurance.

Transgene Provides an Update after the Interim Futility Analysis of the PHOCUS Study of Pexa-Vec in Liver Cancer

On August 7, 2019 Transgene (Paris:TNG), a biotech company designing and developing virus-based immunotherapies for the treatment of solid tumors, reported an update on the interim futility analysis of the PHOCUS study of Pexa-Vec in liver cancer (Press release, Transgene, AUG 7, 2019, View Source [SID1234538332]). The independent Data Monitoring Committee (IDMC) of the PHOCUS trial has recommended to stop the study (see press release distributed on August 2, 2019). Transgene is currently analyzing the data of the trial it received from its partner SillaJen, notably in the context of the ongoing Phase 2 clinical trial evaluating the combination regimen of Pexa-Vec and the immunotherapy nivolumab in the same indication. The recommendation to stop the PHOCUS trial is not caused by safety issues of Pexa-Vec.

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Transgene is convinced of the great potential of oncolytic viruses (OV) as this therapeutic class displays numerous advantages that are acknowledged by the scientific and medical community. These include the ability of the viruses to infect and selectively replicate within the tumor, inducing cancer cell destruction, and to elicit a strong immune response against the tumor.

Transgene novel proprietary OV platform Invir.IO allows the arming of these viruses to trigger the expression of anticancer weapons directly in the tumor, thus increasing the efficacy of these molecules while reducing their possible side effects.

The oncolytic viruses derived from the Invir.IO platform have been designed using an optimized strain of Vaccinia Copenhagen. These carry a double deletion TK-RR-, which makes their replication more selective than viruses carrying a simple TK deletion such as Pexa-Vec. In addition, the Invir.IO platform is designed to incorporate several transgenes encoding for a range of specific anticancer weapons which involve well-established immunotherapy mechanisms such as anti-CTLA-4 antibodies.

The collaboration with AstraZeneca was formed on the merits of this platform. As described in the press release distributed on May 2, 2019, Transgene is currently working to design five new oncolytic viruses for AstraZeneca.

Philippe Archinard, PhD, Chairman and CEO of Transgene, commented: "We are obviously disappointed with the outcome of the PHOCUS study; however we remain convinced in the potential of our oncolytic virus pipeline. Our recent collaborative agreement with AstraZeneca highlights the industry interest in the multi-armed OVs that we can generate using our unique Invir.IO platform. We also expect to announce the first clinical data with TG6002 in patients with colorectal cancer later this year. In addition, we expect to announce important clinical results from our most advanced therapeutic vaccines; TG4010 in lung cancer and TG4001 in HPV-Positive head and neck cancer. With funding through to 2022, a clear strategy and novel technology platforms such as Invir.IO and myvac, Transgene is well placed to demonstrate the potential of its novel medicines designed to improve the treatment of solid tumors."

A conference call in English is scheduled on August 7, 2019, at 6:30 p.m. CET (12:30 pm EST).

Webcast link to English language conference call:
https://channel.royalcast.com/webcast/transgene/20190805_1/

Participant telephone numbers:

France: +33 (0) 1 7037 7166

United Kingdom: +44 20 3003 2666

United States: +1 202 204 1514

Confirmation code: Transgene

A replay of the call will be available on the Transgene website (www.transgene.fr) following the live event.

-Ends-

About the PHOCUS trial
The PHOCUS trial was a Phase 3 clinical trial evaluating the oncolytic immunotherapy Pexa-Vec for advanced liver cancer patients who have not received prior systemic treatment for their cancer. The study was conducted by Transgene’s partner, SillaJen.
In the PHOCUS study, patients were randomized to one of two treatment groups: one receiving Pexa-Vec followed by sorafenib and one receiving sorafenib alone. The primary objective of the study was to determine the overall survival of patients treated with Pexa-Vec, followed by sorafenib versus sorafenib alone. Secondary objectives included safety as well as assessments for tumor responses between the two groups as measured by the following endpoints: time to progression, progression-free survival, overall response rate and disease control rate.

About Pexa-Vec
Pexa-Vec (formerly JX-594/TG6006 – pexastimogene devacirepvec) is an oncolytic immunotherapeutic based on an oncolytic vaccinia virus armed with a GM-CSF gene that promotes an anti-tumor immune response. Pexa-Vec is designed to selectively target and destroy cancer cells through three different mechanisms of action: selectively destroy cancer cells through the direct lysis (breakdown) of cancer cells through viral replication, reduce the blood supply to tumors through vascular disruption, and stimulate the body’s immune response against cancer cells.
In a Phase 2 study, results of patients with advanced liver cancer showed that patients receiving the high dose had a statistically significant clinical improvement in terms of overall survival compared to the group receiving the low dose. Median overall survival was respectively 14.1 months in the high-dose group and 6.7 months in the low-dose group, which compares favorably with current approved treatments. (Heo J. et al., Nature Medicine, March 2013, doi: 10.1038/nm.3089)
Transgene has exclusive rights to develop and commercialize Pexa-Vec for the treatment of solid tumors in Europe. Its partner SillaJen, Inc. is focused on developing Pexa-Vec for the North American market and has also granted exclusive development and commercial rights to Pexa-Vec in Hong Kong and The People’s Republic of China to Lee’s Pharmaceutical.

ANI Pharmaceuticals Reports Second Quarter Results

On August 7, 2019 ANI Pharmaceuticals, Inc. ("ANI") (NASDAQ: ANIP) reported its financial results for the three and six months ended June 30, 2019 and reaffirmed its previously issued guidance for adjusted non-GAAP EBITDA and adjusted non-GAAP earnings per share, while updating its 2019 financial guidance for net revenues (Press release, ANI Pharmaceuticals, AUG 7, 2019, View Source [SID1234538349]). The Company will host its earnings conference call this morning, August 7, 2019, at 10:30 AM ET. Investors and other interested parties can join the call by dialing (866) 776-8875. The conference ID is 3823848.

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Financial Summary

(a) See ANI’s Form 10-Q filed August 7, 2019 for discussion of year-to-date results.

(b) See Table 3 for US GAAP reconciliation.

(c) See Table 4 for US GAAP reconciliation.

Arthur S. Przybyl, President and CEO, stated,

"ANI had a strong second quarter, generating net revenues of $54.4 million, an increase of 15% over the prior year period, and record adjusted non-GAAP EBITDA of $23.7 million, an increase of 24% over the prior year period. More importantly, we remain on track to file our supplemental NDA for Cortrophin Gel in the first quarter of 2020.

Generic product revenues increased 20% over the prior year period and we launched one new generic product in late June, Ranitidine capsules. We also received FDA approval for 250 mg/5 ml Vancomycin Oral Solution, which we intend to launch in the third quarter.

During the second quarter, we continued to add to our generic portfolio, through an April distribution agreement with a specialty injectable manufacturer and the June acquisition of seven development stage generic products, which expanded our pipeline of injectable drugs to six."

ANI Updates Guidance for Net Revenues for the Full Year 2019

ANI has updated its full year guidance for net revenues due to the competitive landscape of the Methylphenidate ER market. ANI’s guidance for adjusted non-GAAP EBITDA and adjusted non-GAAP earnings per diluted share remains unchanged from previously issued guidance.

Revised guidance for 2019 net revenues of $220 million to $226 million reflects sales growth of between 9% and 12% over 2018 full year actual results. Previous guidance for net revenues was $231 million to $245 million.

ANI’s estimates are based upon actual results for the six months ending June 30, 2019 and projected results for the remaining six months of the year. ANI’s full year 2019 financial guidance reflects management’s current assumptions regarding customer relationships, product pricing, prescription trends, competition, inventory levels, cost of sales, operating costs, timing of research and development spend, taxes, and the anticipated timing of future product launches and other key events. For the twelve months ending December 31, 2019, ANI is providing guidance on net revenues, adjusted non-GAAP EBITDA, and adjusted non-GAAP diluted earnings per share.

Generic Pharmaceutical Products

Second Quarter Net Revenues – Results and Update

Net revenues from sales of generic pharmaceuticals increased 20% to $36.3 million from $30.2 million in the prior period, primarily due to the launch of Ezetimibe-Simvastatin, Candesartan, and other products launched in 2018 and 2019, as well as increased unit sales of Vancomycin.

Key Generic Pipeline Product

Branded Pharmaceutical Products

Second Quarter Net Revenues – Results and Update

Net revenues from sales of branded pharmaceuticals increased 33% to $14.0 million from $10.5 million in the prior period, primarily due to sales of Arimidex and Casodex, which were launched under ANI’s label in July 2018 and sales of Atacand and Atacand HCT, which were launched under ANI’s label in October 2018. Prior to the launch under the ANI label, net revenues generated by these products was recorded via royalties received and was reported as Royalty and Other.

Key Brand Pipeline Products

Product

Vancocin for Oral Solution Update

ANI is currently advancing a commercialization effort for Vancocin for oral solution. ANI filed a prior approval supplement ("PAS") in September 2018 and it was approved in June 2019. This product will be manufactured at ANI’s site in Baudette, Minnesota and will compete in a market that currently exceeds $450 million annually.

Cortrophin Gel Re-commercialization Update

ANI continues to successfully progress our Cortrophin re-commercialization program. Significant accomplishments since the first quarter 2019 press release (dated May 9, 2019) include:

The completion of a third commercial scale batch of Corticotropin API. This batch was analytically consistent with previously manufactured batches and met all specifications. ANI expects to complete API process validation and registration stability batch manufacturing in the third quarter of 2019.
The initiation of viral clearance studies, which are expected to be completed in the third quarter of 2019.
The manufacture of two commercial scale batches of Cortrophin Gel using commercial scale API. ANI intends to complete the third and final registration batch of drug product in the third quarter of 2019.
ANI remains on track to file a supplemental NDA in the first quarter of 2020.

For further details, please see ANI’s Cortrophin Gel Re-commercialization Milestone Update in Table 5.

Contract Manufacturing

Second Quarter Net Revenues – Results and Update

Contract manufacturing revenues increased 120% to $3.7 million from $1.7 million in the prior year period, primarily due to the impact of contract manufacturing revenues from ANI’s Canadian subsidiary, ANI Pharmaceuticals Canada Inc. ("ANI Canada"), which was acquired in August 2018.

Royalty and Other

Second Quarter Net Revenues – Result and Update

Royalty and other decreased 91% to $0.4 million from $4.9 million, primarily due to the launches of Atacand, Atacand HCT, Arimidex, and Casodex under the ANI label in the second half of 2018. The net revenues from those products are now included in the net sales of branded pharmaceutical products. This decline was tempered by product development and lab services net revenues from ANI Canada.

Key Royalty Product: Yescarta

ANI is entitled to a percentage of global Yescarta net sales as well as a portion of certain product milestones, such as the recent positive opinion issued by the European Medicines Agency ("EMA") Committee for Medicinal Products for Human Use ("CHMP").

Operating Expenses

Operating expenses increased to $45.1 million for the three months ended June 30, 2019, from $40.0 million in the prior year period. The increase was primarily due to a $4.2 million increase in selling, general, and administrative expense as compared with the prior period, as a result of costs related to the new ANI Canada subsidiary, increased U.S.-based headcount and pharmacovigilance compliance costs in continued support of the expansion of our commercial portfolio, higher GDUFA and PDUFA user fees paid to the U.S. FDA, higher legal fees, and increased sales and marketing-related costs. In addition, depreciation and amortization increased by $1.2 million, primarily due to additional amortization expense associated with a March 2019 asset acquisition and a January 2019 royalty buyout payment related to a prior period asset acquisition. These increases were partially offset by a $1.0 million decrease in cost of sales.

Cost of sales as a percentage of net revenues decreased to 29% during the three months ended June 30, 2019, from 35% during same period in 2018. The decrease was primarily due to lower royalty expense resulting from a royalty buy out and lower sales of products under profit-sharing arrangements.

Net Income and Diluted Earnings per Share

Net income was $6.6 million for the three months ended June 30, 2019, as compared to net income of $2.8 million in the prior year period. The effective consolidated tax rate excluding impacts of discrete items for the three months ended June 30, 2019 was 16.8%.

Diluted earnings per share for the three months ended June 30, 2019 was $0.53, based on 12,269 thousand diluted shares outstanding, as compared to diluted earnings per share of $0.23 in the prior year period. Adjusted non-GAAP diluted earnings per share was $1.44, as compared to adjusted non-GAAP diluted earnings per share of $1.13 in the prior year period. For a reconciliation of adjusted non-GAAP diluted earnings per share to the most directly comparable GAAP financial measure, please see Table 4.

ANI Product Development Pipeline

ANI’s pipeline consists of 111 products, addressing a total annual market size of $5.2 billion, based on data from IQVIA. Of these 111 products, 106 were acquired and of these acquired products, ANI expects that at least 56 can be commercialized based on either CBE-30s or prior approval supplements filed with the FDA.

Non-GAAP Financial Measures

The Company’s fiscal 2019 guidance for adjusted non-GAAP EBITDA and adjusted non-GAAP diluted earnings per share is not reconciled to the most comparable GAAP measure. This is due to the inherent difficulty of forecasting the timing or amount of items that would be included in a reconciliation to the most directly comparable forward-looking GAAP financial measures. Because a reconciliation is not available without unreasonable effort, it is not included in this release.

Adjusted non-GAAP EBITDA

ANI’s management considers adjusted non-GAAP EBITDA to be an important financial indicator of ANI’s operating performance, providing investors and analysts with a useful measure of operating results unaffected by non-cash stock-based compensation and differences in capital structures, tax structures, capital investment cycles, ages of related assets, and compensation structures among otherwise comparable companies. Management uses adjusted non-GAAP EBITDA when analyzing Company performance.

Adjusted non-GAAP EBITDA is defined as net income/(loss), excluding tax expense, interest expense, depreciation, amortization, the excess of fair value over cost of acquired inventory, stock-based compensation expense, expense from acquired in-process research and development, gains, losses, and expenses related to the repurchase of convertible debt, expenses related to debt financing, transaction and integration expenses, and other income / expense. Adjusted non-GAAP EBITDA should be considered in addition to, but not in lieu of, net income or loss reported under GAAP. A reconciliation of adjusted non-GAAP EBITDA to the most directly comparable GAAP financial measure is provided in Table 3.

Adjusted non-GAAP Net Income

ANI’s management considers adjusted non-GAAP net income to be an important financial indicator of ANI’s operating performance, providing investors and analysts with a useful measure of operating results unaffected by purchase accounting adjustments, non-cash stock-based compensation, non-cash interest expense, depreciation and amortization, and non-cash impairment charges. Management uses adjusted non-GAAP net income when analyzing Company performance.

Adjusted non-GAAP net income is defined as net income/(loss), plus the excess of fair value over cost of acquired inventory, stock-based compensation expense, transaction and integration expenses, gains, losses, and expenses related to the repurchase of convertible debt, expenses related to debt financing, non-cash interest expense, depreciation and amortization expense, expense from acquired in-process research and development, and non-cash impairment charges, less the tax impact of these adjustments calculated using an estimated statutory tax rate. Management will continually analyze this metric and may include additional adjustments in the calculation in order to provide further understanding of ANI’s results. Adjusted non-GAAP net income should be considered in addition to, but not in lieu of, net income reported under GAAP. A reconciliation of adjusted non-GAAP net income to the most directly comparable GAAP financial measure is provided in Table 4.

Adjusted non-GAAP Diluted Earnings per Share

ANI’s management considers adjusted non-GAAP diluted earnings per share to be an important financial indicator of ANI’s operating performance, providing investors and analysts with a useful measure of operating results unaffected by purchase accounting adjustments, non-cash stock-based compensation, non-cash interest expense, depreciation and amortization, and non-cash impairment charges.

Management uses adjusted non-GAAP diluted earnings per share when analyzing Company
performance.

Adjusted non-GAAP diluted earnings per share is defined as adjusted non-GAAP net income, as defined above, divided by the diluted weighted average shares outstanding during the period, as adjusted for the dilutive effect of the convertible debt notes, when applicable. Management will continually analyze this metric and may include additional adjustments in the calculation in order to provide further understanding of ANI’s results. Adjusted non-GAAP diluted earnings per share should be considered in addition to, but not in lieu of, diluted earnings or loss per share reported under GAAP. A reconciliation of adjusted non-GAAP diluted earnings per share to the most directly comparable GAAP financial measure is provided in Table 4.

Leap Presents Positive Clinical Results for the Combination of DKN-01 plus KeytrudaÒ
and Provides DKN-01 Program Update

On August 6, 2019 Leap Therapeutics, Inc. (NASDAQ:LPTX) reported that its anti-Dickkopf-1 (DKK1) antibody, DKN-01, in combination with Merck’s anti-PD-1 antibody, KeytrudaÒ (pembrolizumab), demonstrated higher survival and objective response outcomes in patients with advanced gastroesophageal junction and gastric cancer (GEJ/GC) whose tumors expressed high levels of DKK1 (DKK1-high) (Press release, Leap Therapeutics, AUG 6, 2019, View Source [SID1234538168]). DKN-01 plus Keytruda therapy achieved over 22 weeks median progression-free survival (PFS) and nearly 32 weeks median overall survival (OS) with a 50% overall response rate (ORR) and 80% disease control rate (DCR) in patients with DKK1-high GEJ/GC who had not received prior anti-PD-1/PD-L1 therapy. Leap will host a DKN-01 Clinical Perspectives and Program Update for the investment community through a live conference call and webcast with two clinical investigators today at 8:30 AM US Eastern Time.

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"Gastric and gastroesophageal junction cancers represent a major global cancer burden with significant unmet needs, particularly in patients with advanced disease. Outside of rare microsatellite instable tumors and EBV-associated cancers the response rates to immune checkpoint inhibitors are low and median progression free survival remains short, in the range of 6-8 weeks. Oncologists and patients are eager for new therapeutic combinations and biomarkers to help predict patients most likely to benefit from a given treatment," stated Samuel J. Klempner, MD, Assistant Professor, Massachusetts General Hospital Cancer Center and Harvard Medical School.

"The responses and early survival data seen in DKK1-high patients treated with DKN-01 plus pembrolizumab are highly encouraging," commented Dr. Klempner. "This study builds on previously reported positive monotherapy and paclitaxel combination data and importantly suggests that elevated DKK-1 expression is a potential predictive biomarker. DKN-01 warrants further study in gastroesophageal cancers in combination with immune checkpoint inhibitors and with chemotherapy."

Key Findings from KEYNOTE-731 DKN-01 plus Keytruda Combination

The esophagogastric cancer clinical trial is a multipart study of DKN-01 as a monotherapy and in combination with paclitaxel or pembrolizumab. Sixty-three patients were treated with DKN-01 plus Keytruda combination therapy across all arms and dose groups of the study. Fifty-three patients had not received prior PD-1/PD-L1 therapy, and ten patients were refractory to PD-1/PD-L1 therapy. All of the patients enrolled had tumors that were microsatellite stable or unknown. Patients in the study were heavily pretreated having had received one to five prior lines of therapy, with nearly 64% having received a prior taxane regimen, 37% having received prior ramucirumab, and 24% having received prior trastuzumab. The combination therapy was well tolerated with no new safety signals.

The combination of DKN-01 and Keytruda in GEJ/GC patients demonstrated improved outcomes in patients whose tumors are DKK1-high and who were PD-1/PD-L1 naïve. DKK1-high patients experienced

over 22 weeks median PFS and nearly 32 weeks OS, with a 50% ORR and 80% DCR in ten evaluable patients. DKK1-low patients experienced nearly 6 weeks median PFS and over 17 weeks OS, with a 20% DCR in fifteen evaluable patients.

PD-L1 Combined Positive Scores (CPS) did not predict efficacy on the combination of DKN-01 plus Keytruda. In multi-variate analysis, DKK1-high status correlated with longer PFS independent of PD-L1 CPS scores. One-third of patients in the study were DKK1-high.

Among the six GEJ/GC patients who were refractory to PD-1/PD-L1 therapy, three DKK1-high patients had a best response of stable disease, whereas the three patients with DKK1-low tumors had progressive disease.

DKN-01 Clinical Perspectives Conference Call and Webcast

Samuel J. Klempner, MD, Assistant Professor, Massachusetts General Hospital Cancer Center and Harvard Medical School, will describe his experience with treating esophagogastric cancer patients in the DKN-01 study. In addition, Rebecca C. Arend, MD, Assistant Professor and Associate Scientist, Gynecologic Oncology Clinic, UAB Comprehensive Cancer Center Experimental Therapeutics Program, will discuss her experience with endometrial cancer and carcinosarcoma patients treated with DKN-01.

To access the conference call, please dial (866) 589-0108 (US/Canada Toll-Free) or (409) 231-2048 (international) and refer to conference ID 3571417. The presentation will also be webcast live and will be available under "Events & Presentations" in the Investor section of Leap’s website, View Source A replay of the webcast will be available on Leap’s website shortly after the event and will be available for a limited time.