On August 6, 2019 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, reports its financial results for the quarter ended June 30, 2019 and provides a corporate update (Press release, BioLineRx, AUG 6, 2019, View Source [SID1234538198]).

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Highlights and achievements during the second quarter 2019 and subsequent period:

Continued to advance multiple clinical trials of its lead therapeutic candidates, BL-8040 and AGI-134, and anticipates top-line data from the triple combination arm of the COMBAT/KEYNOTE-202 pancreatic cancer trial by year-end.

Received approval from the FDA for an Investigational New Drug (IND) application for AGI-134, which will enable expansion of the ongoing Phase 1/2a study, currently being carried out in the UK and Israel, to the US by the first half of 2020.

"BL-8040, our lead drug candidate, is rapidly advancing in multiple promising programs. The triple combination arm of our ongoing COMBAT/KEYNOTE-202 Phase 2 study of BL-8040, KEYTRUDA and chemotherapy in metastatic pancreatic cancer is progressing as planned, and top-line results are expected by year end. AML consolidation also remains an important indication in our BL-8040 development plan, and we, with our partners, continue to progress our BLAST Phase 2b study towards a robust interim analysis. In addition, the GENESIS Phase 3 study in stem cell mobilization, our most advanced indication and most direct path to registration, is progressing as planned with top-line results expected in the second half of next year," stated Philip Serlin, Chief Executive Officer of BioLineRx.

"Regarding our second clinical candidate, the universal anti-cancer vaccine AGI-134, we continue to advance our phase 1/2a clinical trial toward initial safety data later this year, and with the acceptance of our IND, we look forward to the next part of the study, which will assess potential efficacy. Taken together, we believe our broad development pipeline provides multiple opportunities for long-term value creation and we are diligently working toward that goal," Mr. Serlin concluded.

Upcoming Milestones

Second half of 2019

Top-line results from COMBAT/KEYNOTE-202 Phase 2 pancreatic cancer trial

Initial safety results from part 1 of Phase 1/2a trial of AGI-134

Potential interim results from Phase 2b AML consolidation study

Initiation of monotherapy basket arm of Part 2 of Phase 1/2a trial of AGI-134

2020

Progression-free survival (PFS) and overall survival (OS) data from COMBAT/KEYNOTE-202 trial in mid-2020

Top-line results from Phase 3 GENESIS registration trial in stem-cell mobilization in second half of 2020

Financial Results for the Second Quarter Ended June 30, 2019

Research and development expenses for the three months ended June 30, 2019 were $5.3 million, an increase of $0.8 million, or 18%, compared to $4.5 million for the three months ended June 30, 2018. The increase resulted primarily from higher expenses associated with the BL-8040 GENESIS and COMBAT clinical trials. Research and development expenses for the six months ended June 30, 2019 were $9.7 million, an increase of $0.1 million, or 2%, compared to $9.6 million for the six months ended June 30, 2018. The small increase resulted primarily from higher expenses associated with the BL-8040 GENESIS and COMBAT clinical trials, offset by a decrease in expenses related to BL-1230, a project which was terminated, as well as a decrease in payroll and related expenses.

Sales and marketing expenses for the three months ended June 30, 2019 were $0.2 million, a decrease of $0.1 million, or 37%, compared to $0.3 million for the three months ended June 30, 2018. The decrease resulted primarily from a decrease in payroll and related expenses. Sales and marketing expenses for the six months ended June 30, 2019 were $0.5 million, a decrease of $0.4 million, or 43%, compared to $0.9 million for the six months ended June 30, 2018. The decrease resulted primarily from a decrease in payroll and related expenses, including a one-time compensation payment in the 2018 period.

General and administrative expenses for the three months ended June 30, 2019 were $0.9 million, similar to the comparable period in 2018. General and administrative expenses for the six months ended June 30, 2019 were $1.9 million, similar to the comparable period in 2018.

The Company’s operating loss for the three months ended June 30, 2019 was $6.5 million, compared to $5.7 million for the three months ended June 30, 2018. The Company’s operating loss for the six months ended June 30, 2019 was $12.1 million, compared to $12.4 million for the comparable period in 2018.

Non-operating income for the three and six months ended June 30, 2019 primarily relates to fair-value adjustments of warrant liabilities on the Company’s balance sheet, offset by warrant offering expenses. Non-operating income for the six months ended June 30, 2018 primarily relates to fair-value adjustments of warrant liabilities on the Company’s balance sheet, as well as a capital gain from realization of the investment in iPharma.

Net financial expenses amounted to $0.3 million for the three months ended June 30, 2019 compared to net financial income of $0.3 million for the three months ended June 30, 2018. Net financial expenses for the 2019 period primarily relate to interest paid on loans, offset by investment income earned on bank deposits. Net financial income for the 2018 period primarily relates to investment income earned on bank deposits, offset by losses recorded on foreign currency hedging transactions. Net financial expenses amounted to $0.5 million for the six months ended June 30, 2019 compared to net financial income of $0.3 million for the six months ended June 30, 2018. Net financial expenses for the 2019 period primarily relate to interest paid on loans, offset by investment income earned on bank deposits. Net financial income for the 2018 period primarily relates to investment income earned on bank deposits, offset by losses recorded on foreign currency hedging transactions.

The Company’s net loss for the three months ended June 30, 2019 amounted to $5.5 million, compared with a net loss of $4.8 million for the comparable period in 2018. The Company’s net loss for the six months ended June 30, 2019 amounted to $11.6 million, compared with a net loss of $11.0 million for the comparable period in 2018.

The Company held $35.2 million in cash, cash equivalents and short-term bank deposits as of June 30, 2019.

Net cash used in operating activities was $11.1 million for the six months ended June 30, 2019, compared with net cash used in operating activities of $13.0 million for the six months ended June 30, 2018. The $1.9 million decrease in net cash used in operating activities during the six-month period in 2019, compared to the six-month period in 2018, was primarily the result of changes in operating asset and liability items in the two periods., i.e., a decrease in prepaid expenses and other receivables in 2019 versus an increase in 2018, as well as a higher decrease in accounts payable and accruals in 2018.

Net cash used in investing activities was $3.1 million for the six months ended June 30, 2019, compared to net cash provided by investing activities of $10.8 million for the six months ended June 30, 2018. The changes in cash flows from investing activities relate primarily to investments in, and maturities of, short-term bank deposits and the realization of the investment in iPharma in 2018.

Net cash provided by financing activities was $15.7 million for the six months ended June 30, 2019, compared to net cash provided by financing activities of $2.8 million for the six months ended June 30, 2018. The increase in cash flows from financing activities reflects the underwritten public offering completed in February 2019.

Conference Call and Webcast Information

BioLineRx will hold a conference call today, August 6, 2019 at 10:00 a.m. EDT. To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0644 internationally. The call will also be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until August 8, 2019; please dial +1-888-295-2634 from the U.S. or +972-3-925-5904 internationally.

On August 6, 2019 Dova Pharmaceuticals, Inc. (Nasdaq: DOVA), a pharmaceutical company focused on acquiring, developing, and commercializing drug candidates for diseases where there is a high unmet need, reported its operating and financial results for the second quarter ended June 30, 2019 (Press release, Dova Pharmaceuticals, AUG 6, 2019, View Source [SID1234538197]).

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"The last few months have been a transformational period in Dova’s history, with FDA approval and launch of our thrombopoietin receptor agonist (TPO-RA) DOPTELET for ITP, European approval of DOPTELET for severe thrombocytopenia in adult patients with CLD, and an expanded co-promotion partnership with Salix for CLD. These accomplishments have strengthened our position as a growing leader in the treatment of thrombocytopenia," said Dr. David Zaccardelli, President and Chief Executive Officer of Dova. "Beyond ITP, we look forward to results of our further indication expansion activities, with top-line data from our Phase 3 trial of DOPTELET for the treatment of chemotherapy induced thrombocytopenia (CIT) expected in the first half of 2020."

Second Quarter and Subsequent Highlights

DOPTELET ITP Approval – In late June, the FDA approved a supplemental New Drug Application (sNDA) that expanded the use of DOPTELET to include the treatment of thrombocytopenia in adult patients with ITP who have had an insufficient response to a previous treatment.

DOPTELET ITP Launch – In mid-July, DOPTELET was launched commercially for the ITP indication. A sales force of approximately 60 sales professionals with extensive Hematology/

Oncology experience, are now calling on roughly 6,000 HCPs that represent 96% of the ITP patient potential in the United States.

DOPTELET Differentiating Characteristics in ITP –

◦
DOPTELET is the only oral TPO-RA approved for use in adult patients with ITP that does not have a boxed warning for hepatotoxicity and does not require routine liver enzyme monitoring.

DOPTELET is the only oral TPO approved for use in adult patients with ITP without food-type restrictions.

DOPTELET, as an oral tablet, does not require weekly visits to a health care provider’s office for subcutaneous injections.

DOPTELET increased platelet counts to >50,000 in 66% of ITP patients by Day 8 of treatment in a Phase 3 trial.

DOPTELET ITP Publications – Dova presented four abstracts on the safety and efficacy of avatrombopag from studies in patients with ITP at the 65th International Society on Thrombosis and Haemostasis (ISTH) Annual Scientific Meeting, held July 6-10, 2019, in Melbourne, Australia. These included efficacy data in patients who had previously received another TPO-RA, and the results of a dosing model study.

DOPTELET CLD MAA – In late June, the EC granted marketing authorization for DOPTELET for the treatment of severe thrombocytopenia in adult patients with CLD who are scheduled to undergo an invasive procedure. Further, Dova continues to advance partnering discussions to commercialize DOPTELET in markets outside the US.

Salix Partnership – Dova strategically expanded its co-promotion partnership for CLD in the United States with Salix. Starting on July 1, 2019, in addition to the gastroenterology, colorectal surgery, and proctology segments, Salix has the exclusive right to co-promote DOPTELET for the CLD indication to the hepatology and interventional radiology segments. Dova will continue to pay Salix a commission based on a percentage of net sales in these specialties, which beginning on July 1, 2019, is expected to be in the mid-thirties. In addition, the co-promotion agreement was extended to September 2023.

Phase 3 CIT Clinical Trial – Dova continues to enroll patients in a Phase 3 trial evaluating the safety and efficacy of DOPTELET for the treatment of CIT and plans to report top-line results in the first half of 2020.

DOPTELET CLD Launch Highlights

Net product sales for DOPTELET were $3.5 million for the second quarter.
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Inventory held by specialty pharmacies in Dova’s contracted network decreased by approximately $450,000 from March 31, 2019 to June 30, 2019.
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Prescription referrals increased approximately 7% in the second quarter of 2019 compared to the first quarter of 2019.
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As expected, sales were negatively affected by competitive pricing pressure and payer restrictions in the second quarter of 2019, which Dova is addressing with the new pricing for DOPTELET implemented mid-July 2019.

Early in the second quarter of 2019, a new marketing strategy for the CLD indication was launched, including revised product positioning, messaging, and a new action-oriented brand campaign.

From launch through June 30, 2019, a total of 1,368 health care professionals have prescribed DOPTELET to their patients, with an increasing number of repeat prescribers.

More than 9,200 calls were conducted, reaching more than 4,000 unique health care providers during the second quarter of 2019.

For prescriptions in the second quarter of 2019 that have gone through the adjudication process with payers, 77% of those prescriptions were approved. On average, the time to decision for a referral was 7.4 business days in the second quarter of 2019.

Second Quarter Financial Results

Net product sales for DOPTELET were $3.5 million for the second quarter of 2019. Dova recognizes revenue when products are delivered to its specialty pharmacy partners.

Dova reported a net loss of $17.1 million for the second quarter of 2019, compared to a net loss of $19.7 million for the same period in 2018.

Cost of product sales for the second quarter of 2019 were $0.4 million, which consisted of the cost of inventory, royalty payments and certain distribution and overhead costs.

Research and development expenses were $4.5 million in the second quarter of 2019, compared to $4.5 million for the same period in 2018.

Selling, general and administrative expenses were $15.5 million in the second quarter of 2019, compared to $18.6 million for the same period in 2018. The second quarter of 2018 saw higher expense levels as a result of the full sales staff being onboard for most of the second quarter of 2018 while in 2019 the full sales staff for the launch of DOPTELET for the ITP indication were hired at the end of the second quarter and early third quarter.

As of June 30, 2019, Dova had $76.8 million in cash and equivalents compared to $104.6 million as of December 31, 2018. Additionally, on May 6, 2019, Dova entered into an amended and restated loan agreement with Silicon Valley Bank that extended the interest only period of the existing $20 million loan facility by 12 months and provided additional potential borrowings of $30 million upon achieving certain clinical and revenue milestones. In July 2019, subsequent to the quarter end, Dova drew $10 million on this loan agreement upon approval in ITP.

Company to Host Conference Call

Dova will host a conference call today, August 6, 2019 at 9:00 a.m. ET to discuss second quarter ended June 30, 2019 financial results as well as recent operational highlights. A question-and-answer session will follow Dova’s remarks.

To participate on the live call, please dial 866-550-8145 (domestic) or +1-430-775-1344 (international) and provide the conference ID 7784728 five to 10 minutes before the start of the call.

A live audio webcast of the call will also be available via the "Investor Relations" page of the Dova website, www.dova.com. Please log on through Dova’s website approximately 10 minutes before the scheduled start time. A replay of the webcast will be archived on Dova’s website for 90 days following the call.

Indication and Important Safety Information
INDICATION
DOPTELET (avatrombopag) is indicated for the treatment of thrombocytopenia in adult patients with:

Chronic liver disease who are scheduled to undergo a procedure.

Chronic immune thrombocytopenia who have had an insufficient response to a previous treatment.
IMPORTANT SAFETY INFORMATION FOR DOPTELET
Warnings and Precautions
DOPTELET is a thrombopoietin (TPO) receptor agonist and TPO receptor agonists have been associated with thrombotic and thromboembolic complications in patients with chronic liver disease or chronic immune thrombocytopenia. Portal vein thrombosis has been reported in patients with chronic liver disease, and thromboembolic events (arterial and venous) have been reported in patients with chronic immune thrombocytopenia treated with TPO receptor agonists.
In clinical trials, 0.2% (1/430) of patients with chronic liver disease treated with DOPTELET developed a treatment-emergent event of portal vein thrombosis. In clinical trials in patients with chronic immune thrombocytopenia, 7% (9/128) of patients treated with DOPTELET developed a thromboembolic event.
Consider the potential increased thrombotic risk when administering DOPTELET to patients with known risk factors for thromboembolism, including genetic prothrombotic conditions (Factor V Leiden, Prothrombin 20210A, Antithrombin deficiency or Protein C or S deficiency).
DOPTELET should not be administered to patients with chronic liver disease or chronic immune thrombocytopenia in an attempt to normalize platelet counts. Follow the dosing guidelines to achieve target platelet counts. Monitor patients receiving DOPTELET for signs and symptoms of thromboembolic events and institute treatment promptly.
Contraindications: None
Drug Interactions
Dose adjustments are recommended for patients with chronic immune thrombocytopenia taking moderate or strong dual CYP2C9 and CYP3A4 inducers or inhibitors.
Adverse Reactions
The most common adverse reactions (≥3%) in patients with chronic liver disease were: pyrexia, abdominal pain, nausea, headache, fatigue, and peripheral edema.

The most common adverse reactions (≥10%) in patients with chronic immune thrombocytopenia were: headache, fatigue, contusion, epistaxis, upper respiratory tract infection, arthralgia, gingival bleeding, petechiae, and nasopharyngitis.

On August 6, 2019 Evelo Biosciences, Inc. (Nasdaq:EVLO), a biotechnology company developing oral biologics that act on cells in the small intestine with systemic therapeutic effects, reported positive interim Phase 1b clinical data and provided second quarter 2019 financial results (Press release, Evelo Biosciences, AUG 6, 2019, View Source [SID1234538196]).

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"Evelo is harnessing the therapeutic potential of cells in the small intestine which play a central role in governing the immune, metabolic, and nervous systems. Our platform has generated a diversified portfolio of oral biologic candidates that act on these cells to drive systemic effects relevant to the potential treatment of a broad range of diseases," said Simba Gill, Ph.D., chief executive officer of Evelo. "Today’s positive interim data show, for the first time in patients, clinical and biomarker responses that support our core scientific hypothesis and our vision of developing effective, oral, safe, and affordable medicines for people with major chronic diseases and cancer. We look forward to advancing into later stage trials and continuing to expand our clinical portfolio."

Inflammation: Interim Clinical Data Highlights and Anticipated Milestones

About the EDP1815 and EDP1066 Phase 1b clinical trials
Each of EDP1815 and EDP1066 are in ongoing signal-finding Phase 1b clinical trials in mild to moderate psoriasis and atopic dermatitis. Patients in these trials are randomized 2:1 to receive daily, oral administration of active drug, or placebo, for 28 days. The primary endpoint of these trials is safety and tolerability. Prospectively defined secondary and exploratory endpoints include clinical measures of disease, cellular histological biomarkers and blood immune cell biomarkers taken from biopsies and blood samples, respectively, at the start and end of the 28-day dosing period.

EDP1815 – positive interim Phase 1b clinical data at low dose in mild to moderate psoriasis patients

In a separate press release this morning Evelo reported positive EDP1815 clinical data from an initial 12-patient cohort in its ongoing trial in mild to moderate psoriasis.

EDP1815 was well tolerated with no overall difference reported from placebo.

Patients dosed once per day for 28 days with 550mg (1x dose) of the enteric capsule formulation of EDP1815 showed a statistically significant (p>0.05) reduction in mean lesion severity score (LSS) at 28 days of 2 points, compared to a mean increase of 0.25 points in patients who received placebo. LSS reductions over the dosing period of patients dosed with EDP1815 ranged from 0-67 percent. LSS, a secondary endpoint, is a component of the Psoriasis Area and Severity Index (PASI) score and measures redness, thickness, and scaling of an individual psoriatic lesion across the dosing period and is a sensitive clinical measure for patients with mild to moderate disease.

Analysis of the basal epithelium mitotic count, a secondary endpoint and cellular driver of psoriasis pathology, showed a mean reduction over the dosing period of 2.25 cells/mm2 in patients who received EDP1815 compared to no change in patients receiving placebo. Lower basal epithelium mitotic counts indicate a reduction of psoriasis pathology.

In an analysis of the change over the dosing period of blood immune cell cytokine production following stimulation with lipopolysaccharide, an exploratory endpoint, the EDP1815 dosed patient group showed a reduction in cytokine production indicative of a systemic anti-inflammatory response, compared to no reduction in the placebo group.

Based on these data, Evelo plans to advance EDP1815 into Phase 2 in early 2020. This placebo-controlled dose and formulation optimization trial will investigate daily dosing of EDP1815 in mild to moderate psoriasis patients over 24 weeks.

Enrollment is underway in the ongoing Phase 1b clinical trial of an additional cohort of mild to moderate psoriasis patients to be dosed at a 2.76g (5x) dose of the enteric capsule formulation. Data from this cohort are expected in the fourth quarter of 2019.

EDP1066 – positive interim Phase 1b biomarker data at high dose in mild to moderate psoriasis patients

Today, Evelo reports positive EDP1066 biomarker data at the high dose from its ongoing Phase 1b trial in patients with mild to moderate psoriasis.

EDP1066 was well tolerated with no overall difference reported from placebo.

In an analysis of the change over the dosing period of blood immune cell cytokine production following stimulation by lipopolysaccharide, an exploratory endpoint, patients who received a 3.3g (5x) dose of EDP1066 showed a reduction in cytokine production consistent with a pharmacodynamic effect. No reduction was observed in patients receiving a 660mg (1x) dose of EDP1066 or placebo.

No effects were observed in the secondary endpoints of clinical measures of disease or cellular histological biomarkers at either the 660mg or 3.3g dose of EDP1066.

Evelo is focusing the current EDP1066 Phase 1b trial on investigating the activity of a new formulation, which was up to 30-fold more potent in preclinical models, in a cohort of mild to moderate atopic dermatitis patients. Evelo expects to report data from this cohort in the first quarter of 2020.

Given the EDP1815 data, Evelo will not develop EDP1066 any further in psoriasis.

Oncology: Clinical Studies and Anticipated Milestones

EDP1503 – Phase 1/2

Evelo is conducting a Phase 1/2 clinical trial of EDP1503 in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, in microsatellite stable colorectal cancer, triple-negative breast cancer, and patients with other tumor types that have relapsed on prior PD-1/L1 inhibitor treatment. Initial clinical data is expected in the first half of 2020.

EDP1503 – Phase 2a

The University of Chicago is conducting a Phase 2a investigator-sponsored clinical trial of EDP1503 in combination with KEYTRUDA in naive melanoma patients and melanoma patients who have relapsed on prior PD-1/L1 inhibitor treatment. Evelo will no longer be providing guidance as to timing related to this investigator-sponsored trial.

Business Highlights

In June 2019, Evelo appointed Jose-Carlos Gutiérrez-Ramos, Ph.D. to its board of directors. Dr. Gutiérrez-Ramos brings to the board significant experience in research, clinical development and company building over a long career in senior leadership roles at major pharmaceutical and biotech companies. Dr. Gutiérrez-Ramos has served as chief executive officer and president of Cogen Immune Medicine, Inc., a biotechnology company, since August 2018. Dr. Gutiérrez-Ramos has also served as a venture partner at Flagship Pioneering since 2018. From 2015 to May 2018 he served as chief executive officer and president of Synlogic, Inc. Prior to joining Synlogic, Dr. Gutiérrez-Ramos was group senior vice president of Worldwide Research and Development and global head of Biotherapeutics Research and Development at Pfizer, Inc. Dr. Gutiérrez-Ramos received a B.S. from Universidad Complutense de Madrid and his Ph.D. in immunochemistry from the Universidad Autonoma de Madrid.

In July 2019, Evelo entered into a loan and security agreement with K2 HealthVentures (K2HV). Under the terms of the agreement, Evelo can borrow up to $45 million subject to certain time conditions and clinical development milestones. On closing, Evelo borrowed $20 million; the funds were used to fully repay its $15 million loan facility with Pacific Western Bank and for general corporate purposes.

In July 2019, Evelo entered into a collaboration agreement with Sacco S.r.l. (Sacco), an existing contract manufacturing partner. Under the terms of this 5-year agreement, Sacco will manufacture and supply single strain, non-genetically modified microbes for oral delivery or oral use in pharmaceutical products exclusively for Evelo, with the exception of pre-existing products for pre-existing customers. This collaboration is consistent with Evelo’s manufacturing strategy of combining best-in-class manufacturing partners with internal manufacturing capacity and deep internal expertise in process development and formulation.

Second Quarter 2019 Financial Results

Cash Position: As of June 30, 2019, cash, cash equivalents and investments were $113.5 million, as compared to $178.9 million as of June 30, 2018 and $129.4 million as of March 31, 2019. This decrease was due to cash used to fund operating activities and capital expenditures. Evelo expects that its cash, cash equivalents and investments will enable it to fund its planned operating expenses and capital expenditure requirements, including the planned EDP1815 Phase 2 clinical trial, into the fourth quarter of 2020.

Research and Development Expenses: R&D expenses were $15.5 million for the three months ended June 30, 2019, compared to $10.2 million for the three months ended June 30, 2018. The increase of $5.3 million was due primarily to increases in costs related to Evelo’s inflammation and oncology clinical development programs, and research platform expenses, as well as increased personnel costs.

General and Administrative Expenses: G&A expenses were $5.9 million for the three months ended June 30, 2019, compared to $5.1 million for the three months ended June 30, 2018. The increase of $0.8 million was due primarily to increased personnel costs and professional and consulting fees necessary to support Evelo’s growing organization and corporate operational activities.

Net Loss: Net loss attributable to common stockholders was $20.9 million for the three months ended June 30, 2019, or $(0.65) per basic and diluted share, as compared to a net loss attributable to common stockholders of $16.7 million for the three months ended June 30, 2018, or $(0.85) per basic and diluted share.

Conference Call
Evelo will host a conference call and webcast to discuss these data and the second quarter financial results today at 8:30 a.m. ET. To access the call please dial 866-795-3242 (domestic) and 409-937-8909 (international) and provide the passcode 6380636. A live webcast of the call, including an accompanying slide presentation, will be available on the Investors sections of the Evelo website at www.evelobio.com. The archived webcast will be available approximately two hours after the conference call and will be available for 30 days following the call.

On August 6, 2019 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused pharmaceutical company, reported financial results for the second quarter 2019 (Press release, Karyopharm, AUG 6, 2019, View Source [SID1234538195]). In addition, Karyopharm highlighted select corporate milestones, including an update regarding the initial commercial launch of XPOVIO, and provided an overview of its key clinical development programs.

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"Our second quarter progress was followed by a transformational milestone: the U.S. Food and Drug Administration (FDA) granting accelerated approval of oral XPOVIO (www.XPOVIO.com), indicated for patients with heavily pre-treated multiple myeloma," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "The commercial launch of XPOVIO, our first approved product, is off to a strong start with encouraging early prescribing trends from both academic and community-based physicians throughout the U.S. While we are laser-focused on the success of the initial launch, we remain deeply committed to serving the future needs of patients well beyond those directly indicated in the U.S. accelerated approval. To that end, we eagerly await the clinical trial results from the ongoing Phase 3 BOSTON study and continue to support the evaluation of a Marketing Authorization Application (MAA) of selinexor currently under review by the European Medicines Agency (EMA). And finally, we expect to rapidly advance the regulatory filings for selinexor in both the U.S. and Europe requesting accelerated and conditional approval, respectively, for patients with relapsed or refractory diffuse large B-cell lymphoma."

Second Quarter 2019 Highlights and Recent Progress

Selinexor in Multiple Myeloma

XPOVIO (selinexor) Receives Accelerated Approval from the FDA. On July 3, 2019, the FDA approved oral XPOVIO, Karyopharm’s first-in-class, nuclear export inhibitor. XPOVIO was approved in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. XPOVIO is the first of a novel drug class designated selective inhibitors of nuclear export (SINE) and is the first ever nuclear export inhibitor approved for human use. The first indication is approved under accelerated approval based on response rate. The ongoing Phase 3 BOSTON study will serve as the confirmatory trial for the accelerated approval of XPOVIO. As with all accelerated approvals, continued approval the treatment of myeloma may be contingent upon verification and description of clinical benefit in a confirmatory trial.

XPOVIO Commercial Launch Underway in the U.S. On July 9, 2019, XPOVIO became commercially available to patients in the U.S. The commercial launch of XPOVIO is being supported by approximately 70 Karyopharm sales representatives and nurse liaisons as well as an extensive patient and healthcare provider support program called KaryForwardTM. Karyopharm’s commercial efforts are also being supplemented by patient support initiatives coordinated by our dedicated network of participating specialty pharmacy providers. Early prescribing trends are encouraging with robust demand from both academic and community-based physicians throughout the U.S. with early prescriptions being filled for patients with Medicare and commercial insurance coverage.

European Medicines Agency (EMA) Validates Marketing Authorization Application (MAA). In January 2019, Karyopharm submitted a MAA to the EMA requesting conditional approval for selinexor, in combination with dexamethasone, as a new treatment for patients with heavily pretreated multiple myeloma based on the results of the Phase 2b STORM study. As a customary part of the MAA review process, Karyopharm received the consolidated list of questions from EMA in early May 2019 and received additional feedback, including the integrated inspection report, based on routine site audits and other activities. The Company promptly addressed the questions and feedback with EMA and the evaluation process of the MAA is ongoing. The Company expects to receive a decision on the application by the end of 2019 or early 2020.

Pivotal Phase 3 BOSTON Study in Progress. Karyopharm’s pivotal, randomized Phase 3 BOSTON study is progressing and patient enrollment is complete. Top-line data are expected by the end of 2019 or early 2020 contingent upon the occurrence of progression-free survival (PFS) events, the primary endpoint of the study. The BOSTON study is evaluating 100mg of selinexor dosed once weekly in combination with the proteasome inhibitor Velcade (bortezomib) (once weekly) and low dose dexamethasone (SVd), compared to standard twice weekly Velcade and low dose dexamethasone (Vd) in patients with multiple myeloma who have had one to three prior lines of therapy. Data from the BOSTON study, if positive, is expected to be used to support regulatory submissions to the FDA and EMA requesting the use of selinexor in patients with multiple myeloma who have received at least one prior therapy.

New and Updated Phase 1b/2 STOMP Data Presented at European Hematology Association (EHA) (Free EHA Whitepaper) 2019 Annual Meeting. Three selinexor abstracts highlighting new and updated clinical data from patients receiving a combination of selinexor and standard of care myeloma drugs were presented in June at the EHA (Free EHA Whitepaper) 2019 Annual Meeting. Specifically, clinical data from the Kyprolis (carfilzomib), Darzalex (daratumumab) and Pomalyst (pomalidomide) arms of the Phase 1b/2 STOMP study were presented. For the Kyprolis arm, once weekly oral selinexor in combination with low dose dexamethasone demonstrated a 78% overall response rate (ORR) in patients with heavily pre-treated, Kyprolis-naïve multiple myeloma. For the Darzalex arm, once weekly oral selinexor in combination with low dose dexamethasone demonstrated a 73% ORR in patients with heavily pre-treated, Darzalex-naïve multiple myeloma. And finally, for the Pomalyst arm, once weekly oral selinexor in combination with low dose dexamethasone demonstrated a 57% ORR in patients with Revlimid (lenalidomide)-relapsed or -refractory, Pomalyst-naïve multiple myeloma with PFS of 12.2 months. Across all 3 arms of the STOMP study presented, the most common treatment-related adverse events (AEs) were cytopenias, along with gastrointestinal and constitutional symptoms; most were manageable with dose modifications and/or standard supportive care.
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)

NDA and MAA Expected to be Submitted between Q4 2019 and Q1 2020. Following the positive results from the Phase 2b SADAL study that were first presented at the America Society of Hematology 2018 Annual Meeting and then updated in June at the 2019 International Conference on Malignant Lymphoma, Karyopharm expects to submit a New Drug Application (NDA) to the FDA and an MAA to the EMA requesting accelerated and conditional approval of selinexor, respectively, as a treatment for patients with relapsed or refractory DLBCL after at least two prior multi-agent therapies and who are ineligible for stem cell transplantation including CAR-T (chimeric antigen receptor modified T cell) therapy. In addition to Orphan Drug Designation, selinexor was granted Fast Track designation by the FDA in 2018.
Selinexor in Solid Tumors

Ongoing Phase 3 Portion of the Phase 2/3 SEAL Study in Liposarcoma. Karyopharm previously reported positive results from the Phase 2 portion of the randomized, blinded Phase 2/3 SEAL study evaluating single-agent selinexor versus placebo in patients with previously treated, advanced unresectable dedifferentiated liposarcoma. Enrollment is currently ongoing in the Phase 3 portion of the SEAL study. Assuming a positive outcome on the primary endpoint of PFS, the Company intends to use the data from the SEAL study to support NDA and MAA submissions requesting approval for selinexor for patients with advanced unresectable dedifferentiated liposarcoma. Top-line data from the Phase 3 portion of the SEAL study are anticipated in 2020.

Company-Sponsored Phase 3 SIENDO Study Evaluating Selinexor as Maintenance Therapy in Endometrial Cancer Now Underway. During the first quarter of 2019, an Investigational New Drug Application (IND) was submitted and accepted by the FDA for a randomized, blinded Phase 2/3 study evaluating selinexor versus placebo as a maintenance therapy in patients with advanced or recurrent endometrial cancer following one prior platinum-based treatment. There are currently no approved therapies to treat patients with advanced or recurrent endometrial cancer in the maintenance setting. The primary endpoint of the SIENDO study is PFS. This study is being led by Professor Ignace Vergote, MD, PhD, Head of the Department of Obstetrics and Gynaecology and Gynaecologic Oncology at the Catholic University of Leuven, Belgium. Karyopharm is targeting enrollment completion for SIENDO in 2020.

Updated Phase 2 KING Data in Glioblastoma Presented at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 Annual Meeting. An abstract highlighting updated efficacy and safety results from the Phase 2 KING study evaluating single-agent selinexor in patients with recurrent glioblastoma was presented in June at the ASCO (Free ASCO Whitepaper) 2019 Annual Meeting. The KING study evaluated three different dosing schedules with selinexor (50mg/m2 twice per week, 60mg twice per week and 80mg once per week) and the reported results followed completion of accrual in the non-surgical cohorts in the study (n=68). Based on the efficacy and tolerability results from the study, the 80mg once per week dosing regimen is recommended for further evaluation. Of the 30 patients treated in the 80mg dosing cohort, the overall response rate reported was 10%, with 19% of patients achieving a 6-month PFS rate and 30% of patients achieving a 6-cycle PFS rate. The most common non-hematologic AEs in this cohort were nausea, fatigue, anorexia, and vomiting, all of which were Grade 1 or 2 events. The most common treatment-related hematological AEs were primarily Grade 1 and 2 and included leukopenia, neutropenia, anemia, and thrombocytopenia. There was one case (3%) of Grade 4 treatment-related lymphopenia and no Grade 5 treatment-related AEs were reported.
Corporate Updates

Founder Sharon Shacham Receives New York Intellectual Property Law Association (NYIPLA) Inventor of the Year Award. In May, Karyopharm’s founder, President and Chief Scientific Officer, Sharon Shacham, PhD, MBA, received the esteemed NYIPLA 2019 "Inventor of the Year" award. Dr. Shacham was recognized for her scientific research that led to the development of oral selinexor and related compounds. Past winners of this award have included the inventors of CAR-T therapy, Gleevec, Valium, LASIK laser vision correction and Priceline.com, among many others.
Second Quarter 2019 Financial Results

Cash, cash equivalents and investments as of June 30, 2019, including restricted cash, totaled $217.9 million, compared to $330.9 million as of December 31, 2018.

License and other revenue for the quarter ended June 30, 2019 was $9.5 million, compared to $19.9 million for the quarter ended June 30, 2018, both of which were primarily related to the Company’s license agreements with Antengene and Ono, respectively.

For the quarter ended June 30, 2019, research and development expense was $26.5 million, compared to $44.7 million for the quarter ended June 30, 2018. Karyopharm expects research and development expense to be relatively consistent for the remainder of 2019 compared to the second quarter of 2019. For the quarter ended June 30, 2019, general and administrative expense was $24.7 million compared to $9.5 million for the quarter ended June 30, 2018. The increase in general and administrative expenses compared to the prior year period was due primarily to the hiring of the Karyopharm commercial team and related commercial launch preparation activities to support the U.S. commercial launch of XPOVIO.

Karyopharm reported a net loss of $43.4 million, or $0.71 per share, for the quarter ended June 30, 2019, compared to a net loss of $33.7 million, or $0.60 per share, for the quarter ended June 30, 2018. Net loss includes non-cash stock-based compensation expense of $4.1 million and $4.4 million for the quarters ended June 30, 2019 and June 30, 2018, respectively.

2019 Financial Outlook

Based on its current operating plans, Karyopharm expects its non-GAAP operating expenses, which excludes stock-based compensation expense, for the full year 2019 to be in the range of $200 million to $215 million. The Company expects that its existing cash, cash equivalents and investments will be sufficient to fund its operations into the second half of 2020. Additional key activities expected in 2019 include supporting the ongoing multiple myeloma regulatory filing for selinexor in Europe, progressing the pivotal Phase 3 BOSTON study in multiple myeloma and potentially submitting an NDA and MAA, in the U.S. and Europe, respectively, in DLBCL.

Non-GAAP Financial Information and Other Disclosures

Karyopharm uses a non-GAAP financial measure, non-GAAP operating expense, to provide operating expense guidance. Karyopharm believes this non-GAAP financial measure is useful to investors because it provides greater transparency regarding Karyopharm’s operating performance as it excludes non-cash stock compensation expense. This non-GAAP financial measure should not be considered a substitute or an alternative to GAAP total operating expense and should not be considered a measure of Karyopharm’s liquidity. Instead, non-GAAP operating expense should only be used to supplement an understanding of Karyopharm’s operating results as reported under GAAP. Karyopharm has not provided GAAP reconciliation for its forward-looking non-GAAP annual operating expense because Karyopharm cannot reliably predict without unreasonable efforts the timing or amount of the factors that substantially contribute to the projection of stock compensation expense, which is excluded from the forward-looking non-GAAP financial measure.

Conference Call Information

Karyopharm will host a conference call today, Tuesday, August 6, 2019, at 8:30 a.m. Eastern Time, to discuss the second quarter 2019 financial results, recent accomplishments, clinical developments and business plans. To access the conference call, please dial (855) 437-4406 (local) or (484) 756-4292 (international) at least 10 minutes prior to the start time and refer to conference ID 5550468. A live audio webcast of the call will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived webcast will be available on the Company’s website approximately two hours after the event.

Important Safety Information

The most common adverse reactions observed in patients treated with XPOVIO (incidence ≥20%) are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea, and upper respiratory tract infection.

The treatment discontinuation rate due to adverse reactions was 27%; 53% of patients had a reduction in the XPOVIO dose, and 65.3% had the dose of XPOVIO interrupted. The most frequent adverse reactions requiring permanent discontinuation in 4% or greater of patients who received XPOVIO included fatigue, nausea, and thrombocytopenia. The rate of fatal adverse reactions was 8.9%.

The full Prescribing Information for XPOVIO is available at www.XPOVIO.com.

On August 6, 2019 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage biopharmaceutical company focused on creating innovative cancer therapies, reported financial results for the second quarter ended June 30, 2019 and provided corporate updates (Press release, Arcus Biosciences, AUG 6, 2019, View Source [SID1234538193]).

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"For our lead program, AB928, the first dual adenosine receptor antagonist designed for use in oncology, we have demonstrated excellent safety, maximal receptor coverage, and PK/PD correlation in three different combination regimens. This has enabled broad Phase 1b expansion across multiple tumor types, now including prostate cancer, and we look forward to reporting initial results in mid-2020," said Terry Rosen, Ph.D., Chief Executive Officer of Arcus. "Arcus’s emphasis on selecting science-driven clinical combinations, adaptive clinical design, and an early commitment to clinical and commercial integration provide a framework that enables us to be well positioned to maximize clinical and commercial value from our pioneering drug discovery efforts in the adenosine space and potentially best-in-class molecules."

Recent Corporate Highlights

In addition to the identification of the recommended dose for expansion (RDE) for AB928 in combination with AB122, identified 150 mg once a day as the RDE for two additional combination regimens:

AB928 with pegylated liposomal doxorubicin (PLD, Doxil)

AB928 with mFOLFOX

Initiated broad Phase 1b expansions for AB928 in combinations with AB122 and/or chemotherapy across multiple tumor types.

This expansion includes metastatic castration resistant prostate cancer (mCRPC) across multiple lines of therapy. The company also plans to explore additional combinations across multiple lines of therapy in mCRPC.

Received IND clearance to initiate a biomarker-selected trial of single-agent AB122 in advanced solid tumors, in collaboration with Strata Oncology, using Strata’s proprietary biomarkers which, using observational study data, have demonstrated potential predictive power for anti-PD-1 efficacy across multiple tumor types.

Reported initial PK data from the healthy volunteer Phase 1 study of AB680, the first small-molecule CD73 inhibitor to enter the clinic, which support an every-two-weeks (Q2W) dosing schedule. Received IND clearance to initiate a Phase 1/1b trial of AB680, in combination with AB122 and chemotherapy, in first-line metastatic pancreatic cancer.

Announced the appointment of Eric Hoefer to Chief Commercial Officer. During the span of Mr. Hoefer’s 20-year career in biopharma, he has been instrumental to the development and commercialization of 15 new medicines, including Avastin, Tarceva, Tecentriq, and Imfinzi. Mr. Hoefer was most recently at AstraZeneca, where he led Immuno-oncology (IO) Global Marketing.

Anticipated Upcoming (2H 2019) Milestones

AB928 (dual adenosine receptor antagonist):

Initiate Phase 1 safety dose-escalation in combination with PLD and IPI-549, a phosphoinositide-3-kinase-gamma (PI3Kg) inhibitor, in triple-negative breast cancer (TNBC) in collaboration with Infinity Pharmaceuticals.

Initiate Phase 1b expansion in combination with AB122, carboplatin and pemetrexed in EGFR-mutated non-small cell lung cancer (NSCLC) patients who have failed tyrosine kinase inhibitor (TKI) therapy.

Present additional safety, PK/PD and translational data from the Phase 1 safety dose-escalation portion of the AB928 combination trials at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Meeting at the end of September in Barcelona, Spain.

AB680 (small-molecule CD73 inhibitor):

Initiate Phase 1 safety dose-escalation in combination with AB122, gemcitabine (Gemzar) and nab-paclitaxel (Abraxane) in patients with first-line metastatic pancreatic cancer.

AB122 (anti-PD-1 antibody):

Initiate a tumor-type agnostic biomarker-selected trial of single-agent AB122 in advanced solid tumors in collaboration with Strata Oncology.

AB154 (anti-TIGIT antibody):

Report preliminary safety and PK/PD data from the Phase 1 safety dose-escalation and initiate an expansion study in combination with AB122 in NSCLC.

Discovery Programs:

Identify a potentially best-in-class clinical development candidate targeting HIF-2α.

Please refer to Arcus’s pipeline at www.arcusbio.com for the company’s most current pipeline and development plans.

Financial Results for the Second Quarter 2019

Cash, cash equivalents and investments in marketable securities were $224.4 million as of the second quarter ended June 30, 2019, compared to $243.1 million at March 31, 2019. The decrease was primarily due to the utilization of cash to fund our operations. Based on our current operating plans, we anticipate that our cash, cash equivalents and investments in marketable securities will be sufficient to fund operations into 2021.

Revenues: Collaboration and license revenue for the second quarter ended June 30, 2019 was $1.8 million, compared to $1.3 million for the same period in 2018. The increase in revenue was primarily attributable to the impact of our adoption of Accounting Standards Codification Topic 606, Revenue from Contracts with Customers (ASC 606). Under ASC 606, additional revenue was recognized from our option and license agreement with Taiho Pharmaceutical due to remeasurement of the initial transaction price upon adoption of the new standard. Collaboration and license revenue for the six months ended June 30, 2019 was $3.5 million, compared to $2.5 million for the same period in 2018.

R&D Expenses: Research and development expenses for the second quarter ended June 30, 2019 were $25.0 million, compared to $13.7 million for the same period in 2018. The increase in research and development expenses was primarily due to an increase in clinical activities for our ongoing clinical programs, an increase in R&D headcount, and includes a $7.5 million expense pertaining to the achievement of a clinical development milestone pursuant to our license agreement with WuXi Biologics. Research and development expenses for the six months ended June 30, 2019 were $40.6 million, compared to $25.4 million for the same period in 2018.

G&A Expenses: General and administrative expenses for the second quarter ended June 30, 2019 were $5.9 million, compared to $3.5 million for the same period in 2018. Higher general and administrative expenses were primarily due to an increase in G&A headcount and related costs, as well as costs related to operations as a public company. General and administrative expenses for the six months ended June 30, 2019 were $10.9 million, compared to $6.4 million for the same period in 2018.

Net Loss: Net loss for the second quarter ended June 30, 2019 was $28.1 million, compared to $13.5 million for the same period in 2018. The increase in net loss was primarily attributable to an increase in operating expenses noted above partially offset by an increase in revenues. Net loss for the six months ended June 30, 2019 was $45.8 million, compared to $26.5 million for the same period in 2018.