On August 10, 2020 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported financial results for the second quarter ended June 30, 2020 and provided an update on its recent corporate activities and outlook (Press release, Halozyme, AUG 10, 2020, View Source [SID1234563322]).

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"The second quarter marked the achievement of multiple key milestones for Halozyme including two FDA approvals and one EMA approval for partnered drugs bringing the total number of FDA-approved products utilizing our ENHANZE drug delivery technology to five," said Dr. Helen Torley, president and chief executive officer. "In addition, we are delighted to report that in the second quarter we delivered our first profitable quarter of expected sustainable profitability with earnings per share of $0.19. We see this as an important first step in our transformation to a high growth, high margin business delivering sustainable revenue growth and profitability over the long term. The events of the quarter were highlighted by our partner Janssen receiving approvals in both the U.S. and the EU for the subcutaneous form of DARZALEX utilizing ENHANZE, which is branded as DARZALEX FASPROTM in the U.S. We earned $25 million in total milestone payments from Janssen during the quarter, upon the first commercial sales in both markets. In late June, our partner Roche received FDA approval for Phesgo, a fixed-dose combination of two monoclonal antibodies, Perjeta and Herceptin, utilizing our ENHANZE technology for the treatment of patients with HER2-positive breast cancer. In addition to providing important new treatment options for patients, each of these newly-approved drugs represents the subcutaneous form of a growing, blockbuster franchise, and we expect their adoption to be an important driver of our growth and profitability in the coming years."

"I want to again express my gratitude to the Halozyme team, our partners and suppliers for their tireless work as we all navigate challenges posed by the COVID-19 pandemic," continued Dr. Torley. "Based on the latest information from our partners, I am pleased to report that we are maintaining our financial outlook for the full year 2020. It is possible that our partners’ timelines may change as a result of future changes related to COVID-19. We will continue to monitor this closely and provide updates as appropriate."

Second Quarter 2020 and Recent Highlights Include:

On June 29, the Company announced that Roche received FDA approval for Phesgo (pertuzumab, trastuzumab, and hyaluronidase-zzxf), a fixed-dose combination of Perjeta and Herceptin for subcutaneous injection utilizing ENHANZE technology for the treatment of patients with HER2-positive breast cancer. Phesgo can be administered in approximately eight minutes for the initial loading dose and approximately five minutes for each subsequent maintenance dose. This is compared to approximately 150 minutes for a sequential infusion of a loading dose of Perjeta and Herceptin using the standard intravenous (IV) formulations, and between 60-150 minutes for subsequent maintenance infusions of the two medicines. Phesgo can be administered by a healthcare professional in a treatment center or at a patient’s home.
On June 13, the Company announced that findings from Janssen’s phase 3 ANDROMEDA (AMY3001) study evaluating subcutaneous daratumumab utilizing ENHANZE in light-chain Amyloidosis were presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 25th Annual Congress. Janssen reported that the study met the primary endpoint of percentage of patients with hematologic complete response.
On June 4, the Company announced that Janssen received European marketing authorization for the subcutaneous formulation of DARZALEX (daratumumab) utilizing ENHANZE for the treatment of adult patients with multiple myeloma in all currently approved DARZALEX intravenous (IV) formulation indications in frontline and relapsed / refractory settings. Subsequent launch of the product and first commercial sale in Europe resulted in a $10 million milestone payment in the quarter.
In June 2020, Bristol Myers Squibb initiated a Phase 1/2 study of ipilimumab in combination with nivolumab in multiple tumor types utilizing ENHANZE technology.
On May 1, the Company announced that Janssen received U.S. FDA approval of DARZALEX FASPROTM (daratumumab hyaluronidase human- fihj) in four regimens across five indications in multiple myeloma patients, including newly diagnosed, transplant-ineligible patients as well as relapsed or refractory patients. As a fixed-dose formulation, DARZALEX FASPROTM can be administered subcutaneously over three to five minutes, significantly less time than IV DARZALEX, which requires multi-hour infusions. Subsequent launch of the product and first commercial sale resulted in a $15 million milestone in the quarter.
Second Quarter 2020 Financial Highlights

Revenue for the second quarter was $55.2 million compared to $39.1 million for the second quarter of 2019. The year-over-year increase was primarily driven by $32.3 million in collaboration payments from Janssen and Bristol Myers Squibb in the current period. Revenue for the quarter included $15.8 million in royalties, which compared to $18.1 million in the prior year period.
Research and development expenses for the second quarter were $9.0 million, compared to $33.9 million for the second quarter of 2019. The decrease in expenses was due to a decrease in clinical trial activities-related costs as a result of the Company halting its oncology drug development efforts in November 2019.
Selling, general and administrative expenses for the second quarter were $11.0 million, compared to $17.3 million for the second quarter of 2019. The decrease was due to lower compensation and commercial-related expenses related to the corporate restructuring announced in November 2019.
The Company reported the first quarter of what it expects will be sustainable profitability. Net income for the second quarter was $25.8 million, or $0.19 per share, compared to a net loss in the second quarter of 2019 of $14.6 million, or $0.10 per share.
Cash, cash equivalents and marketable securities were $385.4 million at June 30, 2020, compared to $421.3 million at December 31, 2019.
Financial Outlook for 2020

The Company continues to monitor the impact of the COVID-19 pandemic on its business and receives updates from its partners and suppliers on how their businesses are affected. Based on this information and Halozyme’s planned expenditures for the year, the Company’s 2020 financial guidance remains unchanged. For 2020 Halozyme continues to expect:

Revenues of $230 million to $245 million, representing growth of 17% to 25%;
Earnings per share on a GAAP basis of $0.60 to $0.75.
The Company remains committed to capital return and plans to repurchase an additional number of shares, up to an additional $96 million worth, during the remainder of 2020. The amount and timing of shares repurchased during 2020 will be subject to a variety of factors including market conditions, other business considerations and applicable legal requirements.

Webcast and Conference Call

Halozyme will webcast its Quarterly Update Conference Call for the second quarter of 2020 today, Monday, August 10, 2020 at 4:30 p.m. ET/1:30 p.m. PT. Dr. Torley will lead the call, which will be webcast live through the "Investors" section of Halozyme’s corporate website and a replay will be available following the close of the call. To register for this conference call, please use this link: View Source To access the webcast and additional documents related to the call, please visit halozyme.com approximately fifteen minutes prior to the call to register, download and install any necessary audio software. A telephone replay will be available for two weeks after the call by dialing (800) 585-8367 (domestic callers) or (416) 621-4642 (international callers) using replay ID number 6277618.

On August 10, 2020 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 corporate updates and financial results for the second quarter and six months ended June 30, 2020 (Press release, CymaBay Therapeutics, AUG 10, 2020, View Source [SID1234563321]).

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Specifically, in the second quarter and through early August 2020, CymaBay achieved significant progress in its ongoing efforts to review its strategic options, one of which included completing a scientific investigation and working with the FDA to lift the clinical holds on the seladelpar INDs in nonalcoholic steatohepatitis (NASH), primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC), the three liver diseases in which CymaBay had terminated clinical studies late last year.

Sujal Shah, President and CEO of CymaBay, stated, "We are thrilled with the significant progress made to date in our efforts to conduct a review of strategic options, one of which was to pursue the reinstatement of our seladelpar program. Specifically, in May, we convened a panel of expert liver pathologists and hepatologists that unanimously concluded after a thorough, independent investigation, that there was no clinical, biochemical, or histological evidence of seladelpar-induced liver injury for patients enrolled in our Phase 2b NASH study. We discussed the panel’s findings and related information with the FDA and submitted complete responses to the agency, and in July, the FDA notified us that all clinical holds on seladelpar were lifted. In addition to this favorable outcome, we evaluated and announced last week positive topline results from our ENHANCE study of seladelpar in PBC which, despite being terminated early, provided sufficient data which appear to support seladelpar’s efficacy and tolerability in this patient population. After receiving notification from the FDA, and reviewing the latest clinical data from the ENHANCE study, we stopped our review of strategic options having decided to focus on reinstating the clinical development program for seladelpar in PBC and to continue to evaluate seladelpar for other indications. We have also been very successfully at minimizing our operating expenses through the first half of the year and expect our cash to fund our current operating plan into 2022."

Recent Corporate Highlights

In May 2020, a panel of eight of the world’s foremost expert liver pathologists and hepatologists, whose collective experience relevant to CymaBay’s investigation includes drug-induced liver injury, NASH and cholestatic liver diseases, completed a four-day independent review analyzing findings from CymaBay’s NASH Phase 2b study, and the results of independent pathologist’s reviews of the study biopsies, which included a blinded unpaired review and a paired review blinded to chronologic order of the biopsies. The panel unanimously supported lifting the clinical hold for seladelpar and re-initiation of clinical development pending approval by the FDA. In June 2020, CymaBay discussed the data and the panel’s conclusions with the FDA and submitted complete responses to the agency. In July 2020, the FDA lifted clinical holds on seladelpar in all indications with open INDs (NASH, PBC and PSC).

In August 2020, CymaBay announced positive topline results from ENHANCE for seladelpar in patients with PBC. Topline data for patients through 3 and 6 months demonstrated anti-cholestatic, anti-inflammatory, and anti-pruritic activity. Notably, 78.2% of patients on seladelpar 10 mg versus 12.5% on placebo achieved the primary composite outcome after only 3 months (p<0.0001). In addition, 27.3% of patients on seladelpar 10 mg versus zero on placebo experienced normalization of ALP by 3 months (p<0.0001). Treatment with seladelpar 10 mg also resulted in a statistically significant improvement in pruritus (p<0.05) for patients with moderate-to-severe itch versus placebo. Overall, seladelpar appeared to be safe and well-tolerated in this study.

CymaBay intends to reinitiate the long-term study, a Phase 3 study and other NDA-enabling studies to confirm the potential of seladelpar to be a best-in-class treatment for patients with PBC and to further evaluate suitable strategies to advance seladelpar in other indications.

CymaBay held $168.9 million in cash, cash equivalents and short-term investments as of June 30, 2020 and had no outstanding debt. Cash and investments are deemed sufficient to fund CymaBay’s current operating plan into 2022.

Due to the ongoing effects of the global coronavirus pandemic, CymaBay continues to conduct its operations remotely for all employees, which has allowed business activities to continue as seamlessly as possible. To date, these developments have not had a significant impact on CymaBay’s financial condition or its ability to execute its business plan. CymaBay will continue to closely monitor pandemic developments and their associated risks to the business, including plans to restart clinical development of seladelpar, and will continue to take actions available to mitigate them where possible. Further, all CymaBay’s actions will be guided by a commitment to taking all steps possible to ensure the health and safety of its employees as well as patients enrolled in its clinical studies.
Second Quarter and Six Months Ended June 30, 2020 Financial Results

Research and development expenses for the three months ended June 30, 2020 were $7.9 million, compared to $21.1 million for the three months ended June 30, 2019. Research and development expenses for the six months ended June 30, 2020 were $17.5 million, compared to $39.7 million for the six months ended June 30, 2019. Research and development expense in the three and six months of 2020 was significantly lower than the corresponding periods in 2019 primarily due to declining clinical trial activities related to the Phase 3 PBC, Phase 2b NASH, and Phase 2 PSC clinical trials, and other studies, as efforts continued to shut down these studies which were early-terminated as a result of the FDA’s clinical holds that were placed on the seladelpar program in the fourth quarter of 2019.

General and administrative expenses for the three months ended June 30, 2020 were $3.4 million, compared to $4.5 million for the three months ended June 30, 2019. General and administrative expenses for the six months ended June 30, 2020 were $7.7 million, compared to $10.2 million for the six months ended June 30, 2019. General and administrative expenses in the three and six months of 2020 were lower than the corresponding periods in 2019 due to lower employee compensation and other administrative expenses incurred as a result of a December 2019 reduction-in-force and restructuring effort that was undertaken to reduce costs in response to the FDA’s clinical holds on the seladelpar program.

Net loss for the three months ended June 30, 2020 was $10.7 million, or ($0.16) per diluted share, compared to a net loss of $24.0 million, or ($0.35) per diluted share in the three months ended June 30, 2019. Net loss for the six months ended June 30, 2020 was $23.8 million, or ($0.35) per diluted share, compared to a net loss of $47.1 million, or ($0.72) per diluted share in the six months ended June 30, 2019. Net loss was lower in the three and six months of 2020 compared to the corresponding periods in 2019 primarily due to a decrease in operating expenses, including clinical trial and labor related expenses, as a result of the early-termination of our seladelpar studies and our cost reduction efforts undertaken in response to the FDA’s clinical holds that were placed on the seladelpar program in the fourth quarter of 2019.
Conference Call Details

CymaBay will host a conference call today at 4:30 p.m. ET to discuss second quarter 2020 financial results and provide a corporate 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# 13706143. 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 10, 2020 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported financial results for the three-month period ended June 30, 2020 (Press release, Spectrum Pharmaceuticals, AUG 10, 2020, View Source [SID1234563320]).

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"The recently announced positive results from Cohort 2 are a meaningful development for patients with NSCLC HER2 exon 20 insertion mutations for which there is no approved therapy," said Joe Turgeon, President and CEO, Spectrum Pharmaceuticals. "We are in the process of requesting a pre-NDA meeting with the FDA and look forward to reviewing this data with the agency. In addition, the BLA for ROLONTIS is under active FDA review with a PDUFA date of October 24, 2020. We are in a strong capital position to fund our ongoing development and commercialization of our late stage assets."

Pipeline Updates

Poziotinib, an irreversible tyrosine kinase inhibitor targeting EGFR and HER2 mutations

Poziotinib met the pre-specified primary endpoint for Cohort 2 in the ZENITH20 Phase 2 clinical trial evaluating previously treated non-small cell lung cancer (NSCLC) patients with HER2 exon 20 insertion mutations. Cohort 2 of the ZENITH20 clinical trial enrolled a total of 90 patients who received an oral, once daily dose of 16 mg/day of poziotinib. All responses were read independently and confirmed by a central imaging laboratory using RECIST criteria. The intent-to-treat analysis demonstrated a confirmed objective response rate (ORR) of 27.8% (95% Confidence Interval (CI) 18.9%-38.2%). Based on the pre-specified statistical hypothesis for the primary endpoint, the observed lower bound of 18.9% exceeded the pre-specified lower bound of 17% in this heavily pre-treated population.
The median duration of response was 5.1 (range 1 to >12.3) months, with a median follow up of 8.3 months. The disease control rate (DCR) was 70% and the median progression free survival was 5.5 months.
Spectrum plans to present additional results from Cohort 2 at a medical meeting later in the year.
The company is in the process of requesting a pre-NDA meeting with the FDA based on the positive results from Cohort 2 to seek an indication for the treatment of patients with previously treated locally advanced or metastatic NSCLC with HER2 exon 20 insertion mutations.
Cohort 3 of the ZENITH20 trial in first-line EGFR NSCLC patients is fully enrolled and topline results are expected by year-end 2020.
ROLONTIS (eflapegrastim), a novel long-acting G-CSF

FDA is actively reviewing the BLA for ROLONTIS for the treatment of chemotherapy-induced neutropenia. The PDUFA target action date for the ROLONTIS BLA is October 24, 2020.
Three-Month Period Ended June 30, 2020 (All numbers are from Continuing Operations and are approximate)

GAAP Results

Spectrum recorded a net loss of $32.2 million, or $0.29 loss per basic and diluted share, in the three-month period ended June 30, 2020, compared to a net loss of $28.8 million, or $0.26 loss per basic and diluted share, in the comparable period in 2019. Total research and development expenses were $21.7 million in the quarter, as compared to $17.0 million in the same period in 2019. Selling, general and administrative expenses were $14.7 million in the quarter, compared to $17.2 million in the same period in 2019.

The company ended the quarter with cash, cash equivalents, and marketable securities of $156.5 million. The quarter-end cash balance does not include aggregate net proceeds of $82.1 million, after deducting underwriting discounts and commissions, from our recent underwritten public offering and sales under our at-the-market sales agreement.

Non-GAAP Results

Spectrum recorded a non-GAAP net loss of $31.8 million, or $0.28 per basic and diluted share, in the three-month period ended June 30, 2020, compared to a non-GAAP net loss of $25.2 million, or $0.23 per basic and diluted share, in the comparable period in 2019. Non-GAAP research and development expenses were $20.6 million, as compared to $13.2 million in the same period of 2019. Non-GAAP selling, general and administrative expenses were $11.8 million, as compared to $13.7 million in the same period in 2019.

Conference Call and Webcast

Spectrum’s management will host a webcast and conference call today, August 10, 2020, at 4:30 p.m. ET / 1:30 p.m. PT to discuss the financial results and provide a corporate update. The live call may be accessed by dialing (877) 837-3910 for domestic callers and (973) 796-5077 for international callers and entering the conference ID#: 4093736. A live webcast of the call will be available from the Investor Relations section of the company’s website at View Source and will be archived there shortly after the live event.

On August 10, 2020 Verastem, Inc. (Nasdaq:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to advancing new medicines for patients battling cancer, reported that it has entered into a definitive agreement to sell its global commercial and development rights to COPIKTRA (duvelisib), its marketed oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first FDA-approved dual inhibitor of PI3K-delta and PI3K-gamma, to Secura Bio, Inc., an integrated biopharmaceutical company dedicated to the worldwide commercialization of significant oncology therapies (Press release, Verastem, AUG 10, 2020, View Source [SID1234563319]).

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Verastem’s sale of COPIKTRA follows the Company’s previously announced strategic direction to focus on maximizing the broad potential of its RAF/MEK inhibitor (VS-6766) and FAK inhibitor (defactinib) program in KRAS mutant (KRASmt) solid tumors. Upon closing of the transaction with Secura Bio, Verastem will be dedicated to the development of this program and to deliver on clinical and regulatory milestones for the first potential indications in low-grade serous ovarian cancer (LGSOC) and KRASmt non-small cell lung cancer (NSCLC). Both LGSOC and KRASmt NSCLC are areas of high unmet patient need as there are no approved treatments and existing therapies have low response rates.

"By focusing our expertise and efforts on rapidly advancing the RAF/MEK/FAK development program, we believe we will be providing the best path forward for patients, customers, our shareholders and our company. These strategic decisions will enable us to best deliver on our mission to advance new medicines on behalf of cancer patients," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "The agreement with Secura Bio will ensure COPIKTRA continues to help more patients, leveraging the established commercial structure, support of ongoing clinical study and potential expansion into new indications."

Terms of the Definitive Sale Agreement

Verastem will receive an up-front payment of $70 million upon the closing of the transaction and is eligible to receive up to a total deal value of $311 million if certain regulatory and sales-based milestones are successfully met by Secura Bio and COPIKTRA’s other rest-of-world partners, including:

· A total of $45 million from two separate milestone payments for U.S. Food and Drug Administration (FDA) and European Medicines Agency approvals of COPIKTRA with label indicated for peripheral T-cell lymphoma
· A total of $50 million for cumulative worldwide net sales of COPIKTRA beginning at $100 million of cumulative net sales
· Verastem will receive low double-digit royalties on net sales over $100 million in U.S., Europe and the United Kingdom
· Verastem will also receive 50% of licensing milestones (up to $146 million) and royalties outside of U.S., Europe and the United Kingdom

In exchange, Secura Bio will receive an exclusive worldwide license for the research, development, commercialization and manufacture of COPIKTRA in all oncology indications. Secura Bio will assume all operational and financial responsibility for activities that were previously part of Verastem’s duvelisib program, including commercialization efforts in the United States and Europe, ongoing clinical trials, Verastem’s partnerships with Yakult, CSPC and Sanofi and existing royalty obligations. Secura Bio and Verastem are also in discussions related to the transfer of Verastem’s field sales and medical professionals.

The transaction with Secura Bio is subject to customary closing conditions and is expected to close in the third quarter of 2020.*

VS-6766 and Defactinib Program Progress and Registration-Directed Trials

Verastem announced today that the company met with the FDA in July 2020 to discuss the registration-directed study design for the VS-6766/defactinib combination in patients with LGSOC. The FDA was supportive of the Company’s development strategy and adaptive design for LGSOC.

Verastem’s NSCLC study will also be an adaptive design with a focus on patients with KRAS-G12V mutant tumors. Verastem intends to seek input from the FDA after completing the initial cohort of the lung cancer study. Verastem expects to commence registration-directed clinical trials for potential accelerated approval in LGSOC and KRASmt NSCLC by the end of 2020.

Verastem is continuing its clinical collaboration with the Drug Development Unit at ICR/Royal Marsden Hospital. The ongoing investigator-initiated Phase 1/2 FRAME study evaluating the combination of VS-6766 with defactinib in LGSOC, KRASmt NSCLC and colorectal cancer (CRC) has resumed normal accrual and reporting rates following the global lockdown resulting from the COVID-19 pandemic. The FRAME study is now expanding to include new cohorts in pancreatic cancer, KRASmt endometrial cancer and KRAS-G12V NSCLC. Verastem expects that additional data from the LGSOC cohort of the FRAME study will be made available in September, including presentation at the 2nd Annual RAS-Targeted Drug Development Conference. The Company also expects that additional data from the NSCLC cohort of the FRAME study will be submitted to the International Association for the Study of Lung Cancer (IASLC) World Lung Cancer Conference, taking place in January 2021.

The Company has also begun preclinical combination studies investigating VS-6766 and defactinib in combination with KRAS-G12C inhibitors and initial data will be presented at the 2nd Annual RAS-Targeted Drug Development Conference. Based on the positive preclinical data presented at the AACR (Free AACR Whitepaper) 2020 Virtual Annual Meeting II, Verastem plans to support a Phase 2 investigator-initiated study evaluating the combination of VS-6766 and defactinib in uveal melanoma, which is expected to begin in late 2020.

Corporate and Financial Overview

With the sale of COPIKTRA, Verastem will become a focused development company with reduced annual expenses of approximately $50 million. The company is in a position of financial strength with a cash runway expected to fund the clinical and regulatory milestones and development of VS-6766 and defactinib in LGSOC and KRASmt NSCLC until at least 2024.

About VS-6766
VS-6766 (formerly known as CH5126766, CKI27 and RO5126766) is a unique inhibitor of the RAF/MEK signaling pathway. In contrast to other MEK inhibitors in development, VS-6766 blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows VS-6766 to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors.

About Defactinib

Defactinib (VS-6063) is an oral small molecule inhibitor of FAK and PYK2 that is currently being evaluated as a potential combination therapy for various solid tumors. The Company has received Orphan Drug designation for defactinib in ovarian cancer and mesothelioma in the US, EU and Australia. Preclinical research by Verastem Oncology scientists and collaborators at world-renowned research institutions has described the effect of FAK inhibition to enhance immune response by decreasing immuno-suppressive cells, increasing cytotoxic T cells, and reducing stromal density, which allows tumor-killing immune cells to enter the tumor.1,2

About the VS-6766/Defactinib Combination

RAS mutant tumors are present in 30% of all human cancers and have historically presented a difficult treatment challenge and are often associated with significantly worse prognosis. Challenges associated with identifying new treatment options for these types of cancers include resistance to single agents, identifying tolerable combination regimens with MEK inhibitors and new RAS inhibitors in development addressing only a minority of all RAS mutated cancers.

The combination of VS-6766 and defactinib has been found to be clinically active in KRASmt. In an ongoing investigator-initiated Phase I/2 FRAME study, the combination of VS-6766 and defactinib is being evaluated in patients with LGSOC, KRASmt NSCLC and colorectal cancer (CRC). Preliminary data from this study presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2020 Virtual Annual Meeting I demonstrated a 67% overall response rate and long duration of therapy among patients with KRASmt LGSOC. Based on an observation of higher response rates seen in patients with KRAS-G12V mutations in the study, Verastem will also be further exploring the role of VS-6766 and defactinib in KRAS-G12V NSCLC. The FRAME study is expanding in August 2020 to include new cohorts in pancreatic, KRASmt endometrial and KRAS-G12V NSCLC.

About COPIKTRA (duvelisib)

COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells. PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.3,4,5 COPIKTRA is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies and relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. COPIKTRA is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), for which it has received Fast Track status and Orphan Drug Designation, and is being investigated in combination with other agents through investigator-sponsored studies.6 For more information on COPIKTRA, please visit www.COPIKTRA.com. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov
*MTS Health Partners, L.P and Ropes & Gray acted as advisors to Verastem Oncology on this transaction.

On August 10, 2020 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, reported its second quarter 2020 financial results, and provided recent corporate and program highlights (Press release, Aeglea BioTherapeutics, AUG 10, 2020, View Source [SID1234563318]).

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"Despite the operating challenges posed by the global pandemic, we continued to advance our pegzilarginase program in the first half of the year. The presentation of long-term data showing sustained lowering of arginine levels and durable clinical response with pegzilarginase treatment, as well as progress in our patient identification efforts, reinforce our belief in its potential as a life-changing therapy for those with Arginase 1 Deficiency and lay a strong foundation for the commercial launch of pegzilarginase," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "Additionally, we initiated our Phase 1/2 clinical trial of ACN00177 for Homocystinuria in the second quarter. We are continuing our patient identification activities and look forward to dosing the first patient once the clinical sites are able to begin screening patients."

Recent Highlights and Updates

Pegzilarginase in Arginase 1 Deficiency

In May, Aeglea presented results from its 56 week analysis from the Company’s completed Phase 1/2 clinical trial and ongoing open-label extension study during a late-breaking oral presentation at the 6th Congress of the European Academy of Neurology. Key results include:

Treatment with pegzilarginase resulted in a significant reduction in plasma arginine from baseline with all 13 patients achieving plasma arginine levels within the target range (<200 µM).

85% (11 of 13) of patients were clinical responders based on mobility improvements evaluated using three assessments: 6MWT (6 Minute Walk Test), GMFM (Gross Motor Function) Part D (standing) and Part E (walking, running, and jumping).

Pegzilarginase was shown to have a favorable safety profile with more than 750 doses administered.

To date, Aeglea has identified more than 240 Arginase 1 Deficiency patients. The number of identified patients represents more than 50% and 30% of the estimated genetic prevalence patient population in the U.S. and key European markets (France, Germany, Spain, Italy and the United Kingdom), respectively.

ACN00177 in Homocystinuria

Aeglea initiated its Phase 1/2 clinical trial for ACN00177, a novel engineered human enzyme therapy designed to treat Homocystinuria, a serious metabolic disorder characterized by elevated plasma homocysteine which leads to a wide range of life-altering complications and reduced life expectancy.

Corporate

Eric Bradford, M.D., M.Sc., M.B.A. has been promoted to Chief Development Officer. Dr. Bradford will oversee the clinical programs for pegzilarginase and ACN00177 as well as shape the clinical development strategy for future programs from the Company’s platform of novel human enzymes.

Chief Medical Officer Ravi M. Rao, M.B BChir, Ph.D., will depart the company to pursue other opportunities. Dr. Rao will continue to support Aeglea in a medical advisor role through a transitional period.

"Ravi has been a valued and impactful member of the Aeglea team. While we are disappointed by his planned departure, we wish him the best as he returns to his roots in immunology research and development," said Dr. Quinn. "I look forward to working more closely with Eric as we continue to strengthen our capabilities and advance pegzilarginase towards potential approval and launch."

Upcoming Events

Aeglea will be attending the following virtual investor conferences in the coming quarter.

Wells Fargo Securities Healthcare Conference, September 9-10

H.C. Wainwright Healthcare Conference, September 13-15

Cantor Fitzgerald Global Healthcare Conference, September 15-17

Further, Aeglea’s leadership looks forward to participating in dialogue about the Company’s enzyme therapeutics platform during the following industry events, with additional details to be announced.

World Orphan Drug Congress USA 2020, August 24-26

Child Neurology Society Annual Meeting-International Child Neurology Congress 2020, October 19-23

Second Quarter 2020 Financial Results

As of June 30, 2020, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $159.2 million. Based on Aeglea’s current operating plans, management believes it has sufficient capital resources to fund anticipated operations through 2022.

Research and development expenses totaled $16.9 million for the second quarter of 2020 and $14.8 million for the second quarter of 2019. The increase was primarily associated with investing in manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase; ramp-up in manufacturing activities for ACN00177 in Homocystinuria; and personnel-related expenses offset by decreasing clinical development expenses as a result of completing a Phase 1/2 clinical trial in patients with Arginase 1 Deficiency and closing out cancer trials.

General and administrative expenses totaled $4.7 million for the second quarter of 2020 and $3.8 million for the second quarter of 2019. This increase was primarily due to additional employee headcount, ramping up commercial capabilities, and additional facilities to support company growth.

Net loss totaled $21.4 million and $18.0 million for the second quarter of 2020 and 2019, respectively, with non-cash stock compensation expense of $1.6 million and $1.2 million for the second quarter of 2020 and 2019, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically lowers levels of the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating, progressive disease presenting in childhood with persistent hyperargininemia, spasticity, developmental delay, intellectual disability, seizures and early mortality. Pegzilarginase is intended for use as an enzyme therapy to reduce elevated blood arginine levels in patients with ARG1-D. Aeglea’s Phase 1/2 and Phase 2 open-label extension data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. The Company’s single, global pivotal Phase 3 PEACE trial is designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.

About ACN00177 in Homocystinuria

Aeglea is developing ACN00177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities including severe osteoporosis, developmental delay, intellectual disability, lens dislocation and severe near-sightedness. ACN00177 has been designed as a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. With this mechanism, ACN00177 is intended to lower the abnormally high blood levels of homocysteine in patients with Homocystinuria. Preclinical data demonstrated that ACN00177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. The Company initiated a Phase 1/2 trial in the second quarter of 2020 and continues patient identification and administrative activities. The timing of first patient dosing in this Phase 1/2 trial will depend on determinations by individual sites as they adjust to impacts from COVID-19.