On October 31, 2017 AstraZeneca and its haematology research and development centre of excellence, Acerta Pharma, reported that the US Food and Drug Administration (FDA) has granted accelerated approval to Calquence (acalabrutinib) (Press release, AstraZeneca, OCT 31, 2017, View Source [SID1234521359]). Calquence is a kinase inhibitor indicated for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.1

Calquence is approved under the FDA’s accelerated approval pathway, based on overall response rate, which allows for earlier approval of medicines that treat serious conditions and that fill an unmet medical need based on a surrogate endpoint.2 Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.1

Pascal Soriot, Chief Executive Officer of AstraZeneca, said: “The accelerated approval of Calquence is a landmark moment for our company. It provides an exciting new treatment option for patients with mantle cell lymphoma and marks the first approval of a medicine that will be the cornerstone of our presence in haematology. Furthermore, today’s approval demonstrates our commitment to scientific leadership in Oncology and reinforces our progress towards returning to growth.”

Michael L. Wang, MD, Professor, Department of Lymphoma/Myeloma, The University of Texas MD Anderson Cancer Center, and Principal Investigator of the ACE-LY-004 MCL clinical trial, said: “The acalabrutinib approval represents an important development for patients currently battling mantle cell lymphoma, an aggressive type of blood cancer that is typically diagnosed at an advanced stage and associated with a high relapse rate. In addition to the overall response rate, the high complete response rate of 40% seen in this trial illustrates the potential of acalabrutinib to help patients achieve a deep response.”

Summary of key efficacy results as assessed by Independent Review Committee (IRC) from the ACE-LY-004 trial,1 a Phase II open-label, single-arm clinical trial in 124 adult patients with relapsed or refractory MCL:

Efficacy Measure
Result
Overall Response Rate
80%
(95% CI: 72, 87)
Complete Response
40%
(95% CI: 31, 49)
Partial Response
40%
(95% CI: 32, 50)
Per Lugano classification, CI = Confidence interval

In the ACE-LY-004 trial, the most common adverse reactions (≥20%) of any grade were anaemia (46%), thrombocytopoenia (44%), headache (39%), neutropoenia (36%), diarrhoea (31%), fatigue (28%), myalgia (21%) and bruising (21%). Haematological events were based on laboratory measurements and adverse reactions.1

Dosage reductions or discontinuation due to any adverse reaction were reported in 1.6% and 6.5% of patients, respectively.1 Increases in creatinine 1.5 to 3 times the upper limit of normal occurred in 4.8% of patients.1

These data demonstrate the potential impact that Calquence could have on the management of previously-treated MCL. Calquence is not approved for use outside this labelled indication in the US.

Meghan Gutierrez, Chief Executive Officer, Lymphoma Research Foundation, said: “Relapse is common in mantle cell lymphoma patients and represents disease progression.3 When patients learn there is a new treatment option available for their disease, it brings great hope and an opportunity to participate in shared decision making with their healthcare team.”

Full results from the ACE-LY-004 clinical trial have been submitted for presentation at a forthcoming medical meeting. This will be the first MCL trial data to be presented from the Calquence development programme, which includes both monotherapy and combination therapies in a broad range of blood cancers and solid tumours. Calquence is also being evaluated in combination with bendamustine and rituximab as a potential 1st-line treatment for patients with MCL in the Phase III ACE-LY-308 clinical trial.4

NOTES TO EDITORS

About Calquence

Calquence (acalabrutinib; previously known as ACP-196) is a selective inhibitor of BTK. Calquence binds covalently to BTK, thereby inhibiting its activity, and has demonstrated this with minimal interactions with other immune cells in pre-clinical studies.1,5,6 In B cells, BTK signalling results in activation of pathways necessary for B cell proliferation, trafficking, chemotaxis and adhesion.1

The recommended dose of Calquence is one 100mg capsule taken orally approximately every twelve hours until disease progression or unacceptable toxicity.1 Calquence may be taken with or without food.1

Calquence is also in development for the treatment of multiple B-cell malignancies and other cancers including chronic lymphocytic leukaemia (CLL), MCL, Waldenström macroglobulinaemia (WM), follicular lymphoma, diffuse large B-cell lymphoma, and multiple myeloma. It is also being studied as a monotherapy and in combination trials for solid tumours. More than 35 clinical trials across 40 countries with more than 2,500 patients are underway or have been completed.7

Calquence was granted Orphan Drug Designation by the US FDA for the treatment of adult patients with MCL in September 2015 and by the European Commission in March 2016 for the treatment of adult patients with CLL, MCL and WM. Calquence was granted Breakthrough Therapy Designation by the FDA in August 2017 for the treatment of adult patients with MCL who have received at least one prior therapy.

About Mantle Cell Lymphoma (MCL)

MCL is an aggressive B-cell non-Hodgkin lymphoma (NHL) with poor prognosis.8,9,10,11 MCL accounts for approximately 3% to 6% of new NHL cases in Western countries each year; in the US, approximately 3,300 new cases of MCL are diagnosed each year.9,13 The median age at diagnosis is 68 years, with a 3:1 male predominance.10 While MCL patients initially respond to treatment, there is a high relapse rate.9

About the ACE-LY-004 trial

ACE-LY-004 is a Phase II open-label, single-arm clinical trial in 124 adult patients with relapsed or refractory MCL. The trial showed that 80% (95% CI: 72, 87) of patients treated with Calquence achieved an overall response; 40% (95% CI: 31, 49) achieved a complete response and 40% (95% CI: 32, 50) achieved a partial response per 2014 Lugano classification as assessed by Independent Review Committee.1

About Acerta Pharma

Acerta Pharma, a member of the AstraZeneca Group, is creating novel therapies intended for the treatment of cancer and autoimmune diseases. AstraZeneca acquired a majority stake interest in Acerta Pharma, which serves as AstraZeneca’s haematology research and development centre of excellence. For more information, please visit www.acerta-pharma.com.

About AstraZeneca in Oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that have the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020 and a broad pipeline of small molecules and biologics in development, we are committed to advance New Oncology as one of AstraZeneca’s five Growth Platforms focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy as illustrated by our investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

On October 31, 2017 Kura Oncology, Inc. (NASDAQ:KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported that it will report third quarter 2017 financial results after the close of U.S. financial markets on November 7, 2017 (Press release, Kura Oncology, OCT 31, 2017, View Source [SID1234521367]). Kura Oncology’s management will host a webcast and conference call at 4:30 p.m. EST/1:30 p.m. PST that day to discuss the financial results and provide a corporate update.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The live call may be accessed by dialing 877-516-3514 for domestic callers and 281-973-6129 for international callers and entering the conference code: 2685708. A live webcast and archive of the call will be available online from the investor relations section of the company website at www.kuraoncology.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference code: 2685708.

On October 31, 2017 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that Alan H. Auerbach, Chairman, Chief Executive Officer, President and Founder of Puma, will provide an overview of the Company, including recent developments, at 11:30 a.m. MST (10:30 a.m. PST, 1:30 p.m. EST) on Tuesday, November 7, at the Credit Suisse 26th Annual Global Healthcare Conference (Press release, Puma Biotechnology, OCT 31, 2017, View Source [SID1234521371]). The conference will be held at The Phoenician Resort in Scottsdale, Arizona.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast of the presentation will be available on the Company’s website at www.pumabiotechnology.com . The presentation will be archived on the website and available for 30 days.

Aeolus has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q/A [Amend], Aeolus, 2018, MAY 16, 2016, View Source [SID1234528062]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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On October 31, 2017 Iovance Biotherapeutics, Inc. (NASDAQ:IOVA), a biotechnology company developing novel cancer immunotherapies based on tumor-infiltrating lymphocyte (TIL) technology, reported its third quarter 2017 financial results and provided a corporate update (Press release, Iovance Biotherapeutics, OCT 31, 2017, View Source [SID1234521366]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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“In the third quarter of 2017 we continued to make significant progress in the clinic as the first patient was dosed with LN-145 in the Phase 2 trial for cervical cancer. Regulatory progress was demonstrated with the FDA granting Fast Track designation for LN-144 for the treatment of advanced melanoma and approval of a CTA by the competent authority in the Netherlands for the Phase 2 trial of LN-145 in cervical carcinoma,” said Dr. Maria Fardis, Ph.D., MBA, President and Chief Executive Officer of Iovance Biotherapeutics. “On the corporate front, we successfully completed a common stock offering adding approximately $54.0 million in net proceeds to the cash reserves. In the fourth quarter of 2017, we look forward to sharing new clinical data from Cohort 2 of the C-144-01 metastatic melanoma trial and nonclinical data at the upcoming SITC (Free SITC Whitepaper) meeting.”

Third Quarter 2017 and Recent Highlights and Anticipated Milestones

Corporate News:

Appointed New Chief Financial Officer (CFO): In August, Tim Morris was appointed CFO of Iovance. Mr. Morris brings over 22 years of experience related to the biopharmaceutical industry.

Completion of Public Offering: In September, the Company completed a public offering of 8,846,154 shares of its common stock at a price of $6.50 per share, before underwriting discounts. The shares of common stock issued and sold in the offering at the closing include 1,153,846 shares issued upon the exercise in full by the underwriters of their option to purchase additional shares. The net proceeds from the offering, after deducting the underwriting discounts and commissions and other estimated offering expenses payable by Iovance, are approximately $54.0 million.
Clinical Trial Progress:

C-144-01 Phase 2 Trial in Metastatic Melanoma: In October, preliminary data from Cohort 2 of the ongoing C-144-01 Phase 2 trial of LN-144 was accepted as a late-breaking abstract to be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2017 Annual Meeting.

First Patient Dosed in C-145-04 Phase 2 Trial in Cervical Cancer: In August, the first patient was dosed in the C-145-04 Phase 2 trial of LN-145 for the treatment of patients with recurrent, metastatic or persistent cervical carcinoma.
Manufacturing Updates:

Partnership with TrakCel for Personalized Patient Product Management: In September, Iovance commenced a partnership with TrakCel Ltd. to build a scheduling and logistics tool that automates the supply chain for Iovance’s adoptive cell therapy products that utilize its TIL technology. The TrakCel Solution will electronically link Iovance with clinical sites, contract manufacturing organizations and couriers to schedule and track TIL therapies for each patient. The TrakCel Solution is intended to help manage capacity utilization and throughput as well as providing efficiencies in the delivery of TIL treatment.
Regulatory Updates:

Fast Track Designation Granted for LN-144: In August, the U.S. Food and Drug Administration (FDA) granted Fast Track designation for LN-144, the Company’s adoptive cell therapy using its TIL technology, for the treatment of advanced melanoma.
European Clinical Trial Applications (CTAs): Iovance initiated the submission of CTAs in multiple countries in Europe starting in August 2017 in support of Phase 2 clinical trials of LN-145 in cervical carcinoma and LN-144 in metastatic melanoma. In September, the Company received the first approval from the competent authority in the Netherlands, for LN-145 for the treatment of patients with cervical carcinoma. Subsequent to the end of the quarter, the Company received CTA approvals in Hungary for metastatic melanoma and the United Kingdom for cervical carcinoma and metastatic melanoma.
Research Update and Data Presentations:

Research Collaboration Agreement with Ohio State University: In September, the Company entered into a collaboration with the Ohio State University. The collaboration will initially focus on hematologic malignancies in areas of poor prognostic cancers with high unmet medical need, which include acute myeloid leukemia (AML) and chronic lymphocytic leukemia (CLL).
Poster Presentation at European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper): In August, the Company announced a poster presentation at the ESMO (Free ESMO Whitepaper) 2017 Congress in September with data that demonstrates the ability to produce TIL from lymphoma that have similar functionality as TIL generated from melanoma.
Third Quarter 2017 Financial and Operating Results

As of September 30, 2017, the Company held $163.4 million in cash and cash equivalents and short-term investments, compared to $166.5 million as of December 31, 2016.

The Company is providing both GAAP and non-GAAP financial information. All non-GAAP information excludes amounts related to stock-based compensation. See “Use of Non-GAAP Financial Measures” below for a description of the Company’s non-GAAP Financial Measures. Reconciliation between certain GAAP and non-GAAP measures is provided at the end of this press release.

GAAP and Non-GAAP Net Loss Attributable to Common Stockholders

GAAP net loss attributable to common stockholders for the quarter ended September 30, 2017 was $22.1 million, or ($0.35) per share, compared to GAAP net loss of $68.2 million or ($1.15) per share for the quarter ended September 30, 2016.

Non-GAAP net loss attributable to common stockholders for the quarter ended September 30, 2017 was $19.5 million, or ($0.31) per share, compared to non-GAAP net loss of $10.1 million, or ($0.17) per share for the quarter ended September 30, 2016. The non-GAAP net loss for the quarters ended September 30, 2017 and September 30, 2016 excludes $2.6 million and $8.6 million of non-cash stock-based compensation, and a non-cash deemed dividend of $49.5 million which was recorded in the quarter ended September 30, 2016. The deemed dividend will only impact the prior years’ quarter’s financial statements.

GAAP net loss attributable to common stockholders for the nine months ended September 30, 2017 was $66.2 million, or ($1.06) per share, compared to GAAP net loss attributable to common stockholders of $86.7 million or ($1.64) per share for the nine months ended September 30, 2016. Non-GAAP net loss for the nine months ended September 30, 2017 was $57.0 million, or ($0.91) per share, compared to non-GAAP net loss of $21.4 million or ($0.40) per share for the nine months ended September 30, 2016.

GAAP and Non-GAAP Expenses

GAAP research and development (R&D) expenses were $17.8 million for the quarter ended September 30, 2017, an increase of $9.3 million compared to the quarter ended September 30, 2016. The increase in R&D expense is due to increased spending on clinical activities and manufacturing. In addition, R&D-associated stock based expenses were $1.1 million for the three months ended September 30, 2017 and $4.3 million for the nine months ended September 30, 2017. Non-GAAP R&D expenses were $16.7 million for the quarter ended September 30, 2017, an increase of $8.9 million, compared to $7.8 million for the quarter ended September 30, 2016.

GAAP general and administrative (G&A) expenses were $4.6 million for the quarter ended September 30, 2017, a decrease of $5.9 million compared to the quarter ended September 30, 2016. Non-GAAP G&A expenses were $3.0 million for the quarter ended September 30, 2017, an increase of $0.5 million, compared to $2.5 million for the quarter ended September 30, 2016.

Use of Non-GAAP Financial Measures

This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses. These measures are not in accordance with, or an alternative to, generally accepted accounting principles, or GAAP, and may be different from non-GAAP financial measures used by other companies. The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in this press release are: (i) the non-cash stock-based compensation expense which may fluctuate from period to period based on factors including the timing and accounting of grants for stock options and changes in the Company’s stock price which impacts the fair value of options granted, and (ii) the one-time non-cash deemed dividend related to the conversion feature of the Series B Preferred Stock. The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding various financial and business trends relating to the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of Iovance’s ongoing operating performance. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating operational performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. To the extent this release contains historical or future non-GAAP financial measures, the Company has also provided corresponding GAAP financial measures for comparative purposes. Reconciliation between certain GAAP and non-GAAP measures is provided at the end of this press release.

2017 Year End Guidance for Cash, Cash Equivalents and Short-Term Investments

Iovance anticipates the cash, cash equivalents and short-term investments as of December 31, 2017, to be in excess of $141.0 million.

Webcast and Conference Call

Iovance will host a conference call today at 4:30 p.m. ET to discuss these third quarter 2017 results. The conference call dial-in numbers are: 1-844-646-4465 (domestic) or 1-615-247-0257 (international). The conference ID access number for the call is 1423064. The live webcast can be accessed under “News & Events” in the “Investors” section of the Company’s website at View Source or you may use the link: View Source

A replay of the call will be available one hour after the end of the call on October 31, 2017 until 8:00 p.m. ET on November 30, 2017. To access the replay, please dial 1-855-859-2056 (domestic) or 1-404-537-3406 (international). The conference ID number for the replay is 1423064. The archived webcast will be available for thirty days in the Investors section of Iovance Biotherapeutics’ website at View Source