Eagle Pharmaceuticals Announces Clinical Development Plan of Innovative Product Intended to Deliver Maximum Estrogen Receptor Inhibition in Patients with Estrogen Receptor (ER)-Positive Breast Cancer

On August 5, 2019 Eagle Pharmaceuticals, Inc. ("Eagle" or the "Company") (Nasdaq: EGRX) reported a clinical development plan to support the submission of a New Drug Application (NDA) for the Company’s innovative fulvestrant formulation (Press release, Eagle Pharmaceuticals, AUG 5, 2019, View Source [SID1234538131]). Fulvestrant, an estrogen receptor antagonist with no agonist properties, is approved by the U.S. Food and Drug Administration (FDA) for the treatment of advanced hormone-related breast cancers. The therapeutic effect of fulvestrant relies on its ability to inhibit estrogen receptors (ER) in cancer cells by binding to and downregulating, or blocking, the ER in breast cancer cells. Recent studies have shown that higher residual ER availability is associated with early disease progression.

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!

Eagle’s original formulation of fulvestrant was studied in a clinical trial conducted in 2018 in healthy post-menopausal women. The study was conducted in 600 subjects over 140 days; 300 subjects received the branded product FASLODEX and 300 subjects received Eagle’s formulation. A detailed review of the study data led to the hypothesis that the unique properties of Eagle’s formulation would potentially allow for greater inhibition of estrogen receptors. Based on this hypothesis, Eagle has recently completed additional work designed to further enhance its proprietary drug formulation.

In March and June 2019, Eagle met with the FDA and mutually agreed to a clinical program that could provide an efficient approval pathway for the Company’s fulvestrant formulation. The main goal of the clinical research program is to determine if the unique properties of Eagle’s fulvestrant formulation will result in greater inhibition of estrogen receptors, potentially leading to improved efficacy outcomes, including lower disease progression rates, compared to current treatment options.

Eagle intends to begin a pilot study shortly in healthy female volunteers to evaluate the pharmacokinetics and safety of its novel formulation. Once the pilot study results are reviewed, a clinical pivotal trial designed to evaluate fulvestrant exposure and estrogen receptor inhibition based upon the parameters determined with the FDA will be conducted in a target patient population. Depending on recruitment rates and other factors, Eagle believes the pivotal study could be completed within approximately 12 months of commencing enrollment.

"The opportunity to develop a new treatment option that has the potential to better address the epidemic of breast cancer affecting millions of women in the U.S. and worldwide is an important priority for Eagle. Following a thorough review of the data from our previous study, we believe that our fulvestrant product has a unique profile that may allow it to achieve a greater level of estrogen receptor inhibition, and we are encouraged by the guidance provided by FDA to develop a clinical path to further explore the potential of our novel formulation. If successful, the Eagle product could provide a meaningful improvement and a new option to treat this breast cancer patient population," stated Scott Tarriff, Chief Executive Officer of Eagle Pharmaceuticals.

Breast cancer is the most commonly diagnosed cancer in women, with more than 2.8 million breast cancer survivors in the U.S. today; approximately 290,000 women are diagnosed in the U.S. annually. Hormone receptor-positive (HR+) breast cancer is the most common clinical subtype, with the estrogen receptor (ER) being expressed in approximately 70% of those diagnosed.

About Fulvestrant

Fulvestrant is indicated as a monotherapy treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced breast cancer in postmenopausal women not previously treated with endocrine therapy, or HR-positive advanced breast cancer in postmenopausal women with disease progression following endocrine therapy, or as a combination therapy for the treatment of: (1) HR-positive, HER2-negative advanced or metastatic breast cancer in postmenopausal women in combination with ribociclib as initial endocrine based therapy or following disease progression on endocrine therapy, or (2) HR-positive, HER2-negative advanced or metastatic breast cancer in combination with palbociclib or abemaciclib in women with disease progression after endocrine therapy.

Bellicum Pharmaceuticals Reports Second Quarter 2019 Financial Results and Provides Operational Update

On August 5, 2019 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the second quarter 2019 and provided an operational update (Press release, Bellicum Pharmaceuticals, AUG 5, 2019, View Source [SID1234538130]).

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!

"Thus far in 2019, Bellicum has made significant progress in each of its development programs," said Rick Fair, President and Chief Executive Officer of Bellicum Pharmaceuticals. "We presented encouraging interim data at ASCO (Free ASCO Whitepaper) on our BPX-601 GoCAR-T product candidate, advanced towards a Phase 1 study for BPX-603, our dual-switch HER2-targeted GoCAR-T product candidate, and announced that rivo-cel achieved the primary endpoint in its European registrational study. Looking forward, we have strategically prioritized our GoCAR-T programs and plan to enroll these trials to evaluate how our technology may help extend the impact of CAR-T therapies to the treatment of solid tumors."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

BPX-601 GoCAR-T

Bellicum presented updated safety and activity data for BPX-601 from a Phase 1/2 study in patients with metastatic pancreatic cancer expressing prostate stem cell antigen (PSCA) at the 2019 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The data showed a favorable safety profile—with no dose-limiting toxicities—and provided further evidence that GoCAR-T technology boosts expansion and persistence of CAR-T cells in patients. Of 13 patients evaluable for efficacy treated with BPX-601 and a single dose of rimiducid, 8 patients (62%) achieved stable disease, including 3 with tumor shrinkage of 10% to 24%. As a next step in the study, Bellicum is currently enrolling an additional cohort to evaluate repeat rimiducid dosing to re-activate iMC over time, which is intended to deepen and extend the treatment effect. Initial results from this cohort are expected in late 2019 or early 2020.
Controllable Dual-Switch GoCAR-T Product Candidates

Bellicum believes that its next-generation dual-switch GoCAR-T technology may enhance efficacy relative to current generation CAR-T therapy through iMC activation while enabling clinicians to manage certain treatment-emergent toxicities with CaspaCIDe. The company expects IND clearance for BPX-603, a dual-switch GoCAR-T targeting HER2-expressing solid tumors, later this year. The company also expects to submit an IND application for BPX-802, a dual-switch GoCAR-T product candidate targeting an antigen expressed in hematological malignancies.
Rivo-cel

In July, the company announced that rivo-cel achieved the primary endpoint (Event Free Survival at 180 days) and all secondary endpoints in its BP-004 European registrational trial. Data from this trial is expected to form the basis of anticipated submissions of European Marketing Authorisation Applications (MAAs) for rivo-cel and rimiducid in support of potential regulatory approval. The company is actively seeking a partnership for the continued development and commercialization of rivo-cel.
Second Quarter 2019 Financial Results

Cash Position and Guidance: Bellicum reported cash, restricted cash and investments totaling $60.6 million as of June 30, 2019, compared to $98.0 million at December 31, 2018. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements through at least the end of 2019. During the second quarter, Bellicum utilized its at-the-market financing facility selling 1.2 million shares for net cash proceeds of $4.4 million.

R&D Expenses: Research and development (R&D) expenses were $19.9 million for the second quarter of 2019, compared to $18.4 million for the second quarter of 2018. The higher expenses in the second quarter of 2019 resulted primarily from higher expenditures related to the GoCAR-T platform including initiation of additional clinical sites and costs related to IND filing. R&D expenses for the six months ended June 30, 2019 were $36.7 million compared to $34.9 million for the comparable period in the prior year.

G&A Expenses: General and administrative (G&A) expenses were $7.5 million for the second quarter of 2019 compared to $5.4 million during the comparable period in 2018. The higher expenses in the second quarter 2019 relative to the comparable period in 2018 were primarily due to increased personnel related costs and commercialization preparation activities. G&A expenses for the six months ended June 30, 2019 were $15.1 million compared to $11.1 million for the first six months of 2018.

Net Loss: Bellicum reported a net loss of $26.9 million for the second quarter of 2019 compared to a net loss of $24.2 million for the second quarter of 2018. The results included non-cash, share-based compensation charges of $2.0 million and $3.6 million for the second quarter of 2019 and 2018, respectively. Net loss for the six months ended June 30, 2019 was $51.5 million compared to a loss of $47.0 million for the six months ended June 30, 2018.

Shares Outstanding: At July 31, 2019, Bellicum had 46,254,163 shares of common stock outstanding.

About BPX-601

BPX-601, the company’s first GoCAR-T product candidate, incorporates iMC, Bellicum’s inducible co-activation domain. iMC (inducible MyD88/CD40) is designed to provide a powerful boost to T cell proliferation and persistence and enable the CAR-T to override key immune inhibitory mechanisms, including PD-1 and TGF-beta. BPX-601 is being evaluated as a treatment for solid tumors expressing prostate stem cell antigen (PSCA), including pancreatic, gastric, and prostate cancers.

About Rivo-cel (BPX-501)

Rivo-celTM (rivogenlecleucel) is an allogeneic polyclonal T cell product designed to reduce the rate of relapse of leukemia following a stem cell transplant. The cell treatment contains a diverse repertoire of T cells, which may contribute to a robust graft vs. leukemia effect. Rivo-cel’s antiviral benefits may also reduce morbidity and mortality in patients susceptible to infection following a transplant. The product’s CaspaCIDe safety switch enables this approach by allowing physicians to reduce the number of alloreactive cells in the event of uncontrolled GvHD. Rivo-cel addresses a major unmet need in adult and pediatric leukemia, lymphoma and genetic blood disease patients following a haploidentical stem cell transplant.

Arrowhead Pharmaceuticals Reports Fiscal 2019 Third Quarter Results

On August 5, 2019 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2019 third quarter ended June 30, 2019 (Press release, Arrowhead Research Corporation, AUG 5, 2019, View Source [SID1234538129]). The company is hosting a conference call at 4:30 p.m. EDT to discuss results.

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!

Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and provide Conference ID 3249189.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 855-859-2056 or 404-537-3406 and provide Conference ID 3249189.

Selected Fiscal 2019 Third Quarter and Recent Events

Received U.S. Food and Drug Administration (FDA) clearance to begin an adaptive design Phase 2/3 trial, called SEQUOIA, with the potential to serve as a pivotal registrational study of ARO-AAT, Arrowhead’s second generation subcutaneously administered RNAi therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency

Secured first regulatory clearance in the United Kingdom for the ARO-AAT 2002 study, a pilot open-label, multi-dose, Phase 2 study to assess changes in a novel histological

activity scale in response to ARO-AAT over time in patients with alpha-1 antitrypsin deficiency associated liver disease

Expanded the AROHBV1001 Phase 1/2 study to include a new triple combination cohort that includes: JNJ-3989, formerly ARO-HBV; JNJ-6379, Janssen’s investigational orally administered capsid assembly modulator of the class that forms normal capsid structures; and, a nucleos(t)ide analog, or NUC

In connection with the start of dosing of this cohort, Arrowhead earned a $25 million milestone payment from Janssen

Received orphan drug designation from FDA for ARO-APOC3 for the treatment of familial chylomicronemia syndrome

Received FDA Fast Track designation for ARO-AAT

Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier

Received orphan drug designation from FDA for ARO-ANG3 for the treatment of homozygous familial hypercholesterolemia

Completed discovery and development work on ARO-HSD, a previously undisclosed liver-targeted candidate targeting HSD17B13, a hydroxysteroid dehydrogenase involved in the metabolism of hormones, fatty acids and bile acids, that is now in IND-enabling GLP-toxicology studies, and, pending success in the tox program, on schedule for a CTA filing at the end of 2019

Started IND-enabling GLP-toxicology studies for ARO-HIF2, designed to inhibit the production of HIF-2a for the treatment of clear cell renal cell carcinoma, to support, pending success in the tox program, a CTA filing at the end of 2019

Completed dosing in the single-ascending dose portions of the Phase 1 studies of Arrowhead’s two wholly-owned cardiometabolic candidates, ARO-APOC3 and ARO-ANG3, and progressed towards the multiple-dose portions of the Phase 1 studies in various patient populations

ArQule to Participate in the BTIG Biotechnology Conference on Monday, August 12, 2019

On August 5, 2019 ArQule, Inc. (Nasdaq:ARQL) reported that Dr. Brian Schwartz, Chief Medical Officer and Head of Research and Development, will participate in the BTIG Biotechnology Conference on Monday, August 12, 2019 at The St. Regis New York in New York City (Press release, ArQule, AUG 5, 2019, View Source [SID1234538128]).

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!


Arbutus Reports Second Quarter 2019 Financial Results and Provides Corporate Update

On August 5, 2019 Arbutus Biopharma Corporation (Nasdaq: ABUS), an industry-leading Hepatitis B Virus (HBV) therapeutic solutions company, reported its second quarter 2019 financial results and provides a corporate update (Press release, Arbutus Biopharma, AUG 5, 2019, View Source [SID1234538127]).

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!

"I am excited to join Arbutus at this important inflection point as we look to advance our two lead compounds, AB-506 and AB-729, through Phase1a/1b clinical trials," said William Collier, Arbutus’ President and Chief Executive Officer. "Provided these compounds progress as expected, we anticipate moving into a combination proof-of-concept Phase 2 clinical trial in subjects with chronic hepatitis B in the second half of 2020. We believe a combination regimen that includes several different mechanisms of action will be required to improve upon the existing standard-of-care in HBV."

Recent Clinical Accomplishments and Key Corporate Accomplishments

New President & Chief Executive Officer

William H. Collier was appointed President and Chief Executive Officer of Arbutus and a member of the Board of Directors effective June 24th. Mr. Collier’s appointment filled the vacancy created by the retirement of Mark J. Murray, Ph.D., as Arbutus’ President and Chief Executive Officer, which was effective June 23rd. Mr. Collier has over 30 years of experience as a senior executive in the pharmaceutical industry and previously served as President and General Manager, North America at ViiV Healthcare. At ViiV, he oversaw the industry-leading launches of several new treatments for HIV. Prior to joining ViiV in 2009, Mr. Collier held multiple senior leadership roles at GlaxoSmithKline. Earlier in his career he led the launches of new treatments for herpes and bacterial infections. Mr. Collier received his BSc in Mathematics and Management Sciences from the University of Manchester Institute of Science & Technology, UK, and served on The President’s Advisory Council on HIV/AIDS from 2014 to 2017.
AB-506

In July 2019, Arbutus announced preliminary results from a Phase 1a/1b clinical trial demonstrating that AB-506 is a potent oral capsid inhibitor. These preliminary Phase 1a/1b results support the Company’s confidence in its potential to significantly contribute to the inhibition of HBV replication in a curative combination regimen.

Arbutus expects that safety and efficacy data from this portion of the Phase 1a/1b trial, as well as results from a planned Phase 1 28-day clinical trial in healthy subjects, will be submitted to an appropriate scientific meeting later this year.

Arbutus is planning on dosing additional cohorts and final results of this Phase 1a/1b trial, which are expected in the first half of 2020, will inform next steps toward the combination proof-of-concept Phase 2 clinical trial in subjects with chronic hepatitis B.
AB-729

In July 2019, the Company initiated the healthy subject portion of a single and multiple dose Phase 1a/1b clinical trial for AB-729, a subcutaneously delivered RNAi agent which has been shown in preclinical models to span all HBV transcripts, reduce all viral antigens, including hepatitis B surface antigen (HBsAg) expression, and inhibit HBV replication. In this trial, which will investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AB-729 in healthy subjects and subjects with chronic hepatitis B infection, AB-729 will be dosed monthly.

Preliminary safety and efficacy data from both healthy subjects and several single dose cohorts of subjects with chronic hepatitis B infection are expected in the first quarter of 2020.
Early R&D Programs

Arbutus continues a focused discovery effort on follow-on compounds for its current HBV pipeline, including its HBV RNA destabilizer, AB-452, as well as efforts to identify compounds potentially capable of reawakening HBV patients’ immune response such as PD-L1 blockers and HBV-specific targets such as HBV cccDNA.
ONPATTRO Royalty Entitlement

Arbutus has a royalty entitlement on global net sales of ONPATTRO (Patisiran) for the lipid nanoparticle delivery (LNP) technology licensed by Arbutus to Alnylam Pharmaceuticals, Inc. (Alnylam) for this product. ONPATTRO is an RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis that has been approved by the FDA and the EMA. In July 2019, Arbutus sold this royalty entitlement to OMERS, the defined benefit pension plan for municipal employees based in the Province of Ontario, Canada, effective as of January 1, 2019, for $20 million in gross proceeds before advisory fees. OMERS will retain this royalty entitlement until it has received $30 million in royalties, at which point 100% of this royalty entitlement will revert to Arbutus.

In addition to the royalty entitlement from the Alnylam LNP license agreement, Arbutus is also entitled to a second, lower royalty entitlement on global net sales of ONPATTRO originating from a settlement agreement and subsequent license agreement with Acuitas Therapeutics. The royalty entitlement from Acuitas has been retained by Arbutus and is not part of the royalty entitlement sale to OMERS.
Financial Results

Cash, Cash Equivalents and Investments

Arbutus had cash, cash equivalents and short-term investments totaling $95.3 million as of June 30, 2019, as compared to $124.6 million as of December 31, 2018. The decreased cash balance was due primarily to $34.1 million of cash used in operating activities for the six months ended June 30, 2019, partially offset by $4.7 million of net proceeds from the issuance of shares under its ATM program. In July 2019, the Company received $20 million in gross proceeds from the sale of a portion of its royalty entitlement on net sales of ONPATTRO. The Company believes its cash and investments balance is sufficient to fund operations into the second half of 2020.

Operating Expenses

Research and development expenses were $12.8 million in Q2 2019 compared to $16.3 million in Q2 2018. Research and development expenses in 2019 included costs associated with the Company’s Phase 1a/1b clinical trial for its lead capsid inhibitor (AB-506), pre-clinical studies for its RNAi agent (AB-729), and characterization activities for its HBV RNA Destabilizer (AB-452). The decrease in research and development expenses was due primarily to higher costs in 2018 for AB-452, including drug product manufacturing, and expenses in 2018 associated with the Phase 2 clinical trial for AB-1467, partially offset by increased spending in 2019 for the Phase 1a/1b clinical trial for AB-506 and pre-clinical studies for AB-729. General and administrative expenses were $8.2 million in Q2 2019 compared to $3.8 million in Q2 2018. The increase in general and administrative expenses was due primarily to our former President and Chief Executive Officer’s departure from the Company in June 2019. In accordance with the terms of his legacy employment agreement, he received $2.3 million in cash severance and the Company recognized $2.2 million of non-cash stock-based compensation expense for accelerated vesting of his stock options.

Equity investment loss

As of June 30, 2019, the Company owned approximately 40% of the common equity of Genevant Sciences Ltd. (Genevant), a company launched with Roivant Sciences Ltd. in April 2018. Arbutus recorded a loss of $3.3 million in Q2 2019 for its proportionate share of Genevant’s net loss. In Q2 2018, Arbutus recognized a non-cash gain of $24.9 million in connection with the equity interest received by Arbutus upon Genevant’s formation. Financial results of Genevant are recorded on a one-quarter lag basis.

Net Income (Loss)

Net income (loss) attributable to common shares for Q2 2019 was a net loss of $26.1 million ($0.46 basic and diluted loss per common share) as compared to net income of $0.6 million ($0.01 basic and diluted income per common share) for Q2 2018. Net income (loss) attributable to common shares included $2.8 million of non-cash expense in Q2 2019 and $2.5 million in Q2 2018 for the accrual of coupon on the Company’s convertible preferred shares. Net income in Q2 2018 included a non-cash gain of $24.9 million in connection with the equity interest received by Arbutus upon Genevant’s formation.

Outstanding Shares

The Company had approximately 56.9 million common shares issued and outstanding as of June 30, 2019. In addition, the Company had approximately 8.9 million options outstanding and 1.164 million convertible preferred shares outstanding, which (including the annual 8.75% coupon) will be mandatorily convertible into approximately 23 million common shares on October 18, 2021. Assuming the outstanding options and convertible preferred shares were fully converted, the Company would have had approximately 89 million common shares outstanding as of June 30, 2019.

Conference Call Today

Arbutus will hold a conference call and webcast today, Monday, August 5, 2019 at 8:45 AM Eastern Time to provide a corporate update. You can access a live webcast of the call through the Investors section of Arbutus’ website at www.arbutusbio.com. Alternatively, you can dial (866) 393-1607 or (914) 495-8556 and reference conference ID 2098024.

An archived webcast will be available on the Arbutus website after the event. Alternatively, you may access a replay of the conference call by calling (855) 859-2056 or (404) 537-3406, and reference conference ID 2098024.