On November 7, 2017 Calithera Biosciences, Inc. (Nasdaq:CALA), a clinical stage pharmaceutical company focused on discovering and developing novel small molecule drugs directed against tumor metabolism and tumor immunology targets for the treatment of cancer reported that initial data from the ongoing trial of CB-839 in combination with Opdivo in patients with melanoma, renal cell carcinoma and non-small cell lung cancer will be presented Saturday, November 11th, 2017, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting (Press release, Calithera Biosciences, NOV 7, 2017, View Source [SID1234535250]). Calithera also announced it has expanded its existing clinical collaboration with Bristol-Myers Squibb, evaluating CB-839 in combination with Opdivo. CB-839 is an orally bioavailable glutaminase inhibitor currently in Phase 2 trials, and Opdivo is a PD-1 immune checkpoint inhibitor designed to overcome immune suppression.

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!

"This trial is designed to include patients who are currently receiving checkpoint inhibitor therapy and experiencing disease progression at the time of enrollment. There is significant unmet need among these patients, and the responses observed would not be expected with treatment with Opdivo alone in this population," said Susan M. Molineaux, Ph.D., founder, Chief Executive Officer and President of Calithera Biosciences. "The expanded clinical collaboration with Bristol-Myers Squibb will help us pursue our strategy of developing CB-839 in combination with Opdivo with the hope of improving treatment options for patients with cancer."

Calithera is conducting a Phase 1/2 trial (NCT02771626) in collaboration with Bristol-Myers Squibb designed to evaluate the safety, tolerability and efficacy of CB-839 in combination with Opdivo in patients with renal cell carcinoma, melanoma or non-small cell lung cancer. The study will be expanded to enroll additional melanoma patients. As part of the expanded collaboration, melanoma development costs will be shared, and a joint development committee will be established to guide the development and regulatory strategy.

The ongoing study enrolled three cohorts of patients who have received a checkpoint inhibitor (PD-1/PD-L1) in the most recent line of therapy.

Among 16 evaluable melanoma patients, all of whom were progressing on a checkpoint inhibitor at study entry, one patient achieved a complete response and two patients achieved partial responses. The overall response rate in this cohort was 19%, and the overall disease control rate was 44%.
Among six evaluable non-small cell lung cancer patients, all of whom were progressing on a checkpoint inhibitor at study entry, 67% experienced stable disease.
Among eight evaluable renal cell carcinoma patients, 75% were progressing and 25% had stable disease at study entry.

Stable disease was achieved in 75%, all of whom were progressing on a checkpoint inhibitor at study entry. The study enrolled one cohort of renal cell carcinoma patients who have received a checkpoint inhibitor in any prior line of therapy, but never achieved a response to checkpoint therapy.

Among seven evaluable checkpoint inhibitor experienced renal cell carcinoma patients, with a median of four prior lines of therapy, 57% experienced stable disease.

The study enrolled one cohort of renal cell carcinoma patients who were previously treated with VEGF inhibiting therapy, and were naïve to checkpoint inhibitors. Among 19 evaluable checkpoint inhibitor naïve renal cell carcinoma patients, four patients (21%) achieved a partial response and disease control rate was 74%. Fifty percent of the enrolled patients remain on study treatment.

An analysis of all safety evaluable patients demonstrated that CB-839 was well tolerated when combined with Opdivo in melanoma, renal cell carcinoma and non-small cell lung cancer patients. During dose escalation of the combination therapy, there was one report of dose limiting Grade 3 ALT increase, however no maximum tolerated dose was reported. The majority of adverse events reported have been mild to moderate with the most common being fatigue, nausea and photophobia. With 3.7% immune-related adverse events Grade ≥ 3, the data suggest there was no apparent increase in the rate or severity of immune related events compared to historical rates.

Dr. Funda Meric-Bernstam from MD Anderson Cancer Center will present the results on Saturday, November 11, 2017, in an oral and poster session (Abstract #O16), "A phase 1/2 study of CB-839, a first-in-class glutaminase inhibitor, combined with nivolumab in patients with advanced melanoma, renal cell carcinoma or non-small cell lung cancer." The oral presentation slides and the poster will be available on the Company’s website in the publication section at View Source

Calithera will webcast a clinical update on CB-839 on Saturday, November 11th at 3:30 p.m. Pacific Time/ 6:30 p.m. Eastern Time. The call can be accessed by dialing (855) 783-2599 (domestic) or (631) 485-4877 (international), and referring to conference ID 1878409. To access the live audio webcast or the subsequent archived recording, visit the Investors section of the Calithera website at www.calithera.com. The webcast will be recorded and available for replay on Calithera’s website for 30 days.

About CB-839
Calithera’s lead product candidate, CB-839, is a potent, selective, reversible and orally bioavailable inhibitor of glutaminase. CB-839’s onco-metabolism activity takes advantage of the unique metabolic requirements of tumor cells and cancer-fighting immune cells such as cytotoxic T-cells. It is currently being evaluated in Phase 2 clinical trials in multiple tumor types, in combination with standard of care agents.

On November 7, 2017 BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) reported financial results for the third quarter ended September 30, 2017 (Press release, BioCryst Pharmaceuticalsa, NOV 7, 2017, View Source [SID1234521627]).

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!

"Now that we have completed our end of Phase 2 regulatory interactions with the FDA and EMA and have agreement on the requirements for marketing authorization applications of BCX7353, we are focused on executing the Phase 3 program to support NDA and MAA filings in 2019," said Jon P. Stonehouse, President & Chief Executive Officer. "We expect to start the single required Phase 3 efficacy and the long-term safety trials in first quarter 2018."

Third Quarter Financial Results

For the three months ended September 30, 2017, revenues increased to $8.8 million from $7.8 million in the third quarter of 2016. The increase in revenue was primarily due to a $5.0 million milestone payment associated with the U.S. Food and Drug Administration (FDA) approval of a supplemental New Drug Application (sNDA) for RAPIVAB (peramivir injection), extending its availability for the treatment of acute uncomplicated influenza to pediatric patients two years and older, and the recognition of $1.5 million of peramivir product sales to the Company’s commercial partner, Green Cross Corporation. These increases were offset by a $3.1 million decrease in RAPIACTA royalties primarily due to lower government stockpiling sales by the Company’s commercial partner in Japan, Shionogi & Co. Ltd. (Shionogi), and lower collaboration revenue under U.S. Government development contracts.

Research and Development (R&D) expenses for the third quarter of 2017 increased to $17.5 million from $14.1 million in the third quarter of 2016, primarily due to increased spending on the Company’s hereditary angioedema (HAE) portfolio, including the achievement of a performance-based stock option grant related to the successful completion of the APeX-1 clinical trial. These increases were offset by a decrease in the Company’s galidesivir expenses under U.S. Government development contracts.

General and administrative (G&A) expenses for the third quarter of 2017 increased to $3.3 million compared to $2.8 million of expense in the third quarter of 2016. The increase was primarily due to the achievement of a performance-based stock option grant related to the successful completion of the APeX-1 clinical trial.

Interest expense was $2.1 million in the third quarter of 2017 as compared to $1.5 million in the third quarter of 2016, an increase related primarily to the September 2016 closing of a $23 million senior credit facility. Also, a $84,000 mark-to-market gain on the Company’s foreign currency hedge was recognized in the third quarter of 2017, as compared to a $931,000 mark-to-market loss in the third quarter of 2016. These changes result from periodic changes in the U.S. dollar/Japanese yen exchange rate. During the third quarter of 2017, the Company also realized a currency gain of $45,000 from the exercise of a U.S. Dollar/Japanese yen currency option within its foreign currency hedge.

The net loss for the third quarter of 2017 was $15.1 million, or $0.18 per share, compared to a net loss of $11 .5 million, or $0.16 per share, for the third quarter 2016.

Cash, cash equivalents and investments totaled $169.3 million at September 30, 2017, and reflect an increase from $65.1 million at December 31, 2016. Net operating cash use for the third quarter of 2017 was $10.6 million, and was $31.6 million for the first nine months of 2017, which excludes net proceeds from the March 2017 ($47.8 million) and September 2017 ($85.8 million) public offerings.

Year to Date Financial Results

For the nine months ended September 30, 2017, revenues increased to $21.3 million from $17.4 million in the first nine months of 2016. The increase in revenue was primarily due to $7.0 million of milestone payments associated with the Canadian regulatory and FDA sNDA approvals of RAPIVAB, and to a lesser extent a $1.2 million increase in royalty revenue from Shionogi & Co. Ltd., Green Cross Corporation and Seqirus. The increase in royalty revenue was largely the result of continued Japanese Government stockpiling of RAPIACTA. Future government stockpiling orders are difficult to predict, as they are subject to the relevant appropriation and stockpiling processes. These revenue increases were offset by a $5.5 million decrease in collaboration revenue under U.S. Government development contracts.

R&D expenses for the nine months of 2017 increased to $50.0 million from $48.9 million in the first nine months of 2016, primarily due to the achievement of a performance-based stock option grant related to the successful completion of the APeX-1 clinical trial. These increases were offset by a decrease in galidesivir expenses under U.S. Government development contracts.

G&A expenses for the nine months of 2017 increased to $9.2 million compared to $8.7 million in the first nine months of 2016. The increase was due to the achievement of a performance-based stock option grant related to the successful completion of the APeX-1 clinical trial.

In the nine months of 2017 and 2016, interest expense was $6.3 million and $4.4 million, respectively. The increase in interest expense was related primarily to the September 2016 closing of a $23 million senior credit facility. A $1.9 million mark-to-market loss on the Company’s foreign currency hedge was recognized in the first nine months of 2017, as compared to a $7.4 million mark-to-market loss in the first nine months of 2016. These losses result from periodic changes in the U.S. dollar/Japanese yen exchange rate. During 2017 and 2016, we also realized currency gains of $966,000 and $811,000, respectively, from the exercise of a U.S. Dollar/Japanese yen currency option within our foreign currency hedge.

The net loss for the nine months of 2017 was $46.2 million, or $0.58 per share, compared to a net loss of $50.6 million, or $0.69 per share for the same period last year.

Clinical Development Update & Outlook

In the fourth quarter, BioCryst completed end of Phase 2 regulatory interactions with the FDA and European Medicines Agency (EMA) resulting in finalizing the marketing authorization requirements, including agreement on the design of a single Phase 3 clinical trial and the details regarding a long-term safety trial.

On November 1, 2017, the FDA granted orphan drug designation to BCX7353 for the prevention and treatment of angioedema attacks in patients diagnosed with HAE.

On September 5, BioCryst announced positive final results from its Phase 2 APeX-1 clinical trial in HAE. APeX-1 was a 3-part dose ranging trial designed to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of orally administered once-daily BCX7353 for 28 days, as a preventative treatment to reduce the frequency of attacks in HAE patients.

On September 15, BioCryst announced the completion of an underwritten public offering of 17,864,078 shares of its common stock, including 2,330,097 shares sold pursuant to the exercise in full of the underwriters’ option to purchase additional shares. The gross proceeds from this offering, including from the shares sold pursuant to the underwriters’ option to purchase additional shares, were $92 million before deducting underwriting discounts and commissions and other estimated offering expenses.

On September 21, BioCryst announced that the U.S. FDA approved a sNDA for RAPIVAB (peramivir injection), an intravenous (i.v.) neuraminidase inhibitor, extending its availability for the treatment of acute uncomplicated influenza to pediatric patients two years and older who have been symptomatic for no more than two days.
Financial Outlook for 2017

Based upon development plans and our awarded government contracts, BioCryst expects its 2017 net operating cash use to be in the upper half of its previously disclosed range of $30 to $50 million, and its 2017 operating expenses to be in the upper half of its previously disclosed range of $53 to $73 million. Our operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the Company’s stock, as well as by the vesting of the Company’s outstanding performance-based stock options.

Conference Call and Webcast

BioCryst’s leadership team will host a conference call and webcast Tuesday, November 7, 2017 at 11:00 a.m. Eastern Time to discuss these financial results and recent corporate developments. To participate in the conference call, please dial 1-877-303-8027 (United States) or 1-760-536-5165 (International). No passcode is needed for the call. The webcast can be accessed by logging onto www.BioCryst.com. Please connect to the website at least 15 minutes prior to the start of the conference call to ensure adequate time for any software download that may be necessary.

About BCX7353

Discovered by BioCryst, BCX7353 is a novel, oral, once-daily, selective inhibitor of plasma kallikrein currently in development for the prevention and treatment of angioedema attacks in patients diagnosed with HAE. BCX7353 was generally safe and well tolerated in the recently completed Phase 2 APeX-1 clinical trial for the prophylaxis of angioedema attacks in patients with HAE and in clinical pharmacology studies in healthy volunteers.

About RAPIVAB (peramivir injection)

Approved by FDA in December 2014, RAPIVAB (peramivir injection) is an intravenous viral neuraminidase inhibitor approved for the treatment of acute uncomplicated influenza in patients two years and older who have been symptomatic for no more than two days. Efficacy of RAPIVAB is based on clinical trials of naturally occurring influenza in which the predominant influenza infections were influenza A virus and a limited number of patients infected with influenza B virus. Visit View Source to learn more.

On November 7, 2017 Five Prime Therapeutics, Inc. (NASDAQ:FPRX), a clinical-stage biotechnology company focused on discovering and developing innovative immuno-oncology protein therapeutics, previously reported that the abstract featuring data from the Phase 1a/1b clinical trial evaluating the immunotherapy combination of its CSF-1R antibody, cabiralizumab (FPA008), with Opdivo (nivolumab), Bristol-Myers Squibb’s PD-1 immune checkpoint inhibitor, has been selected for a late-breaking oral presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 32nd Annual Meeting, being held Nov. 8-12, 2017 in National Harbor, Maryland (Press release, Five Prime Therapeutics, NOV 7, 2017, View Source [SID1234521647]).

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!

Five Prime will host a conference call and live audio webcast on Saturday, November 11, 2017, at 5:45 p.m. (ET). Lewis T. "Rusty" Williams, President and Chief Executive Officer, Aron Knickerbocker, Chief Operating Officer, Helen Collins, Chief Medical Officer and Dr. Zev Wainberg, Assistant Professor of Medicine at UCLA, will review the data presented at SITC (Free SITC Whitepaper) earlier that day.

The live audio webcast may be accessed through the "Events & Presentations" page in the "Investors" section of the company’s website at www.fiveprime.com. Alternatively, participants may dial (877) 878-2269 (domestic) or (253) 237-1188 (international) and refer to conference ID 4092848.

The archived conference call will be available on Five Prime’s website beginning approximately two hours after the event and will be archived and available for replay for at least 30 days after the event.

On November 7, 2017 ChemoCentryx, Inc., (Nasdaq:CCXI), a biopharmaceutical company developing new medications targeted at inflammatory and autoimmune diseases and cancer, reported financial results for the third quarter ended September 30, 2017 (Press release, ChemoCentryx, NOV 7, 2017, View Source [SID1234521673]).

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!

"Our pursuit of new and better medicines for people with orphan diseases has been relentless," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "Dedicated to creating value for patients and shareholders alike, we at CCXI started with basic science in the discovery of novel molecules that selectively inhibit chemoattractant receptors, which are the molecular guidance systems of destructive inflammatory cells involved in a wide range of diseases and conditions. Now we have advanced two of those novel molecules, avacopan and CCX140, well into late-stage clinical trials. In doing so, we move closer to the next phase of value creation – the potential commercialization of our targeted medicines to help those suffering from serious renal diseases."

Recent Highlights

ChemoCentryx’s Phase III ADVOCATE trial of avacopan for the treatment of ANCA-associated vasculitis has surpassed 30 percent of its target patient enrollment with more than 185 sites activated. The trial will test the safety and efficacy of avacopan following 12 months of treatment and will include approximately 300 patients. In addition to testing the effect of avacopan on improving active vasculitis, the ADVOCATE trial will also test the effect of avacopan on preventing a recurrence of vasculitis.

ChemoCentryx recently received orphan designation in Switzerland from SwissMedic for avacopan for the treatment of two forms of ANCA-vasculitis: microscopic polyangiitis and granulomatosis with polyangiitis (formerly known as Wegener’s granulomatosis). This designation is in addition to the previously received orphan designations from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for avacopan to treat ANCA-vasculitis.

ChemoCentryx recently launched a registration-supporting clinical trial to study avacopan in a second indication, C3 Glomerulopathy (C3G), a rare disorder that often affects the young, requiring dialysis and often kidney transplant. Sites have been activated for the trial and patient enrollment has begun. Earlier this year ChemoCentryx announced that it had received both EMA orphan medicinal product designation and FDA orphan drug designation for avacopan in the treatment of C3G.

ChemoCentryx is launching a third registration-supporting trial, involving its CCR2 inhibitor, CCX140, to treat the debilitating kidney disorder known as Focal Segmental Glomerulosclerosis (FSGS), for which there is no approved treatment option. The Company plans to launch a trial in the fourth quarter of 2017.
Third Quarter 2017 Financial Results

Pro forma cash, cash equivalents, investments and remaining upfront commitments totaled $154.8 million at September 30, 2017.

Revenue was $9.0 million for the third quarter, compared to $4.1 million for the same period in 2016. The increase in revenue from 2016 to 2017 were due to: (i) amortization of the upfront license fee commitments from Vifor pursuant to the avacopan and CCX140 agreements; as well as (ii) collaboration revenue for development services under the CCX140 Agreement in 2017. These increases were partially offset by lower grant revenue from the FDA to support the clinical development of avacopan for the treatment of patients with ANCA vasculitis.

Research and development expenses were $12.3 million for the third quarter, compared to $8.4 million for the same period in 2016. The increase in research and development expenses from 2016 to 2017 was primarily attributable to the initiation and patient enrollment of the avacopan Phase III ADVOCATE trial in patients with ANCA vasculitis and start-up expenses for the Phase II clinical trial of avacopan for the treatment of C3G. These increases were partially offset by lower costs associated with the completion of the avacopan CLEAR and CLASSIC Phase II clinical trials for the treatment of ANCA vasculitis and enrollment completion of the CCX872 Phase I trial in patients with advanced pancreatic cancer in 2016.

General and administrative expenses were $3.6 million for the third quarter, compared to $3.2 million for the same period in 2016. The increase from 2016 to 2017 was primarily due to accounting related fees associated with preparing to meet the requirements pursuant to the Sarbanes-Oxley Act of 2002.

Net losses for the third quarter were $6.6 million, compared to $7.1 million for the same period in 2016.

Total shares outstanding at September 30, 2017 were approximately 48.8 million shares.

The Company expects to utilize cash and cash equivalents in the range of $50 million and $55 million in 2017, of which $39.0 million has been used for the nine months ended September 30, 2017.

Conference Call and Webcast

The Company will host a conference call and webcast today, November 7, 2017 at 5:00 p.m. Eastern Time / 2:00 p.m. Pacific Time. To participate by telephone, please dial 877-303-8028 (Domestic) or 760-536-5167 (International). The conference ID number is 7796287. A live and archived audio webcast can be accessed through the Investors section of the Company’s website at www.ChemoCentryx.com. The archived webcast will remain available on the Company’s website for fourteen (14) days following the conference call.

On November 7, 2017 OncoResponse reported that it has closed the acquisition of Paganini Biopharma, acquiring all of its assets including an anti-epithelial membrane protein 2 (EMP2) fully human monoclonal antibody, ONCR-201 (Press release, OncoResponse, NOV 7, 2017, View Source [SID1234521815]). EMP2 is implicated as an oncoprotein in cancer biology and in addition to its overexpression associated with tumor-promoting activities in various tumor types, it has been shown to play a role in signaling migration and invasion of cancer stem cells.

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!

"EMP2 is highly over expressed in breast cancer including triple negative, ovarian cancer and endometrial cancer as well as glioblastoma, and the anti-tumor effect of ONCR-201 in pre-clinical models is striking," said Clifford J. Stocks, CEO of OncoResponse. "Preclinical data support the potential of ONCR-201 to work as both a highly-targeted monotherapy as well as synergistically in combination with immunotherapy and current standard of care. ONCR-201 is now on an IND-track toward first-in-human studies to begin in 2019."

Financial terms of the acquisition were not disclosed.

Paganini Biopharma was founded on technology originated in the Department of Pathology and Laboratory Medicine of the School of Medicine at UCLA under a license agreement from the UCLA Technology Development Group. Paganini co-founder Madhuri Wadehra, Ph.D., Associate Professor at UCLA, will be a consultant to OncoResponse. "We are thrilled to put our technology and antibodies in the hands of strong drug developers and look forward to seeing this medicine reach cancer patients," said Gary Lazar, MD, CEO of Paganini.

About ONCR-201

ONCR-201 is a fully human recombinant monoclonal antibody developed to target and inhibit the function of epithelial membrane protein 2 (EMP2).

EMP2 is a cancer promoting protein, highly expressed in epithelial malignancies and associated with tumorigenesis, with triple negative breast cancer highly prominent on that list. EMP2 positive tumors have been shown to be more aggressive and invasive. Expression of EMP2 within the tumor correlates with faster disease progression and worse prognosis and is associated with the regulation of expression of several cancer stem cell-associated markers. Cancer stem cells, typically resistant to chemotherapy and radiation, are believed to be a main cause for cancer persistence and responsible for relapse, metastasis and rise of new tumors.

In preclinical studies, the administration of anti-EMP2 monoclonal antibody significantly reduces primary and secondary tumor load, and expression of cancer stem cell markers, and demonstrates benefit of survival that extends beyond cessation of treatment.