On August 8, 2017 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) reported financial results for the quarter ended June 30, 2017 (Press release, Inovio, AUG 8, 2017, View Source [SID1234520178]).

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Total revenue was $20.4 million for the three months ended June 30, 2017, compared to $6.2 million for the same period in 2016. Total operating expenses were $30.0 million for the current year quarter compared to $24.4 million for the prior year quarter.

The net loss attributable to common stockholders for the quarter ended June 30, 2017 was $9.5 million, or $0.13 per share, compared to $18.7 million, or $0.26 per share, for the quarter ended June 30, 2016. The decrease in net loss for the quarter resulted primarily from higher revenue recognized related to Inovio’s license and collaboration agreement with MedImmune entered into in 2015.

Revenue

The increase in revenue was primarily due to $13.8 million in revenue recognized from MedImmune, as the previously deferred revenue from the up-front payment received in September 2015 for MEDI0457 (INO-3112) was recognized during the three months ended June 30, 2017. This revenue recognition occurred upon MedImmune’s definitive selection of a new cancer product candidate to be tested in clinical trials against an undisclosed cancer target from our on-going research collaboration. The successful advancement of this new product candidate by MedImmune could also trigger future milestone payments and sales-based royalties payable to Inovio.

Operating Expenses

Research and development expenses for the second quarter of 2017 were $23.9 million compared to $19.6 million for the second quarter of 2016. The increase in R&D expenses was related to increased investment in all of Inovio’s product development programs, including its recently commenced phase 3 trial for VGX-3100. General and administrative expenses were $6.2 million for the second quarter of 2017 versus $5.8 million for the second quarter of 2016. The increase in G&A expenses primarily related to an increase in non-cash stock based compensation.

Capital Resources

As of June 30, 2017, cash and cash equivalents and short-term investments were $92.0 million compared with $104.8 million as of December 31, 2016. At quarter end the company had 77.6 million shares outstanding and 87.2 million shares outstanding on a fully diluted basis. During the three months ended June 30, 2017, Inovio sold 2,917,725 shares of common stock under its ATM common stock sales agreement for net proceeds of $24.1 million, with an average sale price of $8.41 per share.

On July 25, 2017, the company closed an underwritten public offering of 12,500,000 shares of common stock at a public offering price of $6.00 per share, for gross proceeds of $75.0 million. The net proceeds, after deducting the underwriters’ discounts and commissions and other estimated offering expenses payable by Inovio, were approximately $70.2 million. Inovio has granted the underwriters an option until August 18, 2017 to purchase up to 1,875,000 additional shares of its common stock on the same terms and conditions.

Inovio’s balance sheet and statement of operations are provided below. The Form 10-Q providing the complete 2017 second quarter financial report can be found at: View Source

Corporate Update

Clinical Developments

VGX-3100: Cervical Pre-Cancer (Phase 3)

In June, Inovio commenced its phase 3 clinical program to evaluate the efficacy of Inovio’s DNA-based immunotherapy, VGX-3100, to treat cervical dysplasia caused by human papillomavirus (HPV). Initiating Inovio’s first phase 3 program marks a significant milestone for the company, for the next generation of DNA-based immunotherapies, and for women’s health. In the phase 3 trial, Inovio will assess the efficacy of VGX-3100 in regressing cervical HSIL (high-grade squamous intraepithelial lesions; also called CIN2 or CIN3), a direct precursor to cervical cancer, and in eliminating the HPV infection that causes these lesions. The pivotal data from this program, if positive, could support the licensure of VGX-3100 as the first immunotherapy for this disease. HPV is the most common sexually transmitted infection, with over 14 million new infections annually.
Inovio satisfied the FDA’s request for information relating to its CELLECTRA 5PSP delivery device, resulting in the FDA removing a previously imposed clinical hold on this program. During the hold period, Inovio prepared investigational sites for the phase 3 study, resulting in the company opening 27 sites in just over the first month since the hold was removed. Inovio is on track to open at least 50 sites by the end of the year.
VGX-3100: Vulvar Pre-Cancer (Phase 2)

In April, Inovio commenced a randomized, open-label phase 2 trial to evaluate the efficacy of VGX-3100 in 36 women with high-grade HPV-related pre-cancerous lesions of the vulva, or vulvar intraepithelial neoplasia, a disease with a high unmet medical need. This is a new therapeutic indication for VGX-3100. The primary endpoint of the study is histologic clearance of high-grade lesions and virologic clearance of the HPV virus in vulvar tissue samples. The study will also evaluate safety and tolerability.
MEDI0457: HPV-Related Head & Neck Cancer (Phase 1/2)

In May, Inovio announced that MedImmune, AstraZeneca’s global biologics research and development arm, commenced a new clinical trial investigating the combination of MEDI0457 (formerly INO-3112) in-licensed from Inovio, an immunotherapy designed to generate antigen-specific killer T cell responses targeting HPV-associated tumors, and durvalumab, MedImmune’s PD-L1 checkpoint inhibitor. The combination trial will enroll patients with metastatic HPV-associated squamous cell carcinoma of the head & neck (SCCHN) with persistent or recurrent disease after chemotherapy treatment. This study marks a significant moment for Inovio as it transitions into a late-stage biotechnology company. MedImmune is investigating the possibility of elevating the response rate of checkpoint inhibitors by using durvalumab in combination with a DNA plasmid vaccine originally licensed from Inovio, which has shown the ability to generate killer T cells.
Combining the company’s first phase 3 program with the previously announced phase 2 clinical trial of VGX-3100 for treating HPV-related vulvar neoplasia, and the MEDI0457 checkpoint inhibitor-based combination study with MedImmune/AstraZeneca targeting HPV-associated, metastatic head and neck cancers, Inovio is well positioned to comprehensively treat HPV-associated diseases across the continuum of HPV infection through to cancer in both women and men.
Infectious Disease Studies

Inovio reported that its HIV vaccine, PENNVAX-GP, produced amongst the highest overall levels of immune response rates (cellular and humoral) ever observed in a human study by an HIV vaccine. The vaccine candidate, PENNVAX-GP, consists of a combination of four HIV antigens designed to cover multiple global HIV strains and generate both an antibody (humoral) immune response as well as a T cell (cellular) immune response to both potentially prevent and treat HIV. These significant results are consistent with Inovio’s recent data reported from its Ebola, Zika and MERS clinical trials in terms of achieving nearly 100% vaccine response rates with favorable safety profiles. Furthermore, Inovio’s newer and more tolerable intradermal vaccine delivery device showed that Inovio can elicit very high immune responses at a much lower dose.
Inovio and its academic and industry collaborators received a multi-year $6.95 million grant in March from the NIH’s National Institute of Allergy and Infectious Diseases to develop a single or combination therapy using Inovio’s PENNVAX-GP, with the goal of attaining long-term HIV remission in the absence of antiviral drugs. Development of Inovio’s PENNVAX-GP immunotherapy, which widely targets multiple major clades of HIV — providing global coverage — has been funded through a $25 million NIAID contract awarded to Inovio and its collaborators. In addition, Inovio and its collaborators were awarded a five-year $16 million Integrated Preclinical/Clinical AIDS Vaccine Development (IPCAVD) grant in 2015 from NIAID. PENNVAX-GP is currently being studied in a phase 1 clinical trial (HVTN-098) to evaluate its safety and immunogenicity in 94 healthy volunteers as a preventive vaccine (see above). The newly funded study will assess the impact of this vaccine approach in a therapeutic setting.
In preliminary results from the expanded stage of Inovio’s phase 1 clinical trial, EBOV-001, 95% (170 of 179) of evaluable subjects generated an Ebola-specific antibody immune response, with the mean antibody titer comparable or superior to those reported from viral vector-based Ebola vaccines, along with a more safety profile than those vaccines. This study was funded by a $45 million contract from DARPA.

Corporate Developments

In May, Inovio and Regeneron entered into an immuno-oncology clinical study agreement for glioblastoma (GBM) combination therapy. The planned Phase 1b/2a clinical trial will combine Regeneron’s PD-1 inhibitor REGN2810 and Inovio’s T cell activator INO-5401 and immune activator INO-9012 for the potential treatment of brain cancer. INO-5401 includes Inovio’s SynCon antigens for WT1, hTERT and PSMA and has the potential to be a powerful cancer immunotherapy in combination with checkpoint inhibitors. The National Cancer Institute previously highlighted WT1, hTERT and PSMA among a list of attractive cancer antigens, designating them as high priorities for cancer immunotherapy development, and placing WT1 at the top of the antigen list. The hTERT antigen is expressed in 85% of cancers; the WT1 and PSMA antigens are also widely prevalent in many cancers. The open-label trial, which is expected to begin later this year, is designed to evaluate the safety and efficacy of the combination therapy in approximately 50 patients. GBM is the most aggressive form of brain cancer, and its prognosis is extremely poor, despite a limited number of new therapies approved over the last ten years. Under the terms of the agreement, the trial will be solely conducted and funded by Inovio, based upon a mutually agreed upon study design, and Regeneron will supply REGN2810. Inovio and Regeneron will jointly conduct immunological analyses in support of the study. Regeneron, in collaboration with Sanofi, is developing REGN2810 both alone and in combination with other therapies for the treatment of various cancers.
In June, Inovio entered into a collaboration agreement with Genentech to commence a clinical trial to evaluate the combination of Inovio’s T cell immunotherapy INO-5401 and Genentech’s PD-L1 checkpoint inhibitor atezolizumab in patients with advanced bladder cancer. The phase 1b/2 immuno-oncology trial will evaluate Genentech’s atezolizumab (TECENTRIQ) in combination with Inovio’s INO-5401, a T cell activating immunotherapy encoding multiple antigens, and INO-9012, an immune activator encoding IL-12. The planned clinical trial is anticipated to start later this year, and is designed to evaluate the safety, immune response and clinical efficacy of the combination therapy in approximately 80 patients with advanced bladder cancer. Combining INO-5401/INO-9012 with atezolizumab may provide a synergistic therapeutic effect as a result of generating higher levels of activated T cells and simultaneously inhibiting PD-L1.
In July, Inovio completed an underwritten public offering of common stock, raising net proceeds of $70.2 million after underwriters’ discounts and commissions and estimated offering expenses. Inovio expects that with the net proceeds of the offering it will be able to advance its ongoing REVEAL 1 and 2 phase 3 trials and four phase 2 immuno-oncology trials and to fund other pipeline advancements. The financing also added new institutional investors to Inovio’s shareholder base.

The recent financing transaction will also support the following expected near-term events:
VGX-3100 phase 3 (cervical pre-cancer) trial – initiated
MEDI0457 phase 1/2 combination (head & neck cancer) study – initiated
INO-5401 Glioblastoma multiforme (brain cancer) phase 1/2 combination study with Regeneron — initiate 2H 2017
INO-5401 Bladder cancer phase 1/2 combination study with Genentech — initiate 2H 2017
INO-5150 Prostate cancer study (phase 1) report data – 3Q 2017
INO-1800 Hepatitis B therapy study (phase 1) report data — 4Q 2017
INO-1400 (hTERT) report interim immune response and safety data — 4Q2017
Vaccine clinical study publications (Zika, Ebola and MERS) – 4Q 2017

As previously announced in February, Inovio entered into a collaboration and license agreement with ApolloBio Corporation. If the agreement receives the requisite approvals from ApolloBio’s stock exchange, its board and its shareholders, the agreement will become effective, at which time Inovio expects to receive up to $50 million in payments from Apollo — $15 million in an upfront cash payment for the license of VGX-3100 in greater China and up to $35 million in the form of an equity investment in Inovio’s common stock.
Preclinical Developments

Nature Communications published a paper entitled "DNA Vaccination Protects Mice Against Zika Virus-Induced Damage to the Testes," reporting the results of a preclinical study in which Inovio’s Zika vaccine prevented the persistence of virus and damage in the male reproductive tract. This published data suggests another avenue of potential protection against the Zika virus. While detrimental effects on sperm and fertility have not yet been reported in Zika-infected human males, persistence of Zika in semen and sperm and sexual transmission by males has been documented. This new preclinical data suggests that our Zika vaccine may represent an opportunity to limit the potential for sexual transmission of the virus. In addition to our ongoing ZIKA-001 and 002 clinical studies, we are planning for a larger phase 2 study in our efforts to bring our Zika vaccine to patients.
Npj Vaccines published a paper entitled "DNA Inoculation of Synthetic Cross-Reactive Antibodies Protects Against Lethal Influenza A and B Infections," co-authored by Inovio scientists and collaborators from the Wistar Institute and MedImmune. The paper reported the results of a preclinical study in which Inovio’s DNA-based monoclonal antibody product candidate for the treatment of influenza produced broadly cross-reactive antibodies that provided complete protection from a lethal challenge with multiple viruses of both influenza A and B types. Following previously reported similar data from Inovio’s dMAb candidates for HIV, dengue, and Chikungunya, this study further validates the potential for Inovio’s dMAb technology platform to be able to use encoded DNA plasmids for in vivo production of monoclonal antibodies and to induce protective immune responses. The goal for this platform is to rapidly generate therapeutic monoclonal antibodies directly in the recipients. Such benefits are complementary to Inovio’s antigen-generating platform in terms of immune mechanism and short response times, and advantages that overcome conventional monoclonal antibodies’ long development lead times and complex manufacturing processes and costs.

On August 8, 2017 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported company highlights and financial results for the second quarter ended June 30, 2017 (Press release, Ignyta, AUG 8, 2017, View Source [SID1234520175]). The company is issuing this press release in lieu of conducting a conference call.

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"During the second quarter, we provided substantial development, regulatory, and commercial strategy updates for our lead product candidate, entrectinib, a novel, investigational, orally available, CNS-active tyrosine kinase inhibitor targeting tumors that harbor TRK, ROS1, or ALK fusions," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "In addition, we received breakthrough therapy designation for entrectinib, presented exciting preclinical immunomodulation data for RXDX-106, and strengthened our balance sheet via an equity offering, providing us with the resources to continue developing meaningful new therapies for patients with cancer."

Company Highlights

Updated Progress Towards Entrectinib Dual TRK and ROS1 NDA and PMA Submissions

In April 2017, we announced a comprehensive program update on entrectinib and the STARTRK-2 trial. As of that update:

More than 50 patients with ROS1 fusion-positive non-small cell lung cancer (NSCLC) were enrolled; interim data from 32 of these patients (as assessed by investigator) demonstrated 75% confirmed RECIST objective response rate (ORR) (24 partial or complete responses out of 32) and 17.2 months median duration of response (DOR)
Entrectinib demonstrated confirmed RECIST Intracranial ORR (IC-ORR) of 83% (5 partial responses out of 6) in ROS1 NSCLC patients with measurable central nervous system (CNS) metastases
The entrectinib program was more than 85% enrolled to goal for the primary efficacy analysis to potentially support a TRK tissue agnostic NDA submission
The program is tracking towards dual NDA submissions in TRK and ROS1 in 2018, if supported by clinical data, with anticipated U.S. commercial launch in both indications in 2019.

Breakthrough Therapy Designation and Orphan Drug Designation for Entrectinib

In May 2017, the company announced that the U.S. Food and Drug Administration (FDA) granted a Breakthrough Therapy Designation (BTD) to entrectinib "for the treatment of NTRK fusion-positive, locally advanced or metastatic solid tumors in adult and pediatric patients who have either progressed following prior therapies or who have no acceptable standard therapies."

In July 2017, the company announced that FDA granted orphan drug designation to entrectinib for "treatment of NTRK fusion-positive solid tumors."

RXDX-106 AACR (Free AACR Whitepaper) Presentations

In April 2017, we presented preclinical data at the Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in Washington D.C. suggesting that RXDX-106 can act as both an anti-tumor immuno-modulator and TYRO3, AXL and MER (or TAM) oncodriver inhibitor, potentially supporting clinical development of RXDX-106 in a wide variety of cancers. RXDX-106 represents a novel class of immunomodulatory agents that appears to restore innate immunity in preclinical models via potent inhibition of the TAM family of receptors.

Financing Transaction

In May 2017, the company issued an aggregate of 14.375 million shares of its common stock in an underwritten public offering at a price to the public of $6.15 per share, which resulted in aggregate gross proceeds of $88.4 million.

Second Quarter 2017 Financial Results

For the second quarter of 2017, net loss was $28.3 million, or $0.56 per share, compared with $26.7 million, or $0.70 per share, for the second quarter of 2016.

Ignyta did not record any revenue for the second quarter of 2017, or for the second quarter of 2016.

Research and development expenses for the second quarter of 2017 were $22.2 million, compared with $20.0 million for the second quarter of 2016. This increase was due to an increase in external clinical development costs and the chemistry, manufacturing and control costs associated with entrectinib and our other product candidates, and increased facilities costs of $1.2 million due to the expansion of our leased facilities space. We also incurred additional stock compensation costs of $0.8 million due in part to the increase in the number of outstanding stock options.

General and administrative expenses were $5.5 million for the second quarter of each of 2017 and 2016, respectively. There was no measurable change in our general and administrative expenses between the two periods, as the increases in facilities costs and outside services expenses were offset by a reduction in our personnel related expenditures.

At June 30, 2017, we had cash, cash equivalents and available-for-sale securities totaling $169.4 million and current and long-term debt of $32.0 million. At December 31, 2016, we had cash, cash equivalents and available-for-sale securities totaling $133.0 million and current and long-term debt of $32.0 million.

On August 8, 2017 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported financial results and recent highlights for the second quarter ended June 30 (Press release, Halozyme, AUG 8, 2017, View Source [SID1234520172]).

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"A clear highlight of the second quarter was approval of Genentech’s RITUXAN HYCELATM in the U.S., and the resulting strong interest from new potential partners who seek to coformulate with our ENHANZE technology," said Dr. Helen Torley, president and chief executive officer. "We have strong momentum in the ENHANZE business with continued growth in royalties and a number of catalysts in the second half of 2017, including Lilly’s recent start of their Phase 1 study, Janssen’s upcoming start of their Phase 3 study of subcutaneous daratumumab and our expectation for a new collaboration agreement.

"At the same time, we are executing well in our HALO-301 study of PEGPH20 with screening and enrollment tracking to our expectations and investigator enthusiasm continuing to be strong. I also remain encouraged by recent progress screening and enrolling patients in the dose expansion portion of our Phase 1b study of PEGPH20 in combination with KEYTRUDA and the recent initiation of Genentech’s study of PEGPH20 with TECENTRIQ.

"With strategic and operational progress across both pillars, we look forward to the potential for additional value inflecting events in the coming months."

Second Quarter 2017 and Recent Highlights include:

Approval of Genentech’s RITUXAN HYCELA by the Food and Drug Administration (FDA). The combination of rituximab and Halozyme’s hyaluronidase human ENHANZE technology has been approved for patients with follicular lymphoma, diffuse large B-cell lymphoma and chronic lymphocytic leukemia and is now available to patients in more than 60 countries worldwide.
Eli Lilly initiating a Phase 1 study of an investigational new therapy in combination with Halozyme’s ENHANZE technology as part of the companies’ 2015 Global Collaboration and Licensing agreement.
Johnson and Johnson highlighting progress with the subcutaneous formulation of DARZALEX (daratumumab) during their 2017 Pharmaceutical Business Review. The formulation, which uses Halozyme’s ENHANZE technology to enable a 5-minute infusion, is currently being studied in a Phase 1b clinical trial and planned to begin a Phase 3 study later this year.
Progress in the HALO-301 study of PEGPH20 in combination with ABRAXANE (nab-paclitaxel) and gemcitabine in first line metastatic pancreas cancer patients with high levels of tumor hyaluronan (HA-High). Screening and enrollment in the study continues to track in line with expectations and investigator enthusiasm remains strong.
Initiation of a Genentech-funded Phase 1b/2 multi-arm clinical trial evaluating PEGPH20 with TECENTRIQ (atezolizumab) in patients with previously treated metastatic pancreatic ductal adenocarcinoma. The study is part of a clinical collaboration agreement announced in 2016 to evaluate PEGPH20 and atezolizumab in up to eight tumor types.
Progress in the dose expansion phase of the ongoing Phase 1b clinical study evaluating PEGPH20 in combination with KEYTRUDA (pembrolizumab) in non-small cell lung and gastric cancer patients. Halozyme may report initial response rate data by the end of the year, depending on the pace of enrollment and time to response.
Oral presentations of Halozyme’s Phase 2 randomized HALO-202 study data at the European Society for Medical Oncology’s World Congress on Gastrointestinal Cancer and American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting. The presentations expanded on the topline results shared in January with additional data from the study as of December 2016.
Second Quarter 2017 Financial Highlights

Revenue for the second quarter was $33.8 million compared to $33.3 million for the second quarter of 2016. The year-over-year increase was driven by growth in royalties from partner sales of Herceptin SC, MabThera SC and HYQVIA, offset by a decrease in research and development reimbursements and license payments from ENHANZE partners. Revenue for the second quarter included $14.7 million in royalties, an increase of 20 percent from the prior-year period, $8.9 million in sales of bulk rHuPH20 primarily for use in manufacturing collaboration products and $3.9 million in HYLENEX recombinant (hyaluronidase human injection) product sales.
Research and development expenses for the second quarter were $38.3 million, compared to $35.5 million for the second quarter of 2016. The increase was primarily due to a ramp in spending associated with the HALO-301 study.
Selling, general and administrative expenses for the second quarter were $13.1 million, compared to $11.2 million for the second quarter of 2016. The increase was primarily due to personnel expenses, including stock compensation, for the period.
Net loss for the second quarter was $30.8 million, or $0.23 per share, compared to net loss in the second quarter of 2016 of $26.9 million, or $0.21 per share.
Cash, cash equivalents and marketable securities were $297.5 million at June 30, 2017, compared to $179 million at March 31, 2017.
Financial Outlook for 2017

Halozyme increased year-end cash guidance and reiterated all other components of its financial guidance, now expecting:

Net revenue of $115 million to $130 million;
Operating expenses of $240 million to $250 million;
Operating cash burn of $75 million to $85 million; and
Year-end cash balance of $245 million to $260 million, an increase from its prior range of $110 million to $125 million to reflect net proceeds of $135 million from a previously announced public offering of 11.5 million shares of common stock.

On August 8, 2017 Five Prime Therapeutics, Inc. (Nasdaq:FPRX), a clinical-stage biotechnology company focused on discovering and developing innovative immuno-oncology protein therapeutics, reported a corporate update and reported financial results for the quarter ending June 30, 2017 (Press release, Five Prime Therapeutics, AUG 8, 2017, View Source [SID1234520168]).

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"We continued to make significant progress on our clinical and pre-clinical programs during the quarter," said Lewis T. "Rusty" Williams, M.D., Ph.D., president and chief executive officer of Five Prime. "We are advancing our large Phase 1a/1b immuno-oncology trial studying cabiralizumab with OPDIVO in multiple tumor settings. We completed enrollment in some of the cohorts and are on track to complete enrollment in all the cohorts by the end of the year. We and BMS also plan to disclose initial clinical trial data at the SITC (Free SITC Whitepaper) annual meeting in November. At the ASCO (Free ASCO Whitepaper) annual meeting, we announced initial clinical trial data from our Phase 1/2 trial of cabiralizumab in pigmented villonodular synovitis, or PVNS, in which cabiralizumab clearly demonstrated clinical benefit in patients. We also reported encouraging monotherapy activity for FPA144 in heavily pretreated gastric cancer patients, and plan to initiate a front-line chemotherapy combination trial. Additionally, we’re on track to file at least one IND application for a new molecule each year, including this year."

Business Highlights and Recent Developments

Clinical Pipeline:

Cabiralizumab (FPA008): an investigational antibody that inhibits CSF1R and has been shown to block the activation and survival of monocytes and macrophages.

– Advanced the ongoing Phase 1a/1b trial of cabiralizumab/OPDIVO in immuno-oncology.

– Five Prime and Bristol-Myers Squibb (BMS), are evaluating the safety, tolerability and preliminary efficacy of the immunotherapy combination of cabiralizumab with the PD-1 immune checkpoint inhibitor OPDIVO (nivolumab) in advanced solid tumors, including non-small cell lung cancer, squamous cell carcinoma of the head and neck, pancreatic cancer, glioblastoma, renal cell carcinoma and ovarian cancer.

– Five Prime completed enrollment in some of the Phase 1b cohorts and expects to complete enrollment in all the cohorts in the second half of 2017.

– Five Prime and BMS expect to present initial clinical trial data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting in November.

– Advanced the ongoing Phase 1/2 trial of cabiralizumab in patients with PVNS.

– Announced initial clinical trial data at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual meeting in June.
– There were no dose-limiting toxicities (DLTs) observed at doses up to 4 mg/kg.
– Cabiralizumab demonstrated clinical benefit in patients with PVNS.
– Most patients enrolled at the 4 mg/kg dose experienced tumor reduction.
– 5 out of 11 patients had a radiographic response (4 confirmed).
– Improvement in median Ogilvie-Harris composite score of pain and function was reported in both responders and non-responders.

– Five Prime plans to enroll additional patients in the Phase 2 portion of the trial to refine the dosing schedule to optimize the therapeutic index of cabiralizumab in this chronic disease setting. These additional data are intended to support a pivotal trial for cabiralizumab in PVNS.
FPA144: an isoform-selective antibody in development as a targeted immuno-therapy for tumors that overexpress FGFR2b, a splice variant of a receptor for some members of the fibroblast growth factor (FGF) family. Five Prime plans to initiate a global pivotal trial of FPA144 combined with chemotherapy as a front-line treatment for metastatic gastric cancer. In addition, Five Prime is adding a blood-based diagnostic tool to increase the addressable population to 10% of patients with gastric cancer.

– Advanced the Phase 1 monotherapy trial of FPA144 in patients with gastric cancer. Enrollment continues in the expansion portion of the trial, evaluating the safety, PK and efficacy of biweekly 15 mg/kg infusions of FPA144 in patients with gastric cancer whose tumors overexpress FGFR2b.

– Announced updated clinical trial data from the Phase 1 trial of single-agent FPA144 at the 2017 ASCO (Free ASCO Whitepaper) Annual Meeting.

– FPA144 was well tolerated at doses tested up to 15 mg/kg in patients with advanced solid tumors, including patients with gastric cancer.
– FPA144 monotherapy demonstrated early evidence of anti-tumor efficacy in heavily pre-treated patients (median of 3 prior lines of therapy).
– Radiographic responses:
– 5 Partial Responses (4 confirmed, 1 unconfirmed) in 21 patients (across three dose levels)
– Objective Response Rate (ORR): 19%
– Median duration of response of 15.4 weeks

– Activities underway to initiate a global registrational clinical trial in 2018 for FPA144 in combination with chemotherapy as a front-line gastric cancer therapy.

– Launched a Phase 1 safety trial for FPA144 monotherapy in patients with gastric cancer in Japan, where the incidence of gastric cancer is high. Completion of this Phase 1 trial is intended to enable the inclusion of Japanese patients in the planned global Phase 3 clinical trial.

– Developing companion diagnostics to identify the 10% of gastric cancer patients eligible for FPA144 therapy. Five Prime plans to use either immunohistochemistry (IHC) or circulating tumor DNA (ctDNA) tests to identify eligible patients for its global Phase 3 clinical trial. By adding the ctDNA test, Five Prime believes it will double the eligible patient population to 10% from the previous 5% identified by IHC testing alone.

– Preparing for a Phase 1 safety trial to test the combination of FPA144 with chemotherapy. Five Prime plans to begin dosing patients in a Phase 1 clinical trial to test the safety of ascending doses of FPA144 in combination with chemotherapy by the end of 2017. This safety trial will support the start of the global Phase 3 clinical trial.

– Discussing design of Phase 3 clinical trial with regulatory authorities. Five Prime has begun discussions with regulatory authorities and is actively working on the design of the Phase 3 clinical trial of FPA144 in combination with chemotherapy as a front-line gastric cancer therapy.

– Advanced the Phase 1 monotherapy trial of FPA144 in patients with bladder cancer. The company opened an additional cohort in the Phase 1 clinical trial to test FPA144 as a treatment for bladder cancer patients whose tumors overexpress FGFR2b, as assessed by the company’s IHC test. Five Prime is adding sites that specialize in bladder cancer to support enrollment in this cohort.

FP-1039: a protein drug designed to block FGF signaling. As a ligand trap, FP-1039 binds to and neutralizes a subset of FGF ligands (such as FGF2), preventing these growth-promoting and angiogenic proteins from reaching FGFR1 on the surface of tumor cells.

– Clinical data from the phase 1b trial in mesothelioma have been accepted as an oral presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress in September. Our former partner, GlaxoSmithKline (GSK), is completing the Phase 1b trial combining FP-1039 with front-line pemetrexed and cisplatin in untreated, unresectable mesothelioma. GSK concluded trial recruitment with 25 patients enrolled at the 15 mg/kg dose in June 2016.
Preclinical Research and Development:

Progress in pre-clinical and research programs.
– Five Prime is on track to achieve the goal of filing at least one IND application for a new molecule each year for the foreseeable future, beginning this year.

Continue to advance three preclinical development candidates in IND-enabling studies.
– FPA150 (anti-B7-H4)
– An antibody designed for two mechanisms of action: to block an inhibitory T cell checkpoint pathway and to enhance killing of B7-H4-expressing tumors by ADCC. B7-H4 is frequently overexpressed in breast, ovarian and endometrial cancers.
– Investigational New Drug (IND) application planned for the fourth quarter of 2017.
– FPA150 was selected for an oral poster discussion during the ESMO (Free ESMO Whitepaper) 2017 Congress.

– FPT155 (CD80-Fc)
– A multi-targeting immune modulator that can stimulate T cell responses through three critical pathways: CTLA4 blockade, CD28 agonism (without superagonism) and PD-L1 blockade.
– Potential 2018 IND application.

– FPA154 (GITR agonist antibody)
– A tetravalent agonist antibody designed for greater GITR activation versus conventional antibodies. Conventional GITR agonist antibodies have two GITR binding sites while FPA154 has four.
– Potential 2018 IND application.
Summary of Financial Results and Guidance:

Cash Position. Cash, cash equivalents and marketable securities totaled $350.7 million on June 30, 2017, compared to $421.7 million on December 31, 2016. The decrease in cash was primarily attributable to cash used in operations to advance the FPA144 clinical development program, the cabiralizumab Phase 2 clinical trial in PVNS and preclinical development programs.

Revenue. Collaboration revenue for the second quarter of 2017 decreased by $1.4 million to $7.8 million from $9.2 million in the second quarter of 2016. This decrease was primarily due to completing the research term of our research collaboration with GSK in respiratory diseases in July 2016 offset by revenue recognized under the 2015 cabiralizumab collaboration agreement with BMS, under which Five Prime is reimbursed for the expenses from the cabiralizumab immuno-oncology trial.

R&D Expenses. Research and development expenses for the second quarter of 2017 increased by $19.5 million to $41.7 million from $22.2 million in the second quarter of 2016. This increase was primarily related to advancing cabiralizumab in the Phase 2 clinical trial in PVNS and the Phase 1a/1b clinical trial in immuno-oncology and advancing pre-clinical development programs towards IND filings.

G&A Expenses. General and administrative expenses for the second quarter of 2017 increased by $1.3 million to $9.4 million from $8.1 million in the second quarter of 2016. This increase was primarily due to increases in payroll and stock-based compensation expenses.
Net Loss. Net loss for the second quarter of 2017 was $44.3 million, or $1.58 per basic and diluted share, compared to a net loss of $13.1 million, or $0.49 per basic and diluted share, for the second quarter of 2016.

Shares Outstanding. Total shares outstanding were 28.8 million as of June 30, 2017.
Cash Guidance. Five Prime expects full-year 2017 net cash used in operating activities to be less than $120 million. The company estimates ending 2017 with slightly less than $300 million in cash, cash equivalents and marketable securities.

On August 8, 2017 Endocyte, Inc. (NASDAQ:ECYT), a leader in developing targeted small molecule drug conjugates (SMDCs) and companion imaging agents for personalized therapy, reported financial results for the second quarter ending June 30, 2017, and provided a clinical and pipeline update (Press release, Endocyte, AUG 8, 2017, View Source [SID1234520166]).

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"We’ve continued to make important progress on each of our three development programs," said Mike Sherman, President and CEO of Endocyte. "Dr. Michael Jensen at the Seattle Children’s Research Institute has exceeded our expectations in the breadth of work he has completed in optimizing the vector to be used in our chimeric antigen receptor T-cell (CAR T-cell) therapy, and we are happy to report that we expect to initiate the CAR T-cell manufacturing process for clinical supply in the fourth quarter of this year. In addition, we have filed an Investigational New Drug (IND) application for EC2629, which we believe is the first agent in development that simultaneously targets cancer cells and the tumor associated macrophages (TAMs) that support and protect them – an approach that could continue to evolve the immunotherapy treatment landscape. Finally, we expect to complete enrollment of taxane-exposed prostate cancer patients in the EC1169, PSMA-tubulysin expansion trial this fall."

"We believe our pipeline has significant potential to create value and we are committed to effective, timely execution in bringing these assets forward through clinical development and identifying paths to accelerate value-driving catalysts. With this in mind, our strategy is to select receptor-positive patients in highly-targeted indications from the beginning of development, including during dose escalation," continued Mr. Sherman. "We will also continue to objectively measure our pipeline investments relative to opportunities to outlicense assets or access external opportunities to ensure we are deploying capital productively."

Development Programs Overview

CAR T-Cell (Bi-specific adaptor molecule): Today, Endocyte announced that Dr. Michael Jensen of Seattle Children’s Research Institute will lead the clinical evaluation of Endocyte’s first CAR T-cell adaptor molecule in patients with osteosarcoma. This is primarily a pediatric indication with a significant need for new therapeutic options. Recent results from tumor micro-array analysis and human intravital fluorescent imaging studies have confirmed this disease as positive for the folate receptor, the target of Endocyte’s first bi-specific adaptor. Pre-clinical evaluations for the CAR T-cell program by Dr. Jensen are expected to be completed in the second half of 2017, in anticipation of a potential IND filing in 2018. Multiple additional adaptor molecules designed to be directed to distinct tumor targets including, potentially, PSMA, NK1R and others, are in development through the company’s collaboration with Purdue University.

In April, Endocyte announced new research in a late-breaking poster session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting on this application of Endocyte’s SMDC technology. Data demonstrated that Endocyte’s bi-specific adaptor molecules can mitigate or eliminate adverse cytokine storms in animal models which could meaningfully improve the safety and tolerability of CAR T-cell therapies.

EC1169 (PSMA-targeted tubulysin): Endocyte is currently enrolling a phase 1 expansion cohort of 40 metastatic castration-resistant prostate cancer (mCRPC) patients who have previously been treated with a taxane-based therapy. Data presented at the annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in June demonstrated EC1169’s anti-tumor activity particularly in patients with previous exposure to taxane therapy. Endocyte stopped enrollment of taxane-naïve mCRPC patients in the trial in June. Updated interim results will be presented at the annual meeting of the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) in September. This presentation is expected to be an incremental update to data presented at ASCO (Free ASCO Whitepaper) a few months prior. Completion of enrollment is expected in the fall.

EC2629 (Folate-targeted PBD): Endocyte recently filed an IND with the U.S. FDA and is planning to initiate a phase 1 clinical trial in patients selected as positive for the folate receptor in cancers where TAMs are known to be prevalent in the tumor micro-environment, for example, breast, endometrial, ovarian, and non-small cell lung cancers. This novel agent leverages a proprietary DNA crosslinking warhead targeted to cancer cells and the TAMs that both support their growth as well as protect them from the immune system. This mechanism is particularly compelling in light of recent research that has identified mechanisms by which TAMs can mediate resistance to the use of checkpoint inhibitor therapies such as PD-1 and PD-L1.

Financial Expectations

The company anticipates its cash, cash equivalents and investments balance at the end of 2017 to be approximately $105 million. As the full expense impact of the company’s restructuring is expected to be realized by the end of the fourth quarter of 2017, the company anticipates cash expenses to be approximately $5 million per quarter prior to potential increases associated with advancing clinical trials and new investment opportunities currently under evaluation.

Second Quarter 2017 Financial Results

Endocyte reported a net loss of $11.7 million, or $0.28 per basic and diluted share, for the second quarter of 2017, compared to a net loss of $14.0 million, or $0.33 per basic and diluted share for the same period in 2016.

In June 2017, the company stopped enrollment in its EC1456 phase 1b trial as the assessment of trial data did not yield the level of clinical activity necessary to support continued advancement of EC1456. The company is, however, continuing enrollment of a small number of patients in its EC1456 ovarian cancer surgical study to inform other SMDC programs in development. In addition, in June, Endocyte narrowed the focus of its EC1169 development program, refocused its efforts on two pre-clinical programs, and reduced its workforce by approximately 40% to align resources to focus aggressively on the company’s highest value opportunities while maintaining key capabilities. Endocyte recorded $2.3 million of restructuring expenses for the three months ended June 30, 2017 as follows:

Included in research and development expenses were expenses for employee termination benefits of $0.9 million, $0.9 million for the remaining EC1456 phase 1b trial expenses, including site close-out expenses, $0.3 million related to other restructuring expenses, and $0.1 million related to fixed asset impairment charges; and
Included in general and administrative expenses were expenses for employee termination benefits of $0.1 million.
Research and development expenses were $8.7 million for the second quarter of 2017, compared to $6.8 million for the same period in 2016. The increase was primarily attributable to $2.2 million of expenses recorded in June due to the company’s restructuring relating primarily to severance for the workforce reduction, EC1456 trial termination expenses and fixed asset impairment charges. Other increases included expenses for the EC1169 phase 1 trial, development of EC2629 and other pre-clinical and general research. These increases were partially offset by a decrease in non-cash stock compensation expense as a result of employee terminations since the second quarter of 2016.

General and administrative expenses were $3.3 million for the second quarter of 2017, compared to $7.4 million for the same period in 2016. The decrease was due to a decrease in compensation expense, including non-cash stock compensation expense and severance expense related to the resignation of our former Chief Executive Officer in June of 2016.

Cash, cash equivalents and investments were $118.4 million at June 30, 2017, compared to
$154.6 million at June 30, 2016, and $138.2 million at December 31, 2016.