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Geron Provides Update on Imetelstat Trials Being Conducted by Janssen

On September 12, 2016 Geron Corporation (Nasdaq:GERN) reported updates on the clinical trials being conducted by Janssen Research & Development, LLC, of the telomerase inhibitor imetelstat (Press release, Geron, SEP 12, 2016, View Source [SID:SID1234515066]). Planned internal reviews of initial data from both trials have been completed by Janssen, and both trials are continuing in order to evaluate additional and more mature data.

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IMbarkTM

IMbarkTM (NCT02426086) was originally designed as a Phase 2 clinical trial to evaluate two dose levels of imetelstat (either 4.7 mg/kg or 9.4 mg/kg administered every three weeks) in approximately 200 patients (approximately 100 patients per dosing arm) with Intermediate-2 or High risk myelofibrosis (MF) who have relapsed after or are refractory to prior treatment with a JAK inhibitor. The co-primary efficacy endpoints for the trial are spleen response rate and symptom response rate at 24 weeks. To date, over 90 patients have been enrolled in the trial across both dosing arms.

To inform an assessment of the appropriate dose and schedule for relapsed or refractory MF patients in IMbarkTM, Janssen conducted a planned internal interim review of safety, efficacy and pharmacokinetic data from 20 patients from each dosing arm who have been followed on the trial for at least 12 weeks. Based on this first internal review at the early 12-week time point, the following has been determined by Janssen:

The safety profile was consistent with previous imetelstat clinical trials in hematologic myeloid malignancies. No new safety signals were identified.

Activity in the 4.7 mg/kg dosing arm does not warrant further investigation of that dose and this arm will be closed to new patient enrollment. An amendment to the trial protocol is planned to allow eligible patients in this arm to increase their dose to 9.4 mg/kg per investigator discretion.

In the 9.4 mg/kg dosing arm, even though at the week 12 data assessment an insufficient number of patients met the protocol defined interim criteria, this arm warrants further investigation because encouraging trends in the efficacy data were observed. Patients already enrolled in this arm may continue to receive imetelstat. New enrollment in this arm will be suspended while the trial continues in order to obtain additional and more mature data that includes a longer follow-up of patients at 24 weeks, consistent with the co-primary efficacy endpoints. The number of patients enrolled to date is expected to be sufficient to inform potential future development of this dose.

Janssen plans to conduct an additional internal data review in the second quarter of 2017 to include a longer follow-up of patients at 24 weeks. Potential outcomes of the second internal review at the 24-week time point could include resuming enrollment in the 9.4 mg/kg dosing arm, with or without changes to the dosing regimen; adding a new dosing arm; or closing the trial.

Any protocol amendments will be subject to review by health authorities around the world.
IMergeTM

IMergeTM (NCT02598661) is a Phase 2/3 clinical trial evaluating imetelstat in transfusion dependent patients with Low or Intermediate-1 risk myelodysplastic syndromes (MDS) who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA). The clinical trial is in two parts: Part 1 is a Phase 2, open-label, single-arm design in approximately 30 patients and Part 2 is a Phase 3, randomized, double-blind, placebo-controlled design in approximately 170 patients. The primary efficacy endpoint is the rate of red blood cell transfusion-independence lasting at least 8 weeks. Part 1 of the trial is fully enrolled.

Janssen has conducted an initial internal review of efficacy, safety and pharmacokinetic data from a subset of patients from Part 1 of IMergeTM and this review indicated that emerging safety and efficacy in IMergeTM is consistent with data reported from the pilot study conducted at Mayo Clinic in MDS patients. IMergeTM will continue unmodified at this time.

Further assessment of data from IMergeTM is expected to occur in the second quarter of 2017 to include longer follow-up of all patients enrolled in Part 1. A decision on whether to move forward to Part 2 of IMergeTM will be based on an assessment of the benefit/risk profile of imetelstat in these patients. If Janssen decides to move forward with Part 2, the Phase 3 clinical trial is expected to be open for patient enrollment in mid-2017.

Janssen expects to submit data from Part 1 of IMergeTM to be considered for presentation at a medical conference in the future.

Celyad completes the NKR-2 Phase I trial with successful safety follow-up of the fourth dose level

On September 9, 2016 Celyad (Euronext Brussels and Paris, and NASDAQ: CYAD), a leader in the discovery and development of cell therapies, reported the successful completion of the 21-day safety follow-up of the last patient enrolled at the fourth dose level in its Phase I clinical trial evaluating the safety and feasibility of its NKR-2 T-cell therapy – in Acute Myeloid Leukemia and Multiple Myeloma patients (Press release, Celyad, SEP 9, 2016, View Source [SID1234516410]). No safety issues or toxicities were reported.

Dr. Christian Homsy, CEO of Celyad: "We are pleased that no adverse safety signal has been reported. Based on the successful completion of the fourth cohort, we are looking forward to starting the global Ph I/IIa multiple dose trial, in the US and EU, of our NKR-2 autologous therapy in the fourth quarter of this year."

Dr. Frédéric Lehmann, Head of Immuno-Oncology at Celyad: "The absence of any safety or toxicity signal up to this point continues to support our belief that this unique engineered T-cell construct can be safely administered and potentially lead to a therapeutic effect providing hope to the thousands of patients who need better treatments for both AML and MM."

8-K – Current report

On September 12, 2016 AVEO Pharmaceuticals, Inc. ("AVEO") reported discontinuation of the FOCAL study, a Phase 2, global, randomized, double-blind, placebo controlled clinical study, evaluating ficlatuzumab, AVEO’s HGF inhibitory antibody, in combination with erlotinib (Tarceva), an epidermal growth factor receptor tyrosine kinase inhibitor (EGFR TKI) in first line, VeriStrat (BDX 004) Poor (VSP), EGFR-mutated NSCLC patients (Filing, 8-K, AVEO, SEP 9, 2016, View Source [SID:SID1234515089]). VeriStrat is a proprietary biomarker test developed by Biodesix to select patients for entry into the trial.

The FOCAL study was prospectively designed to confirm results from a retrospective exploratory analysis of a randomized Phase 2 clinical trial ("Study P06162") comparing the combination of ficlatuzumab and gefitinib (IRESSA), an EGFR TKI therapy, to gefitinib monotherapy in previously untreated Asian patients with advanced NSCLC who had tested positive for both EGFR mutation and VSP. Study P06162 demonstrated a statistically significant improvement in overall survival (OS) and progression free survival (PFS) for patients in the gefitinib plus ficlatuzumab arm that were classified as VSP.

After experiencing slower than expected enrollment, a blinded interim analysis of the FOCAL study was conducted and found that patients who were positive for both VSP and EGFR mutations, who were known to be selected for poor prognosis, experienced materially higher discontinuation rates than observed in both the general ITT population and retrospective exploratory subgroup population of Study P06162, which was the basis for the FOCAL study design. This observation significantly compromised the feasibility of the trial. The parties have mutually agreed to discontinue the study. The parties intend to further discuss the details for the discontinuation of the study and future development of ficlatuzumab.

Champions Oncology Beats Revenue Guidance for Quarter; Reaffirms Full Year Guidance of 43 – 60% Revenue Growth

On September 9, 2016 Champions Oncology, Inc. (Nasdaq: CSBR), engaged in the development of advanced technology solutions and services to personalize the development and use of oncology drugs, reported its financial results for the first quarter ended July 31, 2016 (Filing, Q1, Champions Oncology, 2016, SEP 9, 2016, View Source [SID:1234515018]).

First Quarter and Recent Business Highlights:
• First quarter revenue of $3.7 million; significantly above revenue guidance range of $3.25 – $3.5 million
• Reaffirms full year revenue guidance for FY2017 of $16 – $18 million
• Delivered third consecutive quarter of record TOS bookings
• Signed significant AML study with major pharmaceutical company
• Signed second co-clinical trial
• Enrolled first patient in co-clinical trial

Joel Ackerman, Champions Oncology CEO, stated, "we continued to build momentum achieving another quarter of strong revenue growth and delivering our third consecutive quarter of record TOS bookings, setting the stage for continued positive results heading into next quarter.. We have produced this revenue growth while controlling our expenses, putting us on a path to cash flow breakeven by the end of our fiscal year."

Financial Results

For the first quarter of 2017, revenue was $3.7 million, as compared to $2.8 million for the first quarter 2016, an increase of 30%. Total operating expense for the first quarter 2017 was $6.2 million as compared to $5.7 million for the first quarter 2016, an increase of 8.3%.

For the first quarter of 2017 and 2016, Champions reported a loss from operations of $2.5 million and $2.9 million, respectively. Excluding stock-based compensation of $1.1 million and $775,000 for the three months ended July 31, 2016 and 2015, Champions recognized a loss from operations of $1.4 million and $2.1 million, respectively.

Operating Results

Translational Oncology Solutions (TOS):

Exhibit 99.1

TOS revenue was $3.2 million and $2.3 million for the three months ended July 31, 2016 and 2015, respectively, an increase of $900,000 or 35.2%. The increase is due to increased bookings in prior quarters, both in the number and size of the studies, and the addition of new customers.

TOS cost of sales was $2.0 million and $1.6 million for the three months ended July 31, 2016 and 2015, respectively, an increase of $400,000, or 27.1%. For the three months ended July 31, 2016 and 2015, gross margin for TOS was 35.1% and 31.0%, respectively. The increase in TOS cost of sales was due to an increase in TOS studies. The improvement in gross margin was due to higher TOS revenue leveraged off the fixed cost component of the lab and effective management of the variable lab costs.

Personalized Oncology Solutions (POS):

POS revenue was $511,000 and $485,000 for the three months ended July 31, 2016 and 2015, respectively, an increase of $26,000 or 5.4%. The increase is primarily the result of growth in sequencing revenue of $115,000 offset by a decline in implant and drug panel revenue of $29,000 and $60,000, respectively.

POS cost of sales was $474,000 and $661,000 for the three months ended July 31, 2016 and 2015, respectively, a decrease of $187,000, or (28.3%). For the three months ended July 31, 2016 and 2015, gross margin for POS was 7.2% and negative (36.3%), respectively. The improvement is attributed to the increase in higher margin sequencing revenue and aggressively managing our lab costs.

Research and development expense was $1.2 million and $1.1 million for the three months ended July 31, 2016 and 2015, respectively, an increase of $100,000, or 10.1%. The increase is primarily due to an increase in salary expense. Sales and marketing expense for the three months ended July 31, 2016 and 2015 was $925,000 and $1 million, respectively, a decrease of $75,000, or (10.1%). The decrease is due to the consolidation of sales and marketing personnel resources of the POS and TOS division. General and administrative expense was $1.5 million and $1.3 million for the three months ended July 31, 2016 and 2015, respectively, an increase of $200,000, or 16.5%. The increase is due to an increase of $354,000 in stock compensation expense.

University of Pittsburgh Licenses Novel Microneedle Patch to Pittsburgh Company

On September 8, 2024 SkinJect, Inc., a Pittsburgh-based company, reported its completion of a license with the University of Pittsburgh to its novel, minimally invasive treatment for common forms of non-melanoma skin cancer, basal cell and squamous cell carcinoma (Press release, University of Pittsburgh, SEP 8, 2016, View Source [SID1234646915]). Two million new cases of basal cell cancer are reported each year in the United States, and more than half of all patients suffer a recurrence. The new product under development could dramatically change the way these skin cancers are treated.

The SkinJect patch is a thumb-sized array of dissolvable microneedles that will deliver a chemotherapeutic agent to kill an existing skin cancer. The SkinJect patch will be applied once a week, for 3 weeks, in the doctor’s office. The microneedles, less than a millimeter long, dissolve within 15 minutes of application.

McGowan Institute for Regenerative Medicine affiliated faculty member Louis D. Falo, MD, PhD, who chairs Pittsburgh’s Department of Dermatology, is a co-inventor of the microneedle patch, along with O. Burak Ozdoganlar, PhD, Professor in Mechanical Engineering and Materials Science and Engineering, Carnegie Mellon University. Both inventors serve as scientific advisors to the company as it develops the array.

"The SkinJect patch is a promising novel approach to combating cancer," said Dr. Falo. "Right now, a standard of care for basal cell carcinoma is Mohs micrographic surgery, which can be expensive, painful, and disfiguring. The SkinJect patch will offer a cost-effective way to treat an existing cancer and potentially prevent it from coming back again later in life."

President and CEO of SkinJect Jim Nolan said SkinJect was being watched closely by both the medical and investment communities. "This device has the potential to transform the fields of dermatology and oncology. Its commercial future is extremely promising," said Nolan.

"We’re excited to have concluded this deal with a company headquartered in Pittsburgh," said Marc Malandro, Founding Director of the University of Pittsburgh Innovation Institute, who also serves as Chairman of Pennsylvania Bio, the state’s life sciences trade organization. "A number of local biotech entrepreneurs – including Anthony Florence, who played a pivotal role in providing early seed money for this project – have supported SkinJect. The company’s success is an affirmation of our region’s growing biotech community."

The company plans to file an IND (Investigational New Drug) application, a request to the FDA to begin administering the device to humans, by late 4th quarter 2016 or early 1st quarter 2017.

Skin cancer is the most commonly occurring cancer in the United States. After the age of 65, 50% of the population will develop skin cancer with the majority diagnosed with basal cell carcinoma (BCC). BCC occurs typically on the face, head, or neck and is a highly disfiguring disease. Current therapies involve invasive surgical procedures that are time-consuming, associated with patient recovery/morbidity and are expensive. SkinJect is novel, single use, topical drug delivery patch that, like a ‘Band-Aid’, is applied to the affected skin of those diagnosed with skin cancer. It is based on a proprietary micro-needle array drug delivery platform that uniquely delivers a potent generic chemotherapeutic agent and modifier to kill existing skin cancer and induce a memory immune response to prevent cancer re-occurrence. This project has confirmed high physician and patient acceptance from initial customer feedback.