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.

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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."

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]).

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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.

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.

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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.

On September 8, 2016 BioLineRx Ltd. (NASDAQ/TASE: BLRX) reported that the successful final results of BL-8040’s Phase 2a clinical trial in relapsed or refractory acute myeloid leukemia (r/r AML) were presented yesterday evening at the 4th Annual Meeting of the Society of Hematologic Oncology (SOHO), being held September 7-10, 2016, in Houston, Texas (Press release, BioLineRx, SEP 8, 2016, View Source;p=RssLanding&cat=news&id=2200457 [SID:1234514998]).

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BL-8040, BioLineRx’s lead oncology platform, is a short cyclic peptide that functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. The Phase 2a study assessed the efficacy of BL-8040, as a single agent and in combination with Cytarabine (Ara-C), for the treatment of r/r AML. The reported data set includes 45 patients. The majority of patients in the study were heavily pretreated, with 45% of patients being refractory to one or two remission induction treatments, 19% of patients having relapsed after a short first remission of less than 12 months, and 17% of patients having undergone two or more relapses. In addition, the treated patient population included patients that had relapsed post allogeneic stem-cell transplantation (17%), as well as secondary AML patients (24%), both conditions which represent difficult-to-treat populations with poor prognoses.

Results show that treatment with BL-8040 in combination with Ara-C was safe and well tolerated at all doses tested up to and including the highest dose level of 2.0 mg/kg. Response to treatment was associated with efficient CXCR4 inhibition, resulting in high mobilization of blasts and induction of their differentiation. The composite complete remission rate, including both complete remission (CR) and complete remission with incomplete blood count recovery (CRi), was 38% in subjects receiving up to two cycles of BL-8040 treatment at doses of 1 mg/kg and higher (n=39). In the 1.5 mg/kg dose selected for the expansion phase of the study (n=22), the composite complete remission rate was 41%. These response rates are superior to the historical response rate of approximately 20% reported for high-risk AML patients treated with Ara-C alone.

In addition, the ongoing follow-up of patients participating in the study’s expansion phase and responding to the combination treatment suggests long durability of the remissions achieved, with two-thirds of these patients still alive, based on a follow-up period to date of up to 12 months.

Philip Serlin, Chief Financial and Operating Officer of BioLineRx, commented, "The results from this study clearly confirm the anti-leukemic activity of BL-8040 and reinforce our interest in the AML space. The data demonstrate that sustained inhibition of the CXCR4-CXCL12 axis with BL-8040 is safe and well tolerated, and when given in combination with Ara-C, improves the response rate historically achieved with Ara-C alone. In addition, treatment with BL-8040 as a single agent rapidly and efficiently induces mobilization, differentiation and cell death of AML cells. This selective effect on chemotherapy-resistant cells may be translated into reduction of residual disease, thus pointing to incorporation of BL-8040 into earlier AML treatment lines."

"As previously reported, we are currently in the midst of a large, randomized, controlled, Phase 2b study in the AML consolidation treatment line. In light of the data seen in the Phase 2a study, we have been allocating additional resources to this Phase 2b study. In this regard, we have recently engaged an additional AML research group to participate in the study, and last month we met with regulatory authorities to discuss the development pathway towards registration for this treatment line. In addition, yesterday we announced a significant new cancer immunotherapy collaboration with Genentech, which includes, among other studies, a Phase 1b study in the AML maintenance treatment line. This new study emphasizes our commitment to further develop BL-8040 as a treatment for AML and validates the anti-leukemic effect of BL-8040 seen in the Phase 2a study," concluded Mr. Serlin.

About BL-8040

BL-8040 is a short peptide for the treatment of acute myeloid leukemia, solid tumors, and certain hematological indications. It functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells from the bone marrow, thereby sensitizing these cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing apoptosis. In addition, BL-8040 has also demonstrated robust stem-cell mobilization, including the mobilization of colony-forming cells, and T, B and NK cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

On September 5, 2016, Horizon Pharma Ireland Limited ("Horizon Ireland"), an indirect wholly-owned subsidiary of Horizon Pharma plc ("Horizon Pharma"), reported that it entered into a Fifth Amendment to Commercial Supply Agreement (the "Fifth Amendment") with Bio-Technology General (Israel) Ltd. ("BTG"), which amends that certain Commercial Supply Agreement, dated March 20, 2007, by and between Horizon Ireland and BTG, as amended (the "Agreement") (Press release, Horizon Pharma, SEP 8, 2016, View Source [SID:1234515008]).

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Pursuant to the Fifth Amendment, among other things, BTG rescinded its prior notice of termination that was sent to Crealta Pharmaceuticals LLC in December 2015 (and would have caused the Agreement to terminate on December 15, 2018), the term of the Agreement was extended until December 31, 2030 and renews automatically for successive three year periods unless earlier terminated by either party upon three years prior written notice, and Horizon Ireland agreed to purchase certain minimum annual order quantities. The Agreement, as amended, may be terminated prior to December 31, 2030 by either party in the event of a force majeure, liquidation, dissolution, bankruptcy or insolvency of the other party, uncured material breach by the other party or after January 1, 2024, upon three years prior written notice.

The foregoing description of the Fifth Amendment does not purport to be complete and is qualified in its entirety by reference to the complete text of the Fifth Amendment, which will be filed, with confidential terms redacted, with the Securities and Exchange Commission as an exhibit to Horizon Pharma’s Quarterly Report on Form 10-Q for the quarterly period ending September 30, 2016.