On September 3, 2018 STORM Therapeutics, the drug discovery company focused on the discovery of small molecule therapies modulating RNA epigenetics, and The University of Cambridge reported that they have been granted a Knowledge Transfer Partnership (KTP) (Press release, STORM Therapeutics, SEP 3, 2018, View Source [SID1234561043]). The Grant, of up to £240,000, is to develop an analysis platform using the data warehouse software InterMine to help STORM advance its cancer research.

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STORM has established a pipeline of drug discovery programmes to develop novel, first-in-class drugs for the treatment of specific cancers and other diseases with high unmet medical need.

Keith Blundy, Chief Executive of STORM Therapeutics said: "Biology is an extremely fast paced, changing area. STORM is on the cutting edge of cancer research and keeping up with the data that is constantly being generated is vital. Previously, this data has been spread across a number of silos and gathering all of this information, as well as being able to analyse it efficiently, can be costly and prone to error. Through the KTP programme STORM is now partnered with an open-source data warehouse, InterMine, specifically for the integration and analysis of complex biological data, making it easy to query and analyse."

Gos Micklem, Principal Investigator for the InterMine project based at the Department of Genetics at the University of Cambridge said: "We are excited by this KTP award and the opportunity to collaborate with STORM. We see benefits on both sides – STORM gains our experience in large-scale data integration and the freely available open-source InterMine platform, while the adaptations that will be made to accommodate their data and interests will improve InterMine for the benefit of the broader community."

The KTP programme helps businesses in the UK by linking them with an academic organisation, enabling them to bring in new skills and the latest academic thinking to deliver a specific, strategic innovation project.

On August 31, 2018 Exact Sciences Corp. (Nasdaq: EXAS) reported that company management will be presenting at the following investor conferences during September and invited investors to participate by webcast (Press release, Exact Sciences, AUG 31, 2018, View Source [SID1234529313]).

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Baird 2018 Global Healthcare Conference, New York
Fireside chat on Wednesday, Sept. 5, at 12:15 p.m. ET
Morgan Stanley 16th Annual Global Healthcare Conference, New York
Fireside chat on Wednesday, Sept. 12, at 4:15 p.m. ET

The webcast can be accessed in the investor relations section of Exact Sciences’ website at www.exactsciences.com.

On August 31, 2018 Kiadis Pharma N.V. ("Kiadis Pharma" or the "Company") (Euronext Amsterdam and Brussels: KDS), a clinical-stage biopharmaceutical company, reported its unaudited interim Financial Results for the six months ended June 30, 2018, which have been prepared in accordance with IAS 34 as adopted by the European Union (Press release, Kiadis, AUG 31, 2018, View Source [SID1234529210]).

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Arthur Lahr, CEO of Kiadis Pharma, commented: "We have made tremendous progress in the last six months: ATIR101 is now very close to potential CHMP opinion in 2018, we are on track with our Phase 3 trial, and obtained further confirmatory data from our Phase 2 trials. To allow us to ramp up our Phase 3 trial and prepare for commercialization in the EU we also raised substantial equity and debt facilities that extended our cash runway into the third quarter of 2019, and, upon positive CHMP opinion, potentially into the first quarter of 2020. We have also significantly strengthened our organization in medical, operations, commercial and finance functions. Kiadis is in great shape and well positioned to deliver on the promise of ATIR101."

Operating highlights – ATIR101 (including post reporting period)

European marketing authorization application for ATIR101:
Responses to the Day 120 List of Questions submitted in March 2018;
Day 180 List of Issues received in May 2018 and responses submitted in August 2018;
On track to obtain CHMP opinion from the European Medicines Agency in the fourth quarter of 2018.
Phase 3 trial CR-AIR-009, comparing ATIR101 against the post-transplant cyclophosphamide (PTCy) or ‘Baltimore’ protocol:
Progress in line with internal plans: 14 clinical sites are currently open for recruitment, 16 patients have been enrolled;
Protocol amendment submitted to regulatory authorities: number of patients increased to 250 to further increase power [80% power to detect 16% Graft-versus-host-disease-free and Relapse-Free Survival (GRFS) difference]; interim analysis to occur after 2/3 of GRFS events to increase chance of positive read out, now expected in the second half of 2020; conditioning regimens harmonized between the two treatment arms to reduce heterogeneity.
Phase 2 trial CR-AIR-008 (‘008’): The last patient received a single dose of ATIR101 in January 2018.
Pooled analysis: Further analysis of 1-year Phase 2 pooled data [Intention-To-Treat (ITT), 37 patients] from studies CR-AIR-007 and single dose CR-AIR-008 shows GRFS 53% [95% confidence interval (CI), 39%-72%]; Overall Survival (OS) 58% (95% CI, 44%-77%), in line with Phase 2 CR-AIR-007 trial. For the PTCy/Baltimore protocol, single site data from Johns Hopkins (McCurdy et al. 2017) and Atlanta (Solh et al, 2016) show a disease-risk index (DRI) normalized 1-year GRFS value of 40% and 30%, respectively.
Operating highlights – Organization (including post reporting period)

Robbert van Heekeren resigned as Chief Financial Officer and as member of the Management Board.
Scott A. Holmes appointed as new Chief Financial Officer.
Organization strengthened across all functions, comprises 73 employees, up from 51 a year ago. Key new appointments include head of Medical US (former Iovance/ Dendreon), head of Medical EU (former Genzyme/ AstraZeneca), head of market access EU (former Genzyme/ Novo Nordisk), head of pharmacovigilance (former Astellas), head of facilities (former Merck/ Douwe Egberts).
Otto Schwarz, former Chief Operating Officer of Actelion and Mr. Subhanu Saxena, former Chief Executive Officer of Cipla and former member of the senior executive team of Novartis, were appointed as Supervisory Board members of the Company at the Annual General Meeting of shareholders in June 2018. Mr. Stuart Chapman resigned from the Supervisory Board following the shareholders’ meeting.
Financial highlights (including post reporting period)

In the first six months of 2018, the Company did not generate any revenues. Total operating expenses increased by EUR2.9 million from EUR8.2 million in the first six months of 2017 to EUR11.1 million in the same period of 2018. This increase was primarily caused by a further expansion of the workforce in all areas of the organization, the move to a larger building which includes a commercial manufacturing facility, laboratories and office space, and consultancy expenses for business development and market access.
In the first six months of 2018, net financial result came in at EUR3.0 million compared to EUR0.4 million for the same period of 2017. Higher finance costs were mainly the result of higher interest expenses on loans and borrowings, and a net foreign exchange loss in the first six months of 2018 compared to a net foreign exchange gain in 2017.
The net loss for the six months ended June 30, 2018 came at a level of EUR14.1 million compared to a loss of EUR8.5 million for the six months ended June 30, 2017. Operating expenses and net result for the first six months of 2018 were in line with management expectations.
The Company ended the first six months of 2018 with EUR41.7 million in cash and cash equivalents. In March 2018, the Company issued 2.6 million shares and raised EUR23.4 million in gross proceeds.
On July 31, 2018, the Company received a new debt facility from Kreos Capital V (UK) Ltd providing the Company with up to EUR20 million of additional financing.

On August 31, 2018 Selecta Biosciences, Inc. (Nasdaq: SELB), a clinical-stage biopharmaceutical company focused on unlocking the full potential of biologic therapies by mitigating unwanted immune responses, reported that Chief Scientific Officer Takashi Kei Kishimoto, Ph.D. will present on the poster entitled Ongoing Phase 2 Clinical Trial of SEL-212 Shows Sustained Reduction of Serum Uric Acid by Mitigating Formation of Anti-Uricase Antibodies at the 20thAsia Pacific League of Associations for Rheumatology Congress (APLAR), taking place 6-9 September 2018 at the Kaohsiung Exhibition Centre, Taiwan (Press release, Selecta Biosciences, AUG 31, 2018, View Source [SID1234529216]).

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On August 31, 2018 Genmab A/S (Nasdaq Copenhagen: GEN) reported that the European Commission (EC) has granted marketing authorization for DARZALEX (daratumumab) in combination with bortezomib, melphalan and prednisone (VMP), for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT) (Press release, Genmab, AUG 31, 2018, View Source [SID1234529235]). The EC approval follows a positive opinion issued for DARZALEX by the CHMP of the European Medicines Agency (EMA) in July 2018. In August 2012, Genmab granted Janssen Biotech, Inc. (Janssen) an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

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Genmab will receive a milestone payment of USD 13 million from Janssen in connection with the first commercial sales of DARZALEX under the expanded label. The sales are expected to occur quickly after the approval. This milestone payment was included in the financial guidance issued by Genmab originally on February 21, 2018 and then reiterated in subsequent quarterly financial reports, most recently on August 8, 2018, and as such there is no change to the company’s financial guidance following this approval.

"Approved in this indication in the U.S. since early May, DARZALEX in combination with bortezomib, melphalan and prednisone will now become an option for newly diagnosed multiple myeloma patients in Europe," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab. "We are very pleased that many more patients in need will have the opportunity for treatment with this regimen and we look forward to seeing this combination launched in Europe."

The positive opinion of the CHMP was based on data from the Phase III ALCYONE (MMY3007) study that showed a reduction of the risk of disease progression or death by 50 percent (Hazard Ratio [HR] = 0.50; 95 percent CI [0.38-0.65], p<0.0001) in patients with newly diagnosed multiple myeloma ineligible for ASCT when daratumumab is combined with VMP. The safety of DARZALEX combination therapy was consistent with the known safety profiles of DARZALEX monotherapy and of therapy with bortezomib, melphalan and prednisone, respectively. This data was presented as a Late-Breaking Abstract at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and simultaneously published in The New England Journal of Medicine in December, 2017.

About the ALCYONE study
This Phase III study (NCT02195479) is a randomized, open-label, multicenter study that included 706 newly diagnosed patients with multiple myeloma who are ineligible for ASCT. Patients were randomized to receive 9 cycles of either VMP [bortezomib (a proteasome inhibitor), melphalan (an alkylating chemotherapeutic agent) and prednisone (a corticosteroid)] combined with daratumumab, or VMP alone. In the daratumumab treatment arm, patients received 16 mg/kg of daratumumab once weekly for six weeks (cycle 1; 1 cycle = 42 days), once every three weeks from cycles 2 to 9, and once every 4 weeks from cycle 9 until disease progression. The primary endpoint of the study is progression free survival (PFS).

About multiple myeloma
Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Approximately 35,433 new patients were diagnosed with multiple myeloma and approximately 22,060 people died from the disease in Western Europe in 2016.3 Globally, it was estimated that 138,509 people were diagnosed and 98,437 died from the disease in 2016.3 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.4

About DARZALEX (daratumumab)
DARZALEX (daratumumab) injection for intravenous infusion is indicated in the United States in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.5 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX is indicated in Europe for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy. In Japan, DARZALEX is approved in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for treatment of adults with relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market. For more information, visit www.DARZALEX.com.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a person’s own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).5,6,7,8,9

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and frontline multiple myeloma settings and in amyloidosis. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases, such as NKT-cell lymphoma, myelodysplastic syndromes, B and T-ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA, for multiple myeloma, as both a monotherapy and in combination with other therapies.