On August 31, 2018 Shire plc (LSE: SHP, NASDAQ: SHPG) reported that it has completed the sale of its Oncology franchise to Servier S.A.S. for $2.4 billion (Press release, Shire, AUG 31, 2018, View Source [SID1234529240]). The franchise includes the global rights to ONCASPAR and ex-US and ex-Taiwan rights to ONIVYDE, as well as Oncology pipeline assets. David Lee, who was previously the head of Shire’s Global Genetic Diseases and Oncology franchises, will continue with Servier as CEO of its new US commercial subsidiary, Servier Pharmaceuticals.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The closing of this transaction demonstrates the value embedded in our portfolio and our continued focus on executing against our strategic priorities," said Dr. Flemming Ornskov, Shire CEO. "I am confident that Servier will continue to bring these important therapies to patients worldwide. I would like to thank David Lee and all those transferring to Servier for their ongoing commitment to meeting the needs of the oncology community, and we wish them continued success."

The Oncology sale proceeds are expected to enable Shire to further reduce its leverage. Shire previously announced a leverage target of Non GAAP Net Debt to EBITDA of below 2.5x by the end of 2018. Shire will update its financial guidance, including the impact of the Oncology sale, as part of the Q3 earnings announcement later this year.

Shire first announced its plans to sell its Oncology franchise to Servier on April 16, 2018. This transaction constitutes a Class 2 transaction for the purposes of the U.K. Listing Rules and, as such, Shire shareholder approval was not required. The transaction was approved by the Board of Directors; the Board initiated the potential sale of the Oncology franchise in December 2017.

For further information please contact:

Investor Relations
Christoph Brackmann [email protected] +41 41 288 41 29
Sun Kim [email protected] +1 617 588 8175
Scott Burrows [email protected] +41 41 288 4195

Media
Katie Joyce [email protected] +1 781 482 2779
Annabel Cowper [email protected] +41 79 630 8619

On August 31, 2018 Rubius Therapeutics, Inc. (Nasdaq: RUBY), a biotechnology company pioneering the development of a new class of ready-to-use cellular therapies, reported second quarter 2018 financial results (Press release, Rubius Therapeutics, AUG 31, 2018, View Source [SID1234529444]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

As of June 30, 2018, Rubius had cash, cash equivalents and marketable securities of $181.7 million. This amount excludes $257.9 million of proceeds after deducting underwriting discounts and commissions from Rubius’ initial public offering (IPO), which closed in July 2018. Based upon its current operating plan, Rubius expects its existing capital resources will be sufficient to fund operations into 2021.

"We have made significant progress over the course of this year – the highlight of which was the closing of our IPO," said Pablo J. Cagnoni, M.D., chief executive officer of Rubius Therapeutics, Inc. "The IPO proceeds give Rubius the resources to execute against our vision of creating life-changing cellular medicines to treat patients with rare diseases, cancer and autoimmune diseases. We are on track to file our first Investigational New Drug application for RTX-134 during the first quarter of 2019, for the treatment of patients with phenylketonuria, a rare disease affecting approximately 16,500 people in the U.S."(1)

Recent Business Highlights

Initial Public Offering

· On July 23, 2018, Rubius closed its IPO, raising $257.9 million of proceeds after deducting underwriting discounts and commissions. The proceeds from this offering, together with existing cash, cash equivalents and marketable securities are expected to be used:

·to purchase, renovate, customize and operate its manufacturing facility;

· to advance RTX-134 through a Phase 1/2a clinical proof-of-concept trial;

· to advance and expand its RED PLATFORMä and its research and development pipeline, including early discovery efforts and IND-enabling studies, and to initiate additional proof-of-concept trials in rare diseases, cancer and autoimmune diseases; and for working capital and other general corporate purposes.

Manufacturing Facility

· On July 31, 2018, Rubius purchased an existing 135,000-square foot manufacturing facility in Smithfield, Rhode Island.

· The company plans to invest approximately $95.0 million through 2020, which includes the $8.0 million purchase price.

Strategic Hires and Internal Capabilities

·Continued to build a leading scientific team and attract experienced leadership to deliver against Rubius’ vision, including the hiring of Pablo J. Cagnoni, M.D., as chief executive officer.

· Strengthened internal capabilities in discovery, platform and therapeutic development and manufacturing.

Second Quarter Financial Results

Cash, cash equivalents and marketable securities totaled $181.7 million as of June 30, 2018, compared to $104.3 million as of December 31, 2017. The increase in cash reflects the net proceeds from Rubius’ Series C preferred stock financing during the first quarter of 2018, offset by its cash used in operations during the period. Research and development expenses were $12.0 million during the second quarter of 2018, compared with $4.8 million for the second quarter of 2017. The increase was attributable to costs incurred in preparation of Rubius’ planned Phase 1/2a clinical trial of RTX-134 in phenylketonuria, as well as an increase in headcount in its research and development function and an increase in external manufacturing and research costs to improve and expand its manufacturing capabilities, prepare for clinical scale production and expand its in vivo testing to support clinical candidate selection. General and administrative expenses were $16.3 million during the second quarter of 2018, compared to $4.2 million for the second quarter of 2017. The increase was primarily due to an increase in stock-based compensation and increases in personnel costs and professional fees as Rubius prepared to operate as a public company.

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.