On February 25, 2016 La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, reported fourth quarter and full year 2015 financial results and highlighted 2015 corporate progress (Filing, Q4/Annual, La Jolla Pharmaceutical, 2015, MAR 2, 2016, View Source [SID:1234509952]).

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2015 Corporate Progress

• The ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial, La Jolla’s multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial of LJPC-501, La Jolla’s proprietary formulation of angiotensin II, in catecholamine-resistant hypotension (CRH) was initiated in March 2015. The initiation of the ATHOS 3 trial followed the reaching of an agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA), in which the agreed-upon primary efficacy endpoint in ATHOS 3 is increase in blood pressure. ATHOS 3 is enrolling as planned, and results are expected by the end of 2016.

• A multicenter, open-label, dose-escalation Phase 1 clinical trial of LJPC-401, the Company’s novel formulation of hepcidin, in patients at risk for iron overload due to conditions such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome was initiated in October 2015. Interim results, reported in January 2016, suggested a dose-dependent reduction in serum iron following a single dose of LJPC-401. Additionally, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) designated LJPC-401 an orphan medicinal product for the treatment of beta thalassemia intermedia and major.

• La Jolla entered into exclusive worldwide license agreements with the Indiana University Research and Technology Corporation and the University of Alabama at Birmingham to acquire intellectual property rights covering LJPC-30Sa and LJPC-30Sb in May 2015. LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy.

• La Jolla completed a public offering of common stock in September 2015, whereby La Jolla received approximately $104.6 million, net of issuance costs. La Jolla finished 2015 with $126.5 million in cash and cash equivalents and believes this is sufficient to fund operations into 2018.

"2015 was a very exciting year for La Jolla, highlighted by the initiation and continued progress of our Phase 3 clinical trial of LJPC-501 and encouraging interim data from our recently initiated Phase 1 clinical trial of LJPC-401," said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. "We look forward to a productive 2016, with the continued advancement of our exciting product candidates and results from our LJPC-501 Phase 3 clinical trial expected by the end of the year."

Results of Operations

As of December 31, 2015, La Jolla had $126.5 million in cash and cash equivalents, compared to $48.6 million as of December 31, 2014. The increase in cash and cash equivalents was primarily due to cash provided by our common stock offering that was completed in September 2015, which was partially offset by net cash used for operating activities. Based on current operating plans and projections, La Jolla believes that its current cash and cash equivalents are sufficient to fund operations into 2018.

La Jolla’s net cash used for operating activities for the three and twelve months ended December 31, 2015 was $8.5 million and $25.2 million, respectively, compared to net cash used for operating activities of $5.4 million and $12.9 million, respectively, for the same periods in 2014. La Jolla’s net loss for the three and twelve months ended December 31, 2015 was $11.8 million and $41.9 million, or $0.69 per share and $2.68 per share, respectively, compared to a net loss of $6.8 million and $21.3 million, or $0.45 per share and $2.00 per share, respectively, for the same periods in 2014. During the three and twelve months ended December 31, 2015, La Jolla recognized contract revenue of approximately $0.4 million and $1.1 million, respectively, which was pursuant to a services agreement initiated in 2015 under which La Jolla provides research and development services to a related party. The net loss includes non-cash, share-based compensation expense of $2.8 million and $13.1 million for the three and twelve months ended December 31, 2015, respectively, compared to $2.5 million and $9.1 million of noncash, share-based compensation expense, respectively, for the same periods in 2014.

The increases in net cash used for operating activities and net loss in 2015 as compared to 2014 were primarily due to increased development costs associated with the Phase 3 clinical trial of LJPC-501 in catecholamine-resistant hypotension and the costs associated with the initiation of the Phase 1 clinical trial of LJPC-401 in iron overload. In addition, there were increases in personnel and related costs, which were mainly due to the hiring of additional personnel and increased facility costs to support the increased development activities.

On March 2, 2016 MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) reported financial results for the year ending December 31, 2015, as well as a financial and operational outlook on 2016.

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Financial Year 2015 results and key operational achievements 2015:

FY2015 revenues of EUR 106.2 million (2014: EUR 64.0 million) and EBIT of EUR 17.2 million (2014: EUR -5.9 million) exceed latest financial guidance.

2015 revenues and EBIT have been significantly impacted by a non-recurring effect attributable to the ending of the collaboration with Celgene.

Strong cash position of EUR 298.4 million at end of 2015 (EUR 352.8 million end of 2014).

Product pipeline further expanded in 2015 to 103 programs (89 partnered, 14 proprietary) up from 94 programs (84 partnered, 10 proprietary) at year-end 2014.

Main cancer programs MOR208 and MOR202 with encouraging phase 2 clinical data in hematological indications non-Hodgkin’s lymphoma (NHL) and chronic lymphocytic leukemia (CLL) (MOR208) and multiple myeloma (MOR202).

In EUR million* FY 2015 FY 2014 Q4 2015 Q4 2014

Group Revenues 106.2 64.0 12.3 17.0
Total Operating Expenses 93.7 70.1 30.1 19.0
Earnings Before Interest and Taxes (EBIT) 17.2 (5.9) (17.5) (2.2)
Consolidated Net Profit/(Loss) 14.9 (3.0) (13.3) (1.0)
Diluted Net Profit/(Loss) per Share 0.57 (0.12) (0.51) (0.04)
* Differences due to rounding

Operational and financial outlook 2016:

Partnered discovery: Phase 3 readouts and potential filings for approval for antibodies bimagrumab in sporadic inclusion body myositis (sIBM, Novartis) and guselkumab in psoriasis (Janssen) expected.

Proprietary development: Increase in R&D activities planned to advance existing clinical programs and to begin clinical development of MOR106 and MOR107 in 2016.

R&D budget for proprietary drug development expected to increase to EUR 76 to 83 million in 2016 (2015: EUR 56.6 million). The bulk of these expenses will flow into the clinical development of MorphoSys’s most advanced proprietary drug candidates.

Revenues 2016 expected in the amount of EUR 47 to 52 million. EBIT 2016 expected in a range from EUR -58 to -68 million.

Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG, stated: "The progress made in 2015 has contributed to MorphoSys now having a product pipeline that is broader and more mature than ever before. The first therapeutic antibodies are nearing market approval, bringing us closer to a product-based revenue stream that we expect will grow significantly in the years ahead. Meanwhile, our proprietary development portfolio is expanding and the two most advanced programs are approaching the decisive stage of clinical development. Across our entire pipeline, we see many programs which have the potential to transform the treatment of the diseases they address, to the benefit of all of our stakeholders, not least, the patients who they will help."

"MorphoSys’s portfolio is one of the broadest in the industry. This is supported by financial resources of close to EUR 300 million at the end of 2015 that allows us to operate from a position of strength. Our solid financial position enables us to increase R&D expenses to grow the Company’s value without losing sight of our prudent and efficient use of resources," commented Jens Holstein, Chief Financial Officer of MorphoSys AG.

Update on MorphoSys’s Proprietary Antibody Portfolio

At the end of 2015, MorphoSys’s proprietary portfolio comprised 14 (2014: 10) innovative development programs, of which 4 (2014: 3) are in clinical development.

MOR208 – An anti-CD19 antibody for the treatment of B cell malignancies.

At the 2015 ASCO (Free ASCO Whitepaper) and ASH (Free ASH Whitepaper) meetings, positive data from a phase 2a study with MOR208 in 92 patients with relapsed or refractory non-Hodgkin’s lymphoma (NHL) were presented.

At the 2015 ASH (Free ASH Whitepaper) meeting, investigators at The Ohio State University reported on an investigator-initiated trial (IIT) of a combination of MOR208 with lenalidomide in relapsed/refractory or treatment-naive CLL patients. This study has recently been amended to add MOR208 on top of ongoing therapy with ibrutinib in patients suffering a relapse.

In 2016, MorphoSys will start two clinical combination trials for MOR208 in DLBCL and one in CLL. One of the DLBCL trials is intended to transition into a pivotal study in 2017.

MOR202 – A HuCAL antibody against CD38 for treatment of multiple myeloma (MM).

Encouraging clinical data from the ongoing dose-escalation trial of MOR202 alone and/or in combination with lenalidomide or pomalidomide (IMiDs – immunomodulatory drugs) were presented at ASCO (Free ASCO Whitepaper) and ASH (Free ASH Whitepaper) 2015.

The 16 mg/kg MOR202 confirmation cohort is enrolling and the 16 mg/kg IMiDs combination cohorts will be initiated soon.
Patients receiving MOR202 plus pomalidomide have shown very encouraging responses, which have deepened considerably since first data was reported at ASH (Free ASH Whitepaper) in December 2015.

Updated clinical data will be presented at an upcoming medical conference.
MOR209/ES414 – An anti-PSMA/anti-CD3 bi-specific antibody targeting prostate cancer. The compound is part of a co-development and co-commercialization agreement between MorphoSys and Emergent BioSolutions.

After examination of the initial clinical data, it was decided to adjust the dosing regimen and administration of MOR209/ES414. Clinical development will continue in 2016 using an amended clinical development plan, with recruitment to start around mid-2016.
Under the terms of the updated collaboration agreement, MorphoSys’s cost sharing in the years 2016 to 2018 and future milestone payments payable to Emergent BioSolutions by MorphoSys were reduced.
MOR103 – A HuCAL antibody directed against GM-CSF, out-licensed to GlaxoSmithKline (GSK) in 2013, is being developed by GSK in inflammatory diseases.

In the third quarter of 2015, GSK announced the commencement of a phase 2b study with MOR103 (re-named GSK3196165) in rheumatoid arthritis.
GSK plans to initiate a phase 1b/2a study in osteoarthritis of the hand in 2016.
Progress within MorphoSys’s Partnered Pipeline

During 2015, the number of therapeutic antibodies in MorphoSys’s partner pipeline grew to a total of 89 (December 31, 2014: 84). Of those, 21 antibodies were in clinical development, 25 in preclinical development and 43 in the discovery phase at year-end.
Two new antibodies entered phase 1 clinical development in 2015.
Financial Review for the Fiscal Year 2015 (IFRS)

Group revenues for the full year 2015 increased by 66 % to EUR 106.2 million (2014: EUR 64.0 million). This increase is mainly due to a contribution of approximately EUR 59 million from the recognition of deferred revenues and a one-time payment attributable to the early termination of a collaboration with Celgene. The Proprietary Development segment achieved revenues of EUR 59.9 million (2014: EUR 15.0 million), resulting from the non-recurring effect attributable to the above event. Revenues in the Partnered Discovery segment of EUR 46.3 million included EUR 42.3 million in funded research and licensing fees (2014: EUR 43.6 million) and EUR 4.0 million in success-based payments (2014: EUR 5.4 million).

Total operating expenses for the full year 2015 increased by 34 % to EUR 93.7 million (2014: EUR 70.1 million). Total research and development expenses (R&D) increased by EUR 22.7 million to EUR 78.7 million in 2015 (2014: EUR 56.0 million) mainly due to higher costs for external laboratory services and personnel. R&D expenses for proprietary research and development amounted to EUR 56.6 million (2014: EUR 36.4 million). General and administrative expenses (G&A) amounted to EUR 15.1 million (2014: EUR 14.1 million). Personnel expenses from share-based payments are included in G&A expenses and R&D expenses. These expenses amounted to EUR 3.6 million in 2015 (2014: EUR 4.0 million). Other income and expenses amounted to EUR 4.7 million (2014: income of EUR 0.2 million).

Earnings before interest and taxes (EBIT) amounted to EUR 17.2 million (2014: EUR -5.9 million). The increase is driven by one-off effects in the context of the ending of the MOR202 collaboration with Celgene in the first quarter of 2015. Proprietary Development showed a segment EBIT of EUR 10.7 million (2014: EUR -18.4 million), while the Partnered Discovery segment reported an EBIT of EUR 20.4 million (2014: EUR 25.9 million).

Finance income for 2015 amounted to EUR 3.4 million (2014: EUR 1.6 million). The Group reported income tax expenses of EUR 5.7 million (2014: tax benefit of EUR 1.3 million). Due to the positive one-off effects realized in 2015, MorphoSys generated a consolidated net profit of EUR 14.9 million compared to a net loss of EUR -3.0 million in 2014. The diluted net result per share amounted to EUR 0.57 (2014: EUR -0.12).

On 31 December 2015, the Company had a cash position of EUR 298.4 million, compared to EUR 352.8 million on December 31, 2014. In the balance sheet this cash position is reported under the positions: cash and cash equivalents; available-for-sale financial assets; bonds, available-for-sale; financial assets classified as loans & receivables; and financial assets classified as loans & receivables, net of current portion.

Net cash outflow from operating activities in 2015 totaled EUR 23.5 million (2014: cash outflow EUR 14.2 million). Resulting from the exercise of 80,848 convertible bonds in 2015, the number of shares issued rose to 26,537,682 by December 31, 2015 (December 31, 2014: 26,456,834).

Fourth Quarter of 2015 (IFRS)

In the fourth quarter of 2015, the Company generated revenues in the amount of EUR 12.3 million, compared to EUR 17.0 million in the same quarter of 2014. Total operating expenses amounted to EUR 30.1 million in Q4, compared to EUR 19.0 million in the same quarter of 2014. The increase in operating expenses was mainly due to higher expenses for third party services. The EBIT amounted to EUR -17.5 million (Q4 2014: EUR -2.2 million). Group net loss for the fourth quarter 2015 was EUR -13.3 million, compared to a net loss of EUR -1.0 million in the fourth quarter of 2014.

Outlook for 2016

In line with its intention to advance and broaden its proprietary pipeline, MorphoSys will increase its spending on proprietary drug development in comparison to the previous year. The majority of the expenses will flow into the clinical development of MorphoSys’s most advanced drug candidates.

MOR208: MorphoSys plans to initiate two phase 2 trials, the first of which will combine MOR208 and lenalidomide in DLBCL, the second of which will combine MOR208 and idelalisib in ibrutinib-refractory CLL. In the second half of 2016, the Company will start a further study in DLBCL, combining MOR208 and bendamustine. This trial is intended to lead into a pivotal study in 2017.

MOR202: MorphoSys plans to generate additional clinical data from an ongoing phase 1/2a trial in multiple myeloma (MM) evaluating the confirmation cohort of 16 mg/kg MOR202 alone as well as the cohorts and confirmation cohorts of 16 mg/kg MOR202 in combination with lenalidomide or pomalidomide.

With MOR209, MorphoSys expects to continue the phase 1 trial in metastatic castration-resistant prostate cancer (mCRPC) using an amended dosing scheme.

MorphoSys expects to start clinical development of MOR106 (jointly developed with Galapagos) and of MOR107, the first drug candidate from the Company’s acquisition of Lanthio Pharma.

In the Partnered Discovery segment, MorphoSys expects two phase 3 readouts and potential filings for approval for the antibodies bimagrumab in sIBM (Novartis) and guselkumab in psoriasis (Janssen). This would mark the first approvals of HuCAL antibodies.
MorphoSys expects Group revenues for the 2016 financial year in the range of EUR 47 to 52 million. R&D expenses for proprietary drug development are expected to rise to EUR 76 to 83 million. The Company expects earnings before interest and taxes (EBIT) in a range of between EUR -58 to -68 million. This guidance does not include any potential in-licensing or co-development of further development candidates.

MorphoSys will hold its conference call and webcast today to present the Annual Financial Results 2015 and the Outlook 2016.

On March 2, 2016 OPKO Health, Inc. reported that its GeneDx subsidiary and business unit has been selected to give 11 platform talks and 18 poster presentations on a range of genetics-related topics at the 2016 American College of Medical Genetics and Genomics (ACMG) Annual Meeting (Press release, Opko Health, MAR 2, 2016, View Source [SID:1234509330]). These presentations support GeneDx’s position as an industry leader in the wider genomics industry.

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Marc Grodman MD, CEO of BioReference/GeneDX states, "We are honored to have such a large number of presentations accepted at this year’s ACMG meeting and believe the depth and scope of our contributions demonstrate not only our commitment in expanding the scope of our genomic testing but also a meaningful contribution to enrich the scientific community at large."

This year’s presentations cover a range of topics ranging from inherited cancer testing to whole exome sequencing. Some of GeneDx’s featured presentations at the meeting include:

GeneDx’s experience from the first 100 XomeDxXpress cases performed for patients in critical care including diagnostic yield and how the results impacted medical management;
The potential use of Whole Exome Sequencing for fetal anomalies and fetal demise;
The detection of mosaic variants in genes causing germline neurodevelopmental disorders, such as epilepsy;
Multiple talks describing genes that have recently been associated with neurodevelopmental delay, either as a new gene-disease association or as an expansion of a previously described clinical phenotype.

For a complete list of GeneDx’s platform presentations and posters, please visit the GeneDx website View Source or stop by and visit booth #413 at the 2016 ACMG conference to learn more.

Over the last several years, GeneDx has been striving to better understand the genetic causes of autism and intellectual disability through research and its robust Whole Exome Sequencing program, perhaps best demonstrated by several of the company’s presentations at this year’s ACMG annual meeting. Based on this increased understanding and knowledge, GeneDx is proud to announce the launch of its Autism/Intellectual Disability (ID) Xpanded Panel. This is a dynamic testing panel analyzing approximately 2,000 genes that have been associated with autism, intellectual disability and/or developmental delay.

Effective March 1, 2016 Håkan Wickholm has succeeded Unni Hjelmaas as CEO in Lytix Biopharma.
Mr. Wickholm’s appointment reflects the new phase in Lytix Biopharma’s development with increasing focus on Business Development (Press release, Lytix Biopharma, MAR 1, 2016, View Source [SID:1234509331]). Håkan Wickholm will continue as head of Business Development.

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Board Chair Gert W. Munthe says:

"The entire Board is delighted that Håkan Wickholm now takes on this role as the clinical program is starting to show substantial immune responses in patients treated with LTX-315. This lays the foundation for successful business development activities. At the same time the Board would like to express their thanks to Unni Hjelmaas for her hard work and dedication to Lytix Biopharma in her four years as CEO."

Håkan Wickholm says:

"I am very excited by this opportunity to take on the role of CEO at this stage, when important immune response data develops, allowing us to intensify business development activities and further clinical development. ’’

On March 1, 2018 FLAG Therapeutics Inc. reported that the U.S. Food and Drug Administration’s Office of Orphan Products Development and the European Medicines Agency (EMA) have both granted Orphan Drug Designation to FLAG-003 for the treatment of glioma (Press release, Flag Therapeutics, MAR 1, 2016, View Source [SID1234525570]). Gliomas (including Glioblastoma) are the most aggressive forms of brain cancer and carries a very poor prognosis for survival and is one of the deadliest forms of cancer. Two and 5-year survival rates are 27% and 10%, with the median progression-free survival (PFS) being only 6.9 months.

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Orphan status is granted by the FDA to promote the development of products that demonstrate promise for the treatment of rare diseases – those which affect fewer than 200,000 Americans annually. Orphan drug designation entitles FLAG Therapeutics to 7 years marketing exclusivity following product launch in the United States (10 years marketing exclusivity in the EU) and enables the company to apply for research funding, tax credits, a waiver from FDA (PDUFA) user fees, FDA assistance in clinical trial design, and access to the central authorization procedure within the European Union.

FLAG-003 for the treatment of Glioma

FLAG-003 is a small molecule which exerts both cytotoxic and cytostatic activity due to two distinct and well characterized mechanisms of action. It possesses cytotoxic anti-tubulin activity by binding to the colchicine site of tubulin causing microtubule depolymerization. It also possesses anti-angiogenic activity through binding and inhibition of RTK receptor tyrosine kinase (RTKs) activity. The anti-tubulin and anti-angiogenic activities of FLAG-003 have translated into potent antitumor and anti-vascular effects in vivo with significantly better inhibitory activity on GBM tumor growth and vascularization than the currently approved chemotherapy, temozolomide (TMZ).