On November 21, 2018 C4X Discovery Holdings plc (AIM: C4XD), a pioneering drug discovery company,reported its full year results for the year ended 31 July 2018 (Press release, C4X Discovery, NOV 21, 2018, View Source [SID1234533246]).

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Financial highlights
Revenue increased to £7,064,000 (2017: £143,000), driven entirely by the Indivior licensing agreement.
R&D expenses increased 15% to £6,992,000 (2017: £6,100,000), reflecting the Company’s investment in drug discovery activity and its continued development of lead drug candidates.
Total loss after tax was £1,135,000 or 2.44 pence per share (2017: £6,782,000 or 16.88 pence per share).
Post period end, C4XD completed a £10.1 million fundraise (before expenses) in October 2018 through the placing and open offer of 11,210,674 new ordinary shares in the capital of the Company (Ordinary Shares) at a price of 90 pence (GBX) per Ordinary Share.
Operational highlights
Business model validated

Licensing deal with Indivior in March 2018 for our Orexin-1 antagonist programme; $10 million upfront with up to $284 million in milestone payments.
Discovery Engine progress

C4XD’s proprietary drug asset portfolio has grown from three programmes in 2014 to nine active programmes spread across multiple therapeutic areas.
Disease areas of focus are inflammation, neurodegeneration, immune-oncology/oncology and additional opportunistic areas (e.g., addiction and diabetes).
To date, the Taxonomy3 platform has identified 285 novel, genetically-validated and disease-linked genes that will generate target insights for future discovery programmes in inflammation and neurodegeneration.
Launched Stage 1 of virtual reality-based molecular visualisation tool, 4Sight with the aim of increasing the throughput of the Company’s pre-clinical portfolio by accelerating hit generation and lead optimisation timeframes.
Partnerships

Continued to enhance core, state-of-the-art target identification and drug design capabilities through synergistic strategic partnerships:
o Joint research collaboration with e-Therapeutics plc (AIM:ETX) to identify novel mechanistic insights in Parkinson’s Disease was announced in May 2018.
Senior appointments

Eva-Lotta Allan, Non-Executive Chairman, and Natalie Walter, Non-Executive Director, were appointed to the Board of Directors in July 2018.
Dr Clive Dix, CEO of C4X Discovery, said: "C4X Discovery has had a transformational year in 2018, underlined by the successful completion of our pre-clinical licensing deal with Indivior in March. This pivotal milestone validated our business model. Our pipeline continues to progress, with NRF-2 entering a formal partnering process and excellent in vivo data on IL-17 moving this programme towards partnering. This maturing pipeline, combined with our enhanced commercial capabilities and our innovative collaborations with multiple partners, positions us well to carry out our strategy of becoming the world’s most productive drug discovery engine and delivering returns to our shareholders."

A copy of the final results presentation given by Clive Dix (Chief Executive Officer) will be released later this morning on the Group’s website at www.c4xdiscovery.com.

Analyst conference call today

Dr Clive Dix, Chief Executive Officer, will present the results at 11:30am GMT on 21 November 2018 during a live conference call. A copy of the results presentation will be released later this morning on the Company website at www.c4xdiscovery.com.

On November 21, 2018 MorphoSys AG (FSE: MOR; Prime Standard Segment, MDAX & TecDAX; NASDAQ: MOR) reported that it will present at the following conferences (Press release, MorphoSys, NOV 21, 2018, View Source [SID1234531541]):

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American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2018
Date: December 1 – 4, 2018
Venue: San Diego, CA, U.S.
Participants: Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG
Dr. Malte Peters, Chief Development Officer of MorphoSys AG
Dr. Markus Enzelberger, Chief Scientific Officer of MorphoSys AG

Investor & Analyst Event
Date: December 5, 2018
Venue: New York, NY, U.S.
Participants: Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG
Dr. Malte Peters, Chief Development Officer of MorphoSys AG
Jens Holstein, Chief Financial Officer of MorphoSys AG

Berenberg European Conference 2018
Date: December 6, 2018
Venue: Pennyhill Park, UK
Participants: Jens Holstein, Chief Financial Officer of MorphoSys AG

JPMorgan Annual Healthcare Conference 2019
Date: January 9, 2019, 9:00 am PST (6:00 pm CET, 5:00 pm GMT)
Venue: San Francisco, CA, U.S.
Participants: Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG
Jens Holstein, Chief Financial Officer of MorphoSys AG
Dr. Malte Peters, Chief Development Officer of MorphoSys AG
Dr. Markus Enzelberger, Chief Scientific Officer of MorphoSys AG

Commerzbank German Investment Seminar 2019
Date: January 14, 2019
Venue: New York, NY, U.S.
Participants: Jens Holstein, Chief Financial Officer of MorphoSys AG

Kepler Chevreux German Corporate Conference
Date: January 21, 2019
Venue: Frankfurt, Germany
Participants: Jens Holstein, Chief Financial Officer of MorphoSys AG

PDF versions of the presentations will be provided at www.morphosys.com. The link to the webcasts, if available, will be filed under www.morphosys.com/conference-calls.

On November 21, 2018 Milestone Pharmaceuticals USA, Inc. reported that Joseph G. Oliveto, President and Chief Executive Officer, will present at the 30th Annual Piper Jaffray Healthcare Conference in New York on Tuesday, November 27, 2018 at 9:50 a.m. Eastern Time (Press release, Milestone Pharmaceuticals, NOV 21, 2018, View Source [SID1234531558]).

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On November 21, 2018 Lytix Biopharma AS reported its positive top-line data from a Phase 1 study of its first in class oncolytic peptide, LTX-315, administered as monotherapy or in combination with either ipilimumab or pembrolizumab in patients with transdermally accessible tumors (Press release, Lytix Biopharma, NOV 21, 2018, View Source [SID1234531542]).

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The primary objective of the trial was to assess the safety and tolerability of multiple, intratumoral doses of LTX-315 as a monotherapy or in combination. The study comprised four arms (A, B, C and D), with Arms A and B being monotherapy, Arm C recruiting patients with melanoma receiving combination therapy of LTX-315 with intravenous ipilimumab, and Arm D recruiting patients with triple negative breast cancer receiving LTX-315 in combination with intravenous pembrolizumab. The secondary objectives were to evaluate clinical activity and the ability of LTX-315 to evoke immune responses.

The study demonstrated that LTX-315 is safe to administer with manageable toxicities and with no added safety concerns when given in combination, either with ipilimumab or pembrolizumab. One patient in Arm C and two patients in Arm D achieved partial responses (PR). In the monotherapy arms, where paired biopsy samples were available, the study showed that LTX-315 can induce an increase in CD3+ and CD8+ tumour infiltrating lymphocytes (TILS), turn ‘cold’ tumors ‘hot’, and induce T-cell clonal expansion in peripheral blood as well as in tumor tissue.

"Successful completion of the study is an important milestone in the development of LTX-315", said Edwin Klumper, CEO Lytix Biopharma AS. "Demonstration of safety, and the confirmation of the scientific proof of concept with regards to LTX-315’s ability to invoke immune responses was critical to support further development of the compound. The company will now be pursuing the planned strategy of developing LTX-315 as a technology utilised in adoptive transfer of tumor specific T-cells and as a therapeutic in a combination regimen in a number of indications."

"We were encouraged by the experience of working with this novel treatment modality and by the evidence of its safety and early signs of efficacy that this trial demonstrated", said Professor James Spicer, Principal Investigator and Professor of Experimental Cancer Medicine at King’s College Hospital, London.

"This is the first oncolytic peptide that has entered clinical trials. The early indications of efficacy are promising. Intratumoral use of immunotherapies is currently seen as an innovative way to develop synergistic and safe combinations. LTX-315 has the potential to become a simple and universal means to use the tumor as a vaccine and prime the immune response against any cancer type", said Dr Aurelien Marabelle, Study investigator, Clinical Director, Cancer Immunotherapy Program, Gustave Roussy, France and lead author of HIT-IT (Starting the fight in the tumor: Expert recommendations for the development of human intratumoral immunotherapy (HIT-IT))

About the Phase 1 Trial:
The Phase 1 study was a multicentre study, evaluating LTX-315 in a dose ascending manner to assess safety of monotherapy in different dosing regimens (Arms A and B) and selection of a relevant clinical dose for combination with ipilimumab (Arm C) and pembrolizumab (Arm D). The monotherapy arms recruited all advanced cancer types, Arm C recruited patients with advanced melanoma who had prior exposure to anti PD-1 antibody, and Arm D recruited patients with advanced triple negative breast cancer.

In total 65 patients were recruited into this Phase 1 study. Of these, 4 patients in the early monotherapy arms experienced allergic reactions but these were manageable and reversable. Preventative measures were implemented to rectify this by optimising a prophylactic regimen and amending the dosing schedule. Subsequently, no further major allergic reactions were seen. Treatment emergent adverse events were mainly related to injection administration – transient drops in blood pressure, injection site pain and redness, but all toxicities were managed and reversed. Further, there was no evidence that the combination of LTX-315 with either ipilimumab or pembrolizumab conferred additive toxicities, implying that LTX-315 can be used in combination with checkpoint inhibitors without safety concerns.

One patient in Arm C (12.5%) and 2 patients in Arm D (12.5%) achieved a PR. One patient in Arm D had a PR duration of 12 months.

Of those patients in the intent to treat group that provided paired tumour samples, 100% showed an increase in CD3+ lymphocytes and TILs, 80% of tumour biopsies were converted from ‘cold’ to ‘hot’, and 71% showed a statistically significant expansion of peripheral T-cell clones.

On November 21, 2018 OBI Pharma, Inc., a Taiwan biopharma company (TPEx: 4174), reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for OBI-888 for the treatment of Pancreatic Cancer. OBI-888 is a first in class monoclonal antibody cancer immunotherapy targeting Globo H, a glycolipid antigen (Press release, OBI Pharma, NOV 21, 2018, View Source [SID1234531559]).

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A Phase 1 study of OBI-888 has commenced enrollment at the University of Texas M.D. Anderson Cancer Center in patients with locally advanced or metastatic solid tumors, potentially including Pancreatic, Breast, Gastric, Esophageal, Colorectal and Lung Cancers.

Amy Huang, General Manager of OBI Pharma, noted, "The orphan drug designation for OBI-888 by the FDA is encouraging and is a significant step in the development of this novel monoclonal antibody drug candidate targeting Globo H. In addition to targeting other solid tumors, OBI-888 will be evaluated for the treatment of pancreatic cancer, a disease with very limited treatment options. OBI will continue our utmost efforts to develop innovative therapies for people living with cancer."

About Pancreatic Cancer

Pancreatic Cancer originates in the exocrine or endocrine pancreatic cells and is thought to be caused by poor diet, smoking, and genetic factors. Pancreatic Cancer is a deadly disease that currently affects 69,839 people in the US and has a survival rate of only 8.5% at five years. In addition, treatment options are limited to surgical resection for patients with early stages of the disease and these patients may only have a five-year survival rate of up to 34.3%. As Pancreatic Cancer is asymptomatic in early stages, a majority of patients are undiagnosed or misdiagnosed until advanced stages of the disease. Surgery is no longer effective at this stage of the disease, leaving a large population with limited treatment options.

About Orphan Drug Designation (ODD)

The orphan drug designation provides OBI Pharma with potential benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees, and tax credits for qualified clinical trials. The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the U.S.

About OBI-888

OBI-888 is a novel first-in-class monoclonal antibody, which selectively targets Globo H, an antigen expressed in up to 15 epithelial cancers. This Globo H targeting antibody has been shown to induce tumor-killing via antibody dependent cell-mediated cytotoxicity (ADCC), antibody-dependent cell-mediated phagocytosis (ADCP) and complement dependent cytotoxicity (CDC).

OBI-888 is also anti-immunosuppressive and anti-angiogenic. In pre-clinical xenograft animal models in multiple tumor types (pancreatic, colon, lung, and breast), OBI-888 has demonstrated tumor shrinkage at various doses. In pre-clinical single and repeated dose toxicology studies, OBI-888 was well-tolerated, and no adverse effect was found in all the doses tested.

OBI Pharma owns global rights to OBI-888.