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 20, 2018 Xencor, Inc. (NASDAQ: XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of autoimmune diseases, asthma and allergic diseases and cancer, reported that Bassil Dahiyat, Ph.D., president and chief executive officer, will participate in a fireside chat at the 30th Annual Piper Jaffray Healthcare Conference on Tuesday, November 27, 2018 at 10:30 a.m. ET in New York, NY (Press release, Xencor, NOV 20, 2018, View Source [SID1234531483]).

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A live webcast of the presentation will be available under "Events & Presentations" in the Investors section of the Company’s website located at View Source A replay of the presentation will be posted on the Xencor website approximately one hour after the live event and will be available for 90 days following the presentation.

On November 20, 2018 CymaBay Therapeutics, Inc. (Nasdaq: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported that management will participate in three upcoming investor conferences: the Piper Jaffray 30th Annual Healthcare Conference, the Evercore ISI HealthCONx Conference, and the BMO Prescriptions for Success Conference (Press release, CymaBay Therapeutics, NOV 20, 2018, View Source [SID1234531499]).

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Piper Jaffray 30th Annual Healthcare Conference
Date: Tuesday, November 27
Time: 12:00pm Eastern Time
Location: Lotte New York Palace Hotel

Evercore ISI HealthCONx Conference
Date: Wednesday, November 28
Time: 9:05am Eastern Time
Location: Boston Harbor Hotel

BMO Prescriptions for Success Conference
Date: Wednesday, December 12
Time: 11:40am Eastern Time
Location: Mandarin Oriental NYC
To access a live webcast and subsequent archived recording of each presentation, please visit the "Events" section on the CymaBay website at View Source

On November 20, 2018 Danish dermatology specialists LEO Pharma and California rare disease pioneers PellePharm reported a strategic development and commercialization collaboration to address unmet medical needs across various skin diseases with no approved treatments, advancing innovation and access to potential therapies for patients with life-altering conditions, such as Gorlin Syndrome and High Frequency Basal Cell Carcinoma (BCC), two distinct and rare forms of skin cancer (Press release, Leo, NOV 20, 2018, View Source [SID1234576270]).

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"We are very excited about the partnership with PellePharm, who are pioneers in Gorlin Syndrome and experts in rare skin diseases. Supporting our ambitious 2025 strategy, it marks LEO Pharma’s entrance in rare skin diseases and it offers a unique opportunity to bring the first treatment forward to people suffering from a very severe skin disease for which there currently are no approved therapies," said Thorsten Thormann, vice president of research at LEO Pharma.

Under the terms of the agreement, LEO Pharma has initially committed $70 million comprised of equity financing and financial R&D support to fund the global Phase 3 trial for patidegib topical gel 2% for the prevention and treatment of Gorlin Syndrome, with LEO Pharma securing an option to acquire all shares in PellePharm. PellePharm and its stockholders could receive up to an additional $690 million including merger consideration, and regulatory and commercial milestone payments. In addition, PellePharm stockholders are eligible to receive a double-digit royalty after achieving certain commercial milestones.

"Our company is founded on the commitment to targeting rare dermatologic diseases at the source and bringing new groundbreaking treatments forward to patients as efficiently and effectively as possible. As a global leader in medical dermatology, LEO Pharma is a great fit as both a development and commercialization partner. This collaboration puts us on track to commence our pivotal Phase 3 Gorlin Syndrome trial in early 2019. Then after the potential merger, we look forward to working with LEO Pharma to address other rare skin diseases with unmet needs," said Sanuj Ravindran, president and chief executive officer of PellePharm.

The agreement establishes a joint development committee with PellePharm maintaining responsibility for global development and LEO Pharma supporting in an advising role. Both companies will jointly drive commercialization planning, and Anders Kronborg, chief financial officer of LEO Pharma, will join PellePharm’s board of directors.

"Gorlin Syndrome Group welcomes the news that PellePharm and LEO Pharma are collaborating in developing patidegib topical as a treatment for BCCs. Data from PellePharm’s Phase 2 study suggest patidegib is effective in treating BCCs, with minimal side effects. We hope that by working with LEO, these benefits can be confirmed in a Phase 3 study. An effective topical medication, which can prevent and/or treat BCCs, will avert the need for the painful, disruptive and disfiguring surgeries we currently undergo," said Sally Webster, chairperson and Matthew Helbert, trustee of Gorlin Syndrome Group U.K.

Currently, there are no FDA-approved therapies for Gorlin Syndrome, and the standard of care for this rare disease is surgery. Patients with this lifelong, severe disease can have as many as 30 surgeries per year beginning in their mid-teens.

PellePharm’s 2% topical formulation of patidegib aims to be the first approved therapy for the prevention of Gorlin Syndrome, also known as Basal Cell Carcinoma Nevus Syndrome (BCCNS), and has shown early promise in Phase 2 clinical trials treating patients with Gorlin Syndrome and Sporadic BCC in both the U.S. and the U.K.

"For more than 18 years, our organization has been dedicated to supporting, educating, and seeking much-needed treatments for people living with Gorlin Syndrome (BCCNS), and yet people with this devastating disease still have no FDA-approved therapies," said Jean Pickford, executive director of the BCCNS Alliance. "With the Phase 3 study of patidegib beginning recruitment in early 2019, we continue to be encouraged by PellePharm’s dedication to our community, now in partnership with LEO Pharma."

PellePharm was advised by Rothschild & Co. on this transaction.

About Patidegib

Patidegib topical gel has shown early promise in a Phase 2 clinical study for the mitigation of BCC tumors in Gorlin Syndrome by blocking the disease at its source within the hedgehog signaling pathway. The topical formulation of patidegib was developed to provide the efficacy previously demonstrated by oral patidegib in Phase 1 trials without the adverse systemic side effects. The gel formulation is stable at room temperature for at least two years, making it a viable potential therapy for ongoing, at-home management of Gorlin Syndrome and High Frequency BCC. PellePharm has received both Orphan Drug Designation and Breakthrough Therapy Designation for patidegib topical gel in Gorlin Syndrome from the FDA.

About Gorlin Syndrome

Gorlin Syndrome is a rare, genetic disease characterized by constitutional, heritable mutations in one allele of the tumor suppressor gene encoding PATCHED1 (PTCH1), which acts as the primary inhibitor of the hedgehog signaling pathway. This leads to the formation of multiple basal cell carcinomas, often on the face.

With no FDA-approved drugs available for Gorlin Syndrome, the standard of care is surgery. People with severe Gorlin Syndrome may have as many as 30 surgeries per year, which can be repetitive, scarring and disfiguring. Approximately 10,000 people in the United States, or one in 31,000, are believed to be affected by Gorlin Syndrome. Gorlin Syndrome is known by several names, including Gorlin-Goltz Syndrome, Basal Cell Nevus Syndrome (BCNS), and Nevoid Basal Cell Carcinoma Syndrome (NBCCS).

About High Frequency Basal Cell Carcinoma (BCC)

High Frequency BCC, like Gorlin Syndrome, is a rare disease which is characterized by the development of an abnormally high number of BCCs. Unlike patients with Gorlin Syndrome, patients with High Frequency BCC are not born with a germline PTCH1 mutation and do not suffer from the other systemic manifestations of Gorlin Syndrome. The standard of care for patients with High Frequency BCC is surgery.

On November 20, 2018 Allergan plc (NYSE: AGN), a leading global pharmaceutical company, reported that Chairman and CEO Brent Saunders will participate in a fireside chat at the 2018 Citi Global Healthcare Conference in New York City (Press release, Allergan, NOV 20, 2018, View Source [SID1234531484]). The presentation will begin at 11:00 a.m. Eastern Time on Thursday, December 6, 2018.

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The presentation will be webcast live and can be accessed on Allergan’s Investor Relations website at View Source;. The webcast can also be accessed through the following URL: https://www.veracast.com/webca…

An archived version will be available within approximately one hour of the live presentation and can be accessed at the same location for 90 days