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 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 VBL Therapeutics (Nasdaq: VBLT), a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class treatments for cancer, reported it will provide a corporate update, in a Fireside Chat format, at the Piper Jaffray 30th Annual Healthcare Conference, to be held at the Lotte New York Palace on November 27–29, 2017, in New York City (Press release, VBL Therapeutics, NOV 21, 2018, View Source [SID1234531540]).

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Presentation Details:
Date: Wednesday, November 28
Time: 3:30-3:55 PM Eastern Time
Location: SoHo Track – Hubbard 1, 5th floor
Webcast: View Source;tp_key=0f7f922eb8

On November 21, 2018 Celyad (Paris:CYAD) (Brussels:CYAD) (NASDAQ:CYAD) (Euronext Brussels and Paris, and Nasdaq: CYAD), a clinical-stage biopharmaceutical company focused on the development of CAR-T cell-based therapies, reported its update on key clinical and operational developments for the third quarter ended September 30, 2018 (Press release, Celyad, NOV 21, 2018, View Source [SID1234531539]).

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THIRD QUARTER 2018 HIGHLIGHTS

FDA permitted the IND application for CYAD-101 to go into effect
No toxicity observed in the initial patient treated in the THINK CyFlu cohort evaluating the safety and anti-tumor activity of CYAD-01 after standard preconditioning chemotherapy
Key additions to the Board of Directors and Senior Leadership Team
Cash position of €55.9 million as of September 30, 2018
Dr. Christian Homsy, CEO of Celyad, commented: "We continue to be pleased with the flow of data from our clinical programs for CYAD-01. The data to date for CYAD-01 add to a growing body of evidence showing that our cell therapy has encouraging clinical activity across several indications, including acute myeloid leukemia and metastatic colorectal cancer, and is well-tolerated. In addition, we are excited for our lead allogeneic candidate, CYAD-101, which leverages our understanding of NKG2D biology, to enter the clinic for the treatment of metastatic colorectal cancer by year-end."

THIRD QUARTER 2018 OPERATIONAL AND FINANCIAL REVIEW

Updates to Allogeneic CYAD-101 Program and CYAD-01 THINK CyFlu Cohort

In July 2018, the U.S. Food and Drug Administration (FDA) accepted the Investigational New Drug (IND) application for CYAD-101, the world’s first non-gene edited, allogeneic CAR-T clinical candidate, and permitted it to go into effect. CYAD-101 will initially be evaluated in the alloSHRINK trial. Enrollment in the trial is expected to begin by year-end 2018. In September, Celyad announced the successful injection of the first patient under the amended protocol of the THINK trial, referred to as THINK CyFlu, in patients with metastatic colorectal cancer.

Strengthening our Management Team

In August, the Company announced that Dr. Margo Roberts, former Chief Scientific Officer of Kite Pharma Inc., joined Celyad’s Board of Directors and scientific committee. During the quarter, the Company also announced the appointment of Filippo Petti as Chief Financial Officer and Carri Duncan, PhD, as Vice President Corporate Development & Communications.

Financial review

The Company ended the quarter with €55.9 million in cash, cash equivalents and short-term investments. Use of cash over the third quarter of 2018 amounted to €6.7 million, in line with expectations. The Company confirms its previous guidance that existing cash, cash equivalents and short-term investments should be sufficient to fund operating expenses and capital expenditure requirements, based on the current scope of activities, until mid-2020.

HIGHLIGHTS SUBSEQUENT TO QUARTER-END

In October, Celyad announced an exclusive agreement with Horizon Discovery Group plc (LSE: HZD), for the use of its shRNA technology to generate Celyad’s second non-gene-edited allogeneic platform. Details of the agreement can be found on our website.

In early November, Celyad presented updated clinical results for the CYAD-01 program in solid tumors as well as translational research data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 33rd Annual Meeting. Press release highlighting our advancing clinical and technological platforms can be found here.

Celyad is scheduled to present two abstracts detailing updated clinical results from the Phase 1 THINK dose-escalation trial and upcoming clinical trials for the CYAD-01 program at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego, December 1-4, 2018. Details can be found on our website here.

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.