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Epigenomics AG Reports Financial Results for Fiscal Year 2019 and Preliminary Unaudited Financial Results for the First Quarter of 2020

On April 29, 2020 Epigenomics AG (FSE: ECX, OTCQX: EPGNY, the "Company") reported financial results (according to IFRS) for the fiscal year 2019 (Press release, Epigenomics, APR 29, 2020, View Source [SID1234556714]). In addition, the Company announces preliminary unaudited financial results for the first quarter of 2020.

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At the end of November 2019, a microsimulation model developed by scientists at Harvard Medical School was published in the journal Cancer Medicine. The results show that patient adherence and screening intervals are decisive factors in a successful colorectal cancer (CRC) screening strategy. When these factors are incorporated into the evaluation of various CRC screening measures, blood tests can play a significant role in reducing the incidence and mortality of colorectal cancer.
On February 28, 2020, the Centers for Medicare and Medicaid Services (CMS) opened the National Coverage Determination (NCD) review of Epi proColon, a further significant milestone for Epigenomics AG on its way to U.S. reimbursement by CMS. With the NCD opening, CMS is required by legal statute to publish a decision on the reimbursement of Epi proColon within a maximum period of nine months, i.e. no later than November 28, 2020. A positive reimbursement decision would be a major breakthrough for the marketing of the blood test in the U.S. market.
To finance its ongoing operations, Epigenomics successfully completed a capital increase by way of a private placement on March 31, 2020. The new shares were fully subscribed by institutional investors from Germany and the U.S.A. The gross proceeds of the capital increase amount to approximately EUR 4.0 million and, together with cost reduction measures initiated in connection with the Covid-19 pandemic, will ensure that the Company will have sufficient liquid funds well into Q1 2021.
The US-American National Comprehensive Cancer Network (NCCN) announced on April 23, 2020 that it has included Epi proColon in its updated 2020 NCCN CRC guidelines. Inclusion in the guidelines underscores the potential of Epi proColon to significantly increase screening rates and thereby reach the over 30 million Americans who are at risk for colorectal cancer but do not participate in screening.
Greg Hamilton, CEO of Epigenomics AG: "We have made tremendous progress in the last six months towards Medicare reimbursement, the key prerequisite for full commercialization of Epi proColon. In November, a pivotal microsimulation model was published demonstrating the effectiveness of Epi proColon and in February, CMS initiated its national coverage determination process for the test. In addition, the NCCN CRC screening guidelines were updated to include Epi proColon in a manner consistent with our FDA indications for use as an option for patients who refuse other screening modalities. We believe this positive momentum will culminate in a positive coverage decision later this year."

Financial results 2019

Total revenue decreased to EUR 1.1 million (2018: EUR 1.5 million), mainly due to the decrease in licensing revenue from China. Product revenue increased to EUR 1.0 million from EUR 0.8 million in 2018.
Selling, general and administrative costs increased from EUR 8.7 million (2018) to EUR 8.9 million, mainly due to greater expenses for sales and marketing. Research and development costs rose from EUR 6.4 million (2018) to EUR 7.3 million, largely because of external costs that arose in connection with carrying out the post-approval study for Epi proColon and the HCC cross-section study in the U.S.A.
The decline in EBITDA (before share-based payment expenses) to EUR -13.3 million (2018: EUR -11.4 million) was mainly caused by temporary cost increases in connection with the clinical studies and non-cash expenses.
The net loss for the year increased to approximately EUR -17.0 million (2018: EUR -12.7 million) and was impacted by a one-off non-cash expense of EUR 2.5 million; the loss per share decreased slightly to EUR -0.46 (2018: EUR -0.47).
Cash consumption rose to EUR -13.5 million in fiscal year 2019 (2018: EUR -9.6 million) due to higher operating cash outflows. Furthermore, the prior-year figure was buoyed by a stronger effect from working capital that no longer applied in the reporting period.

Preliminary financial results Q1 2020

Based on preliminary unaudited figures, Epigenomics AG generated revenues of EUR 0.2 million in the first quarter of 2020 (Q1 2019: EUR 0.3 million).
EBITDA (before share-based payment expenses) improved slightly from EUR -3.0 million (Q1 2019) to EUR -2.6 million.
The net loss for the period also improved slightly from EUR -3.0 million (Q1 2019) to EUR -2.9 million.
Cash consumption decreased by EUR 1.0 million to EUR -3.3 million (Q1 2019: EUR -4.3 million).

Outlook 2020

Revenue

The Company expects revenue for the fiscal year 2020 within the range of EUR 1.0 million to EUR 2.0 million.
EBITDA / Cash consumption

Epigenomics’ EBITDA (before share-based payment expenses) is expected to range from EUR -10.5 million to EUR -12.5 million. Based on the Company’s business plan 2020, cash burn is expected to be in line with EBITDA guidance (before share-based payment expenses).

Further Information

The Annual Report 2019 is available on the Epigenomics website:
View Source." target="_blank" title="View Source." rel="nofollow">View Source The Interim Statement for the first quarter 2020 will be published on May 7, 2020 on the Company’s website.

Conference call for analysts and investors

Epigenomics AG will host a conference call for analysts and investors today at 3.00 pm (CET) / 9.00 am (EDT). The webcast can be accessed on the Company’s website: View Source

Incurix introduces ‘c-myc inhibitor anti-cancer drug’ technology from the National Cancer Center

On April 28, 2020 Incurix and the National Cancer Center (NCC), reported their entry into an exclusive license agreement for the new c-myc inhibitor program that the NCC and the Korea Research Institute of Chemical Technology (KRICT) jointly developed (Press release, Incurix, APR 28, 2020, View Source;idx=68&page=1&code=news [SID1234643568]). Under the agreement, Incurix will receive the rights to develop, manufacture and exclusively commercialize myc inhibitors in consideration of upfront, potential development/regulatory milestones and royalties on net sales of the license program of Myc inhibitor.

As the transcription factor engaged in the growth and death of cells through interactions with DNAs, the c-myc protein is involved in the occurrence and growth of cancer and is over-expressed in various cancers. Drugs that have already been developed to control c-myc expression have failed in clinical trials because of their low selectivity over target substances and the issues of side effects caused by toxicity. For this reason, there are no c-myc inhibitor drugs that have been commercialized yet. The program that Incurix has in-licensed takes a differentiated approach by having the c-myc protein directly target the DNA interacting site to increase the selectivity against the target and minimize side effects. "We will ensure the success of commercializing the FIC transcription factor-targeting drug, which many global pharmaceutical companies could not yet succeed in," said Dr. Kyung-Chae Jeong, Incurix CEO and Senior Director at the NCC Research Center, who led the study. "If this technology succeeds in the commercialization of a drug that has so far been known as difficult in clinical applications, this will mark a significant milestone to cancer treatment," National Cancer Center Director Lee Eun-sook said. She added, "This licensing agreement is the result of the NCC’s continuous research, and we will continue our efforts for the success in commercialization through a good research cycle of research to commercialization."

Incurix is a case of the NCC’s researcher-led startup and was established in 2018 by the support of bio healthcare company builder New Flight, which leads the commercialization of bio-medical fields.

PHARMAXIS CANCER DRUG READY TO COMMENCE MYELOFIBROSIS PHASE 2 STUDIES Q4 2020

On April 28, 2020 Pharmaceutical research company Pharmaxis Ltd (ASX: PXS) reported that following positive results from phase 1b and long term toxicity studies, the company is now progressing to a phase 2 study of its oral anti‐fibrotic pan‐Lysyl Oxidase (LOX) inhibitor PXS‐5505 for treatment of the rare bone cancer, myelofibrosis (MF) (Press release, Pharmaxis, APR 28, 2020, View Source [SID1234562006]).

Pharmaxis has received pre IND feedback from the FDA on the PXS‐5505 program in MF and discussed the trial protocol with key opinion leaders in the US, Europe and Australia. Pharmaxis is currently preparing a full IND application for FDA submission mid‐year and appointing a Clinical Contract Research Organisation with a view to commencing recruitment in Q4 2020. The results of the phase 1b study of PXS‐5505 follow a successful phase 1a study reported in October 2019. The phase 1b was a double‐blind placebo controlled study in 16 healthy subjects divided into two groups with each group receiving a different dose or placebo daily for 14 days. The drug was well tolerated and no safety signals were identified during the study. Importantly for potential clinical benefit and in line with the phase 1a results, the data showed a drug with good pharmacokinetics and a dose related strong inhibition of members of the lysyl oxidase family in tissue and blood. PXS‐5505 is an oral drug that inhibits all lysyl oxidase family members (LOX, LOXL1, 2, 3 & 4). The compound successfully cleared pre‐clinical safety including 6‐month toxicity studies and has shown significant reductions in fibrosis in in‐vivo models of kidney, lung, heart, skin and liver fibrosis in addition to myelofibrosis and pancreatic cancer metastases. A recent publication1 reported that two Pharmaxis pan‐LOX inhibitor compounds have significantly decreased the bone marrow fibrotic burden in two different models of primary myelofibrosis. Myelofibrosis is a cancer with a poor prognosis and limited therapeutic options where only allogeneic stem cell transplantation is curative in a small number of patients who are eligible for such a treatment, while administration of a JAK1/2 inhibitor (e.g. ruxolitinib) provides mainly symptomatic relief but carries a risk of worsening blood cell counts. Pharmaxis CEO Gary Phillips said, "With the successful completion of the phase 1b study, 6‐month toxicity studies, support from clinical key opinion leaders and preliminary regulatory feedback, we can now move confidently into a 6‐month phase 2 study in myelofibrosis with meaningful clinical efficacy and safety endpoints. Pharmaxis believes that the current treatments for MF can be augmented by use of a pan‐LOX inhibitor and be disease modifying in a market that is conservatively worth US$1b per annum. We have ongoing discussions with contract research organisations who are confident of a trial recruitment start by the end of the year despite the impact of Covid‐19 on clinical trials worldwide. A number of contingency plans to maintain this timeline are actively being explored." Mr Phillips added, "The proprietary technology Pharmaxis has developed to measure activity and concentration of LOX and its related family members in tissue and blood enables us to clearly understand the role these enzymes have in fibrotic diseases and cancer and will significantly aid patient selection and proof‐of‐mechanism in the upcoming phase 2 trial. We are still gathering data from our academic collaborators who are investigating other cancers where fibrosis plays a significant role. These include pancreatic cancer, oral cancer, glioblastoma and mesothelioma where there is strong pre‐clinical evidence that several members of LOX family play a critical role." Pharmaxis will provide an update on phase 2 trial design once it has received final regulatory clearance in Q3 2020

Prokarium Signs an Exclusive Option Agreement with the Lausanne University Hospital (Centre Hospitalier Universitaire Vaudois – CHUV) for the Treatment of Non-Muscle Invasive Bladder Cancer (NMIBC)

On April 28, 2020 Prokarium, a private biotechnology company, focusing on genetically engineered bacteria for the development of microbial immunotherapy and vaccines, reported it has signed an exclusive, worldwide Option Agreement with the Lausanne University Hospital – CHUV, a Switzerland-based research hospital, to acquire a license to cover the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC) patients with intravesical instillations of Salmonella (Press release, Prokarium, APR 28, 2020, View Source [SID1234561273]).

Prokarium is investigating the use of engineered bacteria for the development of microbial immunotherapy for solid tumours. Their first oncology indication in preclinical development is NMIBC, which accounts for 400,000 new cases yearly worldwide and is currently lacking innovative treatments. Prokarium aims to disrupt the market with their engineered Salmonella that acts by boosting the natural anti-tumour immune response as well as through direct tumour killing.

"The standard of care of NMIBC is surgical removal of the tumour followed by up to 27 intravesical instillations of Bacillus Calmette–Guérin (BCG), which, despite the initial high response rate, has a 40-80% recurrence rate within 5 years" said Ted Fjallman, Ph.D, Chief Executive Officer, Prokarium. "Additionally, there is a significant worldwide shortage of BCG and many oncology patients are not able to receive their treatments."

Prokarium started a collaboration with a team at the Department of Urology of CHUV, led by Dr. Denise Nardelli-Haefliger, in 2019. The group, with Prof. Patrice Jichlinski and Dr. Ilaria Lucca, is running a Phase I trial in NMIBC patients investigating the intravesical administration of Salmonella Typhi Ty21a. Prokarium is working with this medical team to generate an adequate preclinical data package to file an IND.

"We are happy that thanks to the partnership with Prokarium, the results of our long-lasting immunotherapeutic research will soon be developed to benefit NMIBC patients" said the inventors Sonia Domingos-Pereira, Patrice Jichlinski and Denise Nardelli-Haefliger.

CRISPR Therapeutics Provides Business Update and Reports First Quarter 2020 Financial Results

On April 28, 2020 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the first quarter ended March 31, 2020 (Press release, CRISPR Therapeutics, APR 28, 2020, View Source [SID1234556776]).

"We made substantial progress in the last quarter despite the challenges posed by COVID-19. We are now progressing five cell therapy clinical trials in parallel targeting hemoglobinopathies and various cancers," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "Our CTX001 and CTX110 programs continue to enroll patients, and we expect to report data for these programs this year. We are pleased to have begun treating patients in our CTX120 trial for the treatment of relapsed or refractory multiple myeloma and we expect to begin treating patients in our CTX130 trial in renal cell carcinoma and T-cell and B-cell hematologic malignancies in the second half of this year. Despite these unprecedented times, we continue to execute on our programs and we remain focused on our commitment to patients and their families."

Recent Highlights and Outlook

Beta Thalassemia and Sickle Cell Disease

CRISPR Therapeutics and its partner Vertex remain on track to provide additional data from the two ongoing Phase 1/2 studies of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with transfusion-dependent beta thalassemia and in patients with severe sickle cell disease in 2020. New data expected in 2020 include initial data from additional patients dosed in each of the Phase 1/2 studies and longer duration follow-up data for the first patients dosed in each study. Screening, enrollment and mobilization in these studies is ongoing, however no additional patients are scheduled to initiate conditioning or dosing at this time due to COVID-19.

Immuno-Oncology

Patient enrollment continues in a clinical trial to assess the safety and efficacy of CTX110, CRISPR Therapeutics’ wholly-owned allogeneic CAR-T cell therapy targeting refractory CD19+ B-cell malignancies. The multi-center, open label clinical trial is designed to enroll up to 95 patients and investigate several dose levels of CTX110. If successful, CTX110 could enable off-the-shelf use of cell therapies and greatly expand their applicability and accessibility in treating patients with these hematologic malignancies. The Company expects to report top-line data for CTX110 at the end of 2020.

CRISPR Therapeutics has begun treating patients in a clinical trial to assess the safety and efficacy of CTX120, its wholly-owned allogeneic CAR-T cell therapy targeting BCMA for the treatment of relapsed or refractory multiple myeloma. The multi-center, open label trial is designed to enroll up to 80 patients and investigate several dose levels of CTX120.

Earlier this year, the U.S. Food and Drug Administration (FDA) accepted CRISPR Therapeutics’ Investigational New Drug (IND) application for CTX130, its wholly-owned allogeneic CAR-T cell therapy targeting CD70 for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. Additionally, CRISPR Therapeutics has obtained approval from Health Canada for its Clinical Trial Application (CTA). The Company expects to begin treating patients with CTX130 in the second half of this year.

Other Corporate Matters

Under the June 2019 collaboration agreement with Vertex to discover and develop gene editing therapies for the treatment of Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1), CRISPR Therapeutics received a payment of $25 million from Vertex related to the achievement of a research milestone in the DM1 program. CRISPR Therapeutics is eligible to receive additional milestone payments from Vertex of up to $800 million for these two programs.

CRISPR Therapeutics is scheduled to present two posters during the virtual American Society of Gene & Cell Therapy 2020 Annual Meeting, to be held from May 12 to 15, 2020, via View Source, as follows:

Title: Dual Guide CRISPR/Cas9 Editing of the CCR5 Gene Provides Complete Protection Against HIV in Humanized Mouse Models (abstract #1046)
Session Title: Gene Targeting and Gene Correction
Date and Time: Thursday, May 14, 2020; 5:30 PM – 6:30 PM

Title: Multiplexing of Up to 10 Gene Edits Using CRISPR/Cas9 to Generate CAR-T Cells with Improved Function (abstract #1151)
Session Title: Cancer – Immunotherapy, Cancer Vaccines
Date and Time: Thursday, May 14, 2020; 5:30 PM – 6:30 PM

In February, CRISPR Therapeutics announced its proposal to elect Doug Treco, Ph.D. to its Board of Directors at the Company’s upcoming annual general meeting to be held later this year.
First Quarter 2020 Financial Results

Cash Position: Cash and cash equivalents as of March 31, 2020, were $889.7 million, compared to $943.8 million as of December 31, 2019, a decrease of $54.1 million. The decrease in cash was primarily driven by cash used in operating activities of $52.2 million to support spending on the Company’s clinical and pre-clinical programs, as well as payroll and payroll-related expenses to support growth. In April 2020, the Company received a milestone payment of $25 million from Vertex under the collaboration agreement for DMD and DM1, resulting in pro forma cash exceeding $900 million.

Revenue: Total collaboration revenue was $0.2 million for the first quarter of 2020 compared to $0.3 million for first quarter of 2019. Collaboration revenue primarily consisted of charges to partners for research activities.

R&D Expenses: R&D expenses were $54.2 million for the first quarter of 2020 compared to $33.8 million for the first quarter of 2019. The increase in expenses was driven by increased headcount and development activities supporting the advancement of the hemoglobinopathies program and wholly-owned immuno-oncology programs.

G&A Expenses: General and administrative expenses were $19.6 million for the first quarter of 2020 compared to $14.9 million for the first quarter of 2019. The increase in general and administrative expenses for the year was driven by headcount-related expense and higher facilities cost.

Net Loss: Net loss was $69.7 million for the first quarter of 2020 compared to net loss of $48.4 million for the first quarter of 2019.
About CTX001
CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

About CTX110
CTX110 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 19, or CD19, for the treatment of CD19+ malignancies. A wholly-owned asset of CRISPR Therapeutics, CTX110 is being investigated in a clinical trial designed to assess the safety and efficacy of CTX110 for the treatment of relapsed or refractory B-cell malignancies. The multi-center, open-label clinical trial is designed to enroll up to 95 patients and investigate several dose levels of CTX110.

About CTX120
CTX120 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting B-cell maturation antigen, or BCMA. A wholly-owned asset of CRISPR Therapeutics, CTX120 is being investigated in a clinical trial designed to assess the safety and efficacy of CTX120 for the treatment of relapsed or refractory multiple myeloma. The multi-center, open-label clinical trial is designed to enroll up to 80 patients and investigate several dose levels of CTX120.

About CTX130
CTX130 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. A wholly-owned asset of CRISPR Therapeutics, CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies.