On March 19, 2020 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics targeting infectious diseases and cancers based on its proprietary arenavirus platform, reported its financial results and corporate update for the fourth quarter and full year 2019 (Press release, Hookipa Pharma, MAR 19, 2020, View Source [SID1234555695]).

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"In 2019, HOOKIPA achieved its development progress and financial goals," commented Joern Aldag, HOOKIPA’s Chief Executive Officer. "We dosed our first patient in HB-201, our first immuno-oncology clinical trial in Human Papillomavirus-positive cancers, continued enrolling our Cytomegalovirus prophylaxis trial HB-101, progressed our collaboration with Gilead for HBV and HIV, and executed our Series D and IPO early in the year to fund our clinical trials beyond proof of concept. Well prepared for an important, data-rich year 2020, we now have to assume that the coronavirus pandemic will very likely affect how we will operate in order to fully execute our 2020 strategic plan in order to remain on track for our previously announced clinical milestones. Focusing first on our people and then on business continuity, we have instituted a work from home policy for those who do not need the company infrastructure for their work. We maintain our labs for business critical projects and stay in close connection with manufacturers and clinical trial sites. Our people are remarkable in doing everything under our control to deliver in the context of this situation."

R&D Pipeline Update and Clinical Progress

HB-101, lead product candidate in infectious diseases
HOOKIPA’s VaxWave-based prophylactic Cytomegalovirus (CMV) vaccine candidate, HB- 101, continues to enroll patients in a Phase 2 randomized, double-blinded clinical trial in CMV-negative patients awaiting kidney transplantation from CMV-positive donors. Based on HB-101’s tolerability profile in the target patient population dosed to date and to gain further insights that will inform the Phase 3 trial design, the Company added a new cohort of CMV-positive recipients awaiting kidney transplantation from CMV-positive or -negative donors to the trial protocol in early 2020. HOOKIPA expects to report safety and immunogenicity data in the first half of 2020 from approximately one-third of the total 150 patients to be enrolled, including placebo recipients. The immunogenicity data set will contain both CMV-specific antibody (gB) and CMV-specific CD8+ T cell responses. Preliminary efficacy data is on track to follow late in the second half of 2020.

HB-201 and HB-202, programs for the treatment of Human Papillomavirus-positive cancers
HB-201 and HB-202, HOOKIPA’s TheraT-based lead oncology product candidates, are in development for the treatment of Human Papillomavirus serotype 16-positive (HPV16+) cancers. HOOKIPA dosed the first patient in an open label, dose escalating Phase 1/2 clinical trial for HB-201 in December 2019, HOOKIPA’s first clinical trial in immuno-oncology. The Company expects preliminary results in late 2020 or early 2021. HOOKIPA’s second planned Phase 1/2 clinical trial will assess the safety and efficacy of the combination of HB‑201 and HB‑202 in HPV16+ cancers, with or without an approved checkpoint inhibitor. HOOKIPA remains on track to file the HB‑202 Initial New Drug submission with the U.S. Food and Drug Administration in the first half 2020. The planned HB-202/201 clinical trial is an open label, dose escalation Phase 1/2 trial with the primary endpoint to evaluate safety and tolerability. That trial is expected to commence later in 2020.

Strategic Collaborations

Gilead Sciences Collaboration for HIV and HBV Therapeutic Vaccines
During 2019, HOOKIPA received $6.0 million in milestone payments from Gilead for the delivery of research vectors and advancing the programs closer to clinical trials. Based on preclinical data generated to date, Gilead committed to preparations to advance the HBV and HIV vectors toward development, with the HBV development decision triggering a milestone payment of $4.0 million, which the Company received in early 2020. To enable the development activities and expanded research programs, Gilead agreed to reserve manufacturing capacity and increase reimbursement budgeted for the Company’s expanded resources allocated to the Gilead collaboration.

Fourth Quarter and Full Year 2019 Financial Results

Cash Position: HOOKIPA’s cash, cash equivalents and restricted cash as of December 31, 2019 was $113.6 million compared to $48.6 million as of December 31, 2018. The increase was primarily attributable to $37.3 million in net proceeds received from the issuance of shares of Series D convertible preferred stock in February 2019, and $74.6 million in net proceeds received from HOOKIPA’s initial public offering in April 2019, offset by cash used in operating and investing activities. On April 23, 2019, HOOKIPA completed an initial public offering of its common stock by issuing 6.0 million shares of its common stock, at $14.00 per share.

Revenue was $3.6 million for the three months ended December 31, 2019, and $11.9 million for the full year ended December 31, 2019 compared to $5.1 million for the three months ended December 31, 2018 and $7.6 million for the full year ended December 31, 2018. Revenue was driven by the recognition of milestone payments and partial recognition of the upfront payment as well as cost reimbursements received under the Collaboration Agreement with Gilead.

Research and Development Expenses: HOOKIPA’s research and development expenses were $11.2 million for the three months ended December 31, 2019, and $46.3 million for the full year ended December 31, 2019 compared to $4.6 million for the three months ended December 31, 2018 and $22.0 million for the full year ended December 31, 2018.

The primary drivers of the increase for 2019 were an increase in direct research and development expenses by $19.9 million, and an increase in personnel expenses by $3.1 million. Direct research and development expenses increased primarily due to the costs for conducting a Phase 2 clinical trial for the Company’s HB-101 program the preparation costs of clinical trials for HOOKIPA’s HB-201 and HB‑202 programs, expansion of earlier stage programs and costs in connection with securing manufacturing capacity for production of clinical trial material. In addition, costs related to HOOKIPA’s collaboration with Gilead contributed to the increase in direct expenses.

General and Administrative Expenses: General and administrative expenses were $5.7 million for the three months ended December 31, 2019, and $16.7 million for the full year ended December 31, 2019, compared to $2.7 million for the three months ended December 31, 2018 and $6.8 million for the full year ended December 31, 2018. The increase was mainly due to the growth in personnel related expenses, an increase in professional and consulting fees as well as costs associated with ongoing business activities and costs to operate as a public company.

Net Loss: HOOKIPA’s net loss was $10.2 million for the three months ended December 31, 2019 and $43.0 million for the full year ended December 31, 2019, compared to a net loss of $1.9 million for the three months ended December 31, 2018 and $16.2 million for the full year ended December 31, 2018. This increase was due to an increase in research and development expenses, mainly driven by the progression of HOOKIPA’s oncology programs, and an increase in general and administrative expenses following HOOKIPA’s IPO.

Conference Call

To access the live conference call, please dial +1 631 510 7495 (from the US) or +44 207 192 8000 (international) and refer to conference ID 1769733. A live audio webcast of the event will also be available within the Investors & Media section of HOOKIPA’s website at View Source An archived replay will be accessible for 30 days following the event.

On March 19, 2020 GT Biopharma, Inc. (OTCQB:GTBP)(GTBP.PA) a biotherapeutics company focused on developing innovative therapeutic treatments in oncology and infectious diseases based on its proprietary NK cell engager TriKE platform reported that it had successfully completed dosing of the first patient in a Phase I/II clinical trial of GTB-3550 for the treatment of relapsed/refractory acute myeloid leukemia (AML) (Press release, GT Biopharma, MAR 19, 2020, View Source [SID1234555694]).

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All patients enrolled in the open label clinical trial receive a single course of GTB-3550 given as four, 24-hour consecutive continuous infusions, for three consecutive weeks. The first patient with advanced relapsed/refractory AML who enrolled in the clinical trial was treated at a dose of 5µg/kg/day with GTB-3550. There were no observed adverse or serious adverse events recorded during the course of therapy, and the patient achieved stable disease as measured by AML blast count. We observed an increase in the patient’s total NK cell population that we believe is attributable to the IL-15 component of the TriKE molecule. and with no appreciable increase of any hyper-active T-cell populations, which otherwise results in cytokine storm or other T-cell associated toxicities.

GTB-3550 is a tri-specific recombinant fusion protein conjugate composed of the variable regions of the heavy and light chains of anti-CD16 and anti-CD33 antibodies and a modified form of IL-15. The NK cell stimulating cytokine human IL-15 portion of the molecule provides a self-sustaining signal that activates NK cells and enhances their cytotoxic activity.

Acute myeloid leukemia is a heterogeneous hematologic stem cell malignancy in adults with incidence rate of 3% to 5% per 100,000 people. The median age at the time of diagnosis is 65 to 69 years. AML is an aggressive cancer that is fatal on the absence of treatment. The five-year expected overall survival rate for AML is 27.4 percent, according to the National Cancer Institute (NCI).

Mr. Anthony Cataldo, the Chairman and Chief Executive Officer of GT Biopharma commented, "we are pleased to see these results in our first patient who is being administered GTB-3550." Mr. Cataldo further commented, "we remain optimistic that GTB-3550 will demonstrate therapeutic benefit in patients who have relapsed/refractory AML and other hematologic malignancies that are at an advanced stage." Mr. Cataldo added, "we believe the robustness of the TriKE platform will enable the development of therapeutics for the treatment of other cancers and certain infectious diseases, such as HIV and coronavirus infections."

On March 19, 2020 FORMA Therapeutics, Inc. ("FORMA"), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, reported that it has sold select hit discovery capabilities and related assets to a biotech startup ("NewCo") that aims to increase the efficiency of medicine development using computational-enabled capabilities (Press release, Forma Therapeutics, MAR 19, 2020, View Source [SID1234555693]).

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Under the deal terms, FORMA will receive cash and equity in NewCo as consideration. FORMA will also be eligible to receive royalties on net sales of certain products identified using NewCo’s discovery platform. FORMA retains a wholly-owned pipeline with assets ranging from early drug discovery into clinical development, as well as critical drug discovery capabilities tailored to advance its programs in rare hematologic diseases and cancers. FORMA has also secured an option from NewCo to access early discovery capabilities for a select number of programs.

Per the agreement, NewCo has taken ownership of a defined set of assets outside of FORMA’s primary areas of focus, specifically research and discovery compounds and libraries, a related global intellectual property portfolio, and tangible and intangible assets including equipment and the lease of FORMA’s discovery laboratories located in Branford, Connecticut. NewCo has also hired a group of former FORMA colleagues to further advance the transferred discovery platforms.

"This deal supports FORMA’s strategic shift to specialize in rare hematology and oncology indications, focusing our efforts on FORMA’s rich pipeline of high-quality, small molecule product candidates, including lead product candidates, FT-4202 for the treatment of sickle cell disease and FT-7051 for the treatment of castration resistant prostate cancer with AR-v7 resistant mutation, as well as multiple preclinical programs," said Frank Lee, chief executive officer of FORMA Therapeutics.

On March 19, 2020 Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) reported that it will report its financial results for 2019 on Thursday, 26 March 2020 (Press release, Evotec, MAR 19, 2020, View Source;announcements/press-releases/p/evotec-se-to-report-fiscal-year-2019-results-on-26-march-2020-5913 [SID1234555692]).

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The Company is going to hold a conference call to discuss the results as well as to provide an update on its performance. Furthermore, the Management Board will present an outlook for fiscal year 2020. The conference call will be held in English.

Conference call details

Date: Thursday, 26 March 2020

Time: 02.00 pm CET (09.00 am EDT, 01.00 pm GMT)

From Germany: +49 69 201 744 220

From France: +33 170 709 502

From Italy: +39 02 3600 6663

From the UK: +44 20 3009 2470

From the USA: +1 877 423 0830

Access Code: 97681453#

A simultaneous slide presentation for participants dialling in via phone is available at View Source

Webcast details

To join the audio webcast and to access the presentation slides you will find a link on our home page www.evotec.com shortly before the event.

A replay of the conference call will be available for 24 hours and can be accessed in Europe by dialling +49 69 20 17 44 221 (Germany) or +44 20 3364 5150 (UK) and in the USA by dialling +1 844 307 9362. The access code is 315578854#. The on-demand version of the webcast will be available on our website: View Source

On March 19, 2020 AstraZeneca reported that Lynparza (olaparib) has been granted orphan drug designation (ODD) in Japan for the maintenance treatment of germline BRCA-mutated (gBRCAm) curatively unresectable pancreatic cancer (Press release, AstraZeneca, MAR 19, 2020, View Source [SID1234555691]). Lynparza is co-developed and co-commercialised with MSD Inc., Kenilworth, N.J., US (MSD: known as Merck & Co., Inc. inside the US and Canada).

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The Japanese Ministry of Health, Labour and Welfare grants ODD to medicines intended for the treatment of diseases that affect fewer than 50,000 patients in Japan and for which there is a high unmet medical need.

Pancreatic cancer has the lowest survival rate of the most common cancers and is the only major cancer with a five-year survival rate below 10% in almost every country.

José Baselga, Executive Vice President, R&D Oncology, said: "Japan has the fifth-highest incidence of pancreatic cancer worldwide and patients have seen limited treatment advances over the last few decades. This designation is an important step forward in bringing the first targeted medicine to biomarker-selected patients with advanced pancreatic cancer in Japan."

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: "A pancreatic cancer diagnosis is devastating and we are committed to research that aims to change the prognosis for patients. The POLO trial demonstrated that treatment with Lynparza extended time without disease progression or death in patients with germline BRCA-mutated metastatic pancreatic cancer and we are hopeful that we will be able to bring this treatment to patients in Japan soon."

The Phase III POLO trial showed Lynparza nearly doubled the time patients with gBRCAm metastatic pancreatic cancer lived without disease progression or death to a median of 7.4 months versus 3.8 months on placebo. The safety and tolerability profile of Lynparza in the POLO trial was in line with that observed in previous trials.

Lynparza was approved in the US as a 1st-line maintenance treatment for patients with gBRCAm metastatic pancreatic cancer in December 2019 with ongoing regulatory reviews in the EU and other jurisdictions.

Pancreatic cancer

Pancreatic cancer is a deadly cancer with a high unmet medical need. The disease has the lowest survival rate of the most common cancers.1 Japan has the fifth-highest rate of pancreatic cancer in the world with 43,000 new cases diagnosed in 2018.2,3 Pancreatic cancer is the fourth leading cause of cancer death in Japan, resulting in 37,000 deaths in 2018.3

Globally, pancreatic cancer is the 11th-most commonly occurring cancer and the seventh leading cause of cancer death.4,5 There were approximately 460,000 new cases worldwide in 2018.5 As there are often no symptoms, or symptoms may be non-specific in the early stages, it is most commonly diagnosed at an incurable stage.6,7 Around 80% of pancreatic cancer patients are diagnosed when the disease has metastasised, at which point average survival is less than a year.8 Despite advances in treatment, few improvements have been made in diagnosis and treatment in the past few decades.9 Current treatment is surgery (for which approximately only 10-20% of patients are eligible), chemotherapy and radiotherapy, highlighting a critical unmet medical need for more effective treatment options.10

POLO

POLO is a Phase III randomised, double-blind, placebo-controlled, multi-centre trial of Lynparza tablets (300mg twice daily) as maintenance monotherapy versus placebo. The trial randomised 154 patients with gBRCAm metastatic pancreatic cancer whose disease had not progressed on 1st-line platinum-based chemotherapy. Patients were randomised (3:2) to receive Lynparza or placebo until disease progression. The primary endpoint was progression-free survival (PFS) and key secondary endpoints included overall survival (OS), time to second disease progression, overall response rate and health-related quality of life.

The results showed a statistically significant and clinically meaningful improvement in PFS, where Lynparza nearly doubled the time patients with gBRCAm metastatic pancreatic cancer lived without disease progression or death to a median of 7.4 months versus 3.8 months on placebo and reduced the risk of disease progression or death by 47% (HR 0.53 [95% confidence interval (CI), 0.35-0.82], p=0.004). In patients with measurable disease at baseline, 23% responded to Lynparza versus 12% on placebo (odds ratio, 2.30 [95% CI, 0.89-6.76]) and had a median duration of treatment in excess of two years (24.9 months [95% CI, 14.8-could not be calculated]) versus 3.7 months on placebo (95% CI, 2.10-could not be calculated). The median OS, a secondary endpoint, at interim analysis and at a data maturity of 46% was 18.9 months for Lynparza versus 18.1 months for placebo but did not reach statistical significance (HR=0.91; p=0.68).

The safety and tolerability profile of Lynparza in the POLO trial was in line with that observed in previous trials.

BRCA mutations

BRCA1 and BRCA2 (breast cancer susceptibility genes 1/2) are human genes that produce proteins responsible for repairing damaged DNA and play an important role in maintaining the genetic stability of cells. When either of these genes is mutated, or altered, such that its protein product either is not made or does not function correctly, DNA damage may not be repaired properly, and cells become unstable. As a result, cells are more likely to develop additional genetic alterations that can lead to cancer.

Lynparza

Lynparza (olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in homologous recombination repair, such as mutations in BRCA1 and/or BRCA2. Inhibition of PARP with Lynparza leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death. Lynparza is being tested in a range of PARP-dependent tumour types with defects and dependencies in the DDR pathway.

Lynparza is currently approved in a number of countries, including those in the EU, for the maintenance treatment of platinum-sensitive relapsed ovarian cancer. It is approved in the US, the EU, Japan, China, and several other countries as 1st-line maintenance treatment of BRCA-mutated advanced ovarian cancer following response to platinum-based chemotherapy. It is also approved in the US, Japan, and a number of other countries for germline BRCA-mutated, HER2-negative, metastatic breast cancer, previously treated with chemotherapy; in the EU, this includes locally advanced breast cancer. In 2019, Lynparza was additionally approved in the US for the treatment of germline BRCA-mutated metastatic pancreatic cancer. Regulatory reviews are underway in several jurisdictions for ovarian, breast, pancreatic and prostate cancers.

Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, has been used to treat over 30,000 patients worldwide. Lynparza has the broadest and most advanced clinical trial development programme of any PARP inhibitor, and AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types. Lynparza is the foundation of AstraZeneca’s industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.

The AstraZeneca and MSD strategic oncology collaboration

In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and potential new medicine selumetinib, a MEK inhibitor, for multiple cancer types. Working together, the companies will develop Lynparza and selumetinib in combination with other potential new medicines and as monotherapies. Independently, the companies will develop Lynparza and selumetinib in combination with their respective PD-L1 and PD-1 medicines.

AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With six new medicines launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to AstraZeneca’s main capabilities, the Company is actively pursuing innovative partnerships and investment that accelerate the delivery of our strategy, as illustrated by the investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and, one day, eliminate cancer as a cause of death.