On March 24, 2020 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported company highlights and financial results for the fourth quarter and year ended December 31, 2019 (Press release, Alector, MAR 24, 2020, View Source [SID1234555781]).

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"At Alector, we envision a world where dementia and neurodegeneration are illnesses of the past, and even amidst the uncertainties we face today in light of the COVID-19 pandemic, we remain committed to this vision. As of today, four of our novel immuno-neurology programs are advancing in the clinic, we have received two Fast Track designations from the FDA, generated sufficient data to support the launch of a pivotal Phase 3 trial in with AL001 in FTD-GRN patients, and expanded our Alzheimer’s disease portfolio from two to three prioritized development product candidates," said Arnon Rosenthal, Ph.D., Co-founder, and Chief Executive Officer of Alector. "We look forward to progressing our clinical and research programs forward in 2020, and with our strong cash position and experienced team, we believe we are well-positioned to execute on our milestones throughout the year."

Recent Business Highlights

Progranulin Portfolio: AL001, AL101

In December 2019, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation for AL001 for the treatment of FTD-GRN patients.

In December 2019, Alector reported Phase 1b results of AL001 in symptomatic FTD-GRN patients. In the trial, AL001 met all primary and secondary endpoints. Treatments with AL001 also showed a statistically significant normalization in a number of disease-associated proteins and an initial trend of decreasing levels of plasma Neurofilament (NfL) in patients compared to baseline.

Alector initiated an open-label Phase 2 trial investigating the use of AL001 in patients with FTD-GRN and FTD-C9orf72 in September 2019. The company plans to present data from the ongoing Phase 2 study of AL001 patients at a conference later this year and advance AL001 into a pivotal Phase 3 study in 2020.

In January 2020, Alector initiated a Phase 1 study of AL101 in healthy volunteers.

In February 2020, the FDA granted AL101 Fast Track Designation for the treatment of patients with FTD-GRN.
Alzheimer’s Disease Portfolio: AL002, AL003, AL014

In December 2019, Alector reported results from the single ascending dose phase of the Phase 1 study of AL002 in healthy volunteers at the 12th Clinical Trials on Alzheimer’s Disease (CTAD) Meeting. AL002 achieved all primary and secondary endpoints, including safety. AL002 was generally well-tolerated and reduced CSF soluble TREM2 (sTREM2) in a dose-dependent manner. AL002 is being developed by Alector in collaboration with its partner AbbVie.

In December 2019, Alector designated AL014 as its third prioritized product candidate in Alzheimer’s disease. AL014 targets MS4A4A, a transmembrane receptor protein that is expressed selectively in microglia in the brain and is associated with control of microglia functionality and/or viability. Alector plans to initiate Phase 1 development for AL014 within the next 12-15 months.

In January 2020, Alector announced the dosing of the first Alzheimer’s disease patient in the Phase 1b portion of the clinical study of AL003. AL003 is being developed in collaboration with its partner, AbbVie.
Other Business Highlights

In December 2019, Alector announced the appointment of Shehnaaz Suliman, M.D., MBA, M.Phil., as the company’s President and Chief Operating Officer. Dr. Suliman brings over 20 years of business development, drug development, strategic and operational expertise, and executive leadership skills to Alector.

In December 2019, the company also announced the appointment of Richard Scheller, Ph.D., and Thomas Südhof, M.D., as Co-chairs of the company’s Strategic Portfolio Advice and Review Committee (SPARC). Dr. Scheller, brings a strong track-record of developing new medicines and leading multiple R&D organizations. Dr. Südhof is a renowned neuroscientist and winner of the Nobel Prize in Physiology or Medicine in 2013.

In February 2020, the company announced the closing of an underwritten public offering with total gross proceeds of approximately $240.1 million.

Alector is proactively monitoring and assessing the current COVID-19 global pandemic. The Company is focused on avoiding and minimizing delays to its business initiatives, with the safety and well-being of employees, patients and partners as its highest priority. As of early March, the Company activated a taskforce focused on closely monitoring the situation daily, as well as assessing the potential business impact that may result from this rapidly evolving crisis. Following the completion of its recent public follow-on offering, the Company is financially well positioned to continue executing on its key business priorities.
Fourth Quarter and Full Year 2019 Financial Results

Revenue. Collaboration revenue for the quarter ended December 31, 2019, was $6.0 million compared to $9.1 million for the same period in 2018. Collaboration revenue for the year ended December 31, 2019, was $21.2 million compared to $27.5 million for the same period in 2018. This decrease was primarily due to an increase in total expected costs for the AL002 and AL003 programs through the completion of proof-of concept.

R&D Expenses. Total research and development expenses for the quarter ended December 31, 2019, were $25.8 million compared to $24.1 million for the same period in 2018. Total research and development expenses for the year ended December 31, 2019, were $100.5 million compared to $73.0 million for the same period in 2018. This increase was driven by an increase in personnel-related expenses as headcount grew to support the advancement of the clinical and preclinical programs. There was an increase in facilities and other unallocated research and development related to the lease expense for the new headquarters and higher depreciation expense. Expenses for AL101 increased as well as for other early stage programs as investment in research and clinical pipeline continues.

G&A Expenses. Total general and administrative expenses for the quarter ended December 31, 2019 were $12.6 million compared to $4.1 million for the same period in 2018. Total general and administrative expenses for the year ended December 31, 2019 were $35.1 million compared to $11.9 million for the same period in 2018. This increase was primarily due to an increase in personnel-related expenses due to increased headcount. Facilities and general overhead expenses increased due to additional lease expense related to the lease for the new headquarters, higher depreciation expense, directors and officers’ insurance, and additional legal, information technology, and human resources costs to operate as a public company.

Net Loss. For the quarter ended December 31, 2019, Alector reported a net loss of $30.5 million, compared to a net loss of $17.4 million for the same period in 2018. For the year ended December 31, 2019, Alector reported a net loss of $105.4 million, compared to a net loss of $52.2 million for the same period in 2018.

Cash Position. Cash, cash equivalents, and marketable securities were $353.1 million as of December 31, 2019.

On March 24, 2020 IDEAYA Biosciences, Inc. (Nasdaq:IDYA), an oncology-focused precision medicine company committed to the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics, provided a business update and reported financial results for the fourth quarter ended December 31, 2019 (Press release, Ideaya Biosciences, MAR 24, 2020, View Source [SID1234555778]).

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"IDEAYA is building a leading synthetic lethality biopharmaceutical company, advancing a broad pipeline of synthetic lethality programs, including our MAT2A program for which we have selected a lead compound MAT2A inhibitor. We also continue to advance development of IDE196 in our Phase 1/2 tissue-agnostic basket trial in patients with solid tumors harboring GNAQ or GNA11 (GNAQ/11) mutations such as metastatic uveal melanoma (MUM), cutaneous melanoma and colorectal cancer, including evaluation of IDE196 in combination with binimetinib under a clinical trial collaboration and supply agreement with Pfizer," said Yujiro S. Hata, Chief Executive Officer and President at IDEAYA Biosciences.

We continue to progress our MAT2A synthetic lethality program for tumors with MTAP deletion. We have selected a lead compound which we believe has favorably differentiated activity, physical properties and tolerability, and have scaled this lead compound for non-GLP toxicology studies in two species to support selection of a development candidate in the second quarter of 2020.

We also continue to progress our broad pipeline of synthetic lethality programs, including Pol theta for tumors with BRCA or other homologous recombination deficiency (HRD) mutations, Werner (WRN) for tumors with high microsatellite instability (MSI), and PARG for tumors with BRCA2 mutations, impaired base excision repair, or replication stress signature. We are applying our fully integrated research and translational capabilities to these programs. We have solved the crystal structures for each of these research programs, and we are conducting preclinical in vivo efficacy studies in three of our synthetic lethality programs.

Key highlights for IDEAYA’s research and development programs include:

Clinical Program IDE196

IDE196

Advanced IDEAYA’s Phase 1/2 tissue-type agnostic basket trial, initiated in June 2019, to evaluate IDE196 in solid tumors harboring activating GNAQ/11 mutations, entitled "A phase 1/2 study of IDE196 in patients with solid tumors harboring GNAQ/11 mutations or PRKC fusions" (ClinicalTrials.gov Identifier: NCT03947385). As of March 15, 2020:

Enrolled 53 patients in IDE196 monotherapy arm of Phase 1/2 clinical trial

Ongoing evaluation of IDE196 monotherapy arm in MUM, with aggregate enrollment of 49 patients in the Phase 1 dose escalation and tablet formulation studies

Initiated the Phase 2 expansion for IDE196 as a monotherapy in solid tumors other than MUM having GNAQ or GNA11 hotspot mutations, with aggregate Phase 1/2 enrollment of 4 cutaneous melanoma patients

Selected 400mg BID (with one week 200 mg BID run-in) as Phase 2 monotherapy dose; observed higher average steady state exposure of free IDE196 (AUCfree, increase of approximately 44%) and higher trough concentration of IDE196 (Cmin, increase of approximately 40%) at 400 mg BID relative to 300 mg BID dose

Evaluating tablet formulation of IDE196 in MUM patients in a Phase 1 sub-study, with the pharmacokinetic profile of the tablet formulation comparable to the powder-in-capsule form of IDE196

Completed in-life portion of the ongoing 13-week GLP-compliant toxicology studies in two species, initiated in November 2019

Interim data from the monotherapy arm of the Phase 1/2 basket trial targeted for second half 2020

Entered into a clinical trial collaboration and supply agreement with Pfizer; targeting to initiate combination arm of Phase 1/2 clinical trial in mid-2020 to evaluate safety and efficacy of IDE196 in combination with binimetinib, a MEK inhibitor, in patients having tumors with activating GNAQ or GNA11 hotspot mutations, including in metastatic uveal melanoma and other solid tumors

Design and initiation of potential registration-enabling study in MUM will be evaluated based on results of ongoing IDE196 monotherapy arm and planned IDE196 / binimetinib combination arm of the Phase 1/2 clinical trial

Preclinical Synthetic Lethality Programs

MAT2A

Observed single agent in vivo efficacy of our MAT2A inhibitors, including tumor growth inhibition or tumor regression in multiple MTAP -/- endogenous models

Selected a lead compound which we believe has favorably differentiated in vivo activity, physical properties and tolerability profile relative to published Agios compounds

Scaled the MAT2A lead compound for non-GLP toxicology studies in two species to support selection of a development candidate in the second quarter of 2020

Expect to file an IND for MAT2A inhibitor development candidate in fourth quarter of 2020

Pol Theta

Observed monotherapy activity, showing cell viability activity and in vivo tumor growth inhibition in a DLD1 BRCA2 -/- engineered model

Observed combination activity with a PARPi, Olaparib, as well as synergistic cell viability activity and synergistic in vivo tumor growth inhibition in the DLD1 BRCA2 -/- engineered model, with a weak drug-drug interaction signal

Targeting designation of Pol-theta inhibitor development candidate in second half of 2020

Werner (WRN)

Observed dose-dependent cellular viability effect in multiple endogenous MSI high cell lines, with an expected lack of activity in microsatellite stable, or MSS, cell lines

Observed dose-dependent cellular pharmacodynamic (PD) response in multiple endogenous MSI high cell lines

Solved crystal structure of WRN helicase domain

Targeting to demonstrate in vivo proof of concept in relevant animal models in 2020

PARG

Observed dose-dependent cellular pharmacodynamic response and cellular viability effect in a HCC1806 XRCC1 -/- cell line

Expanded research collaboration with Cancer Research UK (CRUK) and the University of Manchester, UK, to evaluate IDEAYA’s potent selective PARG inhibitors in vitro and in vivo in multiple ovarian cancer cell lines and xenograft models, respectively, and to evaluate replication stress signature as a potential patient selection biomarker

"We continue to advance our programs, expand our capabilities and enhance our team. We believe that the IDE196 clinical program and our preclinical pipeline of synthetic lethality programs are maturing, moving forward toward our goal of improving lives through transformative precision medicines," said Yujiro S. Hata, Chief Executive Officer and President at IDEAYA Biosciences.

Corporate Updates

IDEAYA anticipates that existing cash, cash equivalents, and short-term and long-term marketable securities of $100.5 million (as of December 31, 2019) will be sufficient to fund planned operations into the end of 2021 to early 2022.

Our updated corporate presentation is available on our website, in the Presentations section of our Investor Relations page. See: View Source

Financial Results

As of December 31, 2019, IDEAYA had cash, cash equivalents, and short-term and long-term marketable securities totaling $100.5 million. This compared to cash, cash equivalents and short-term marketable securities of $90.0 million at December 31, 2018. The increase was primarily due to the receipt of $50.2 million in net proceeds from IDEAYA’s initial public offering, which was completed in May 2019, offset by cash used in operations.

Research and development expenses for the three months ended December 31, 2019 totaled $8.5 million compared to $7.6 million for the same period in 2018. The increase

was primarily due to costs in connection with IDEAYA’s Phase 1/2 clinical trial to evaluate IDE196 in solid tumors, and costs for personnel and consulting in support of our research programs during the three months ended December 31, 2019.

General and administrative expenses for the three months ended December 31, 2019 totaled $2.8 million compared to $1.6 million for the same period in 2018. The increase was primarily due to an increase in costs for personnel and directors’ and officers’ liability insurance premiums in connection with becoming a publicly traded company.

Research and development expenses for the year ended December 31, 2019 totaled $34.3 million compared to $31.7 million for 2018. The increase was primarily due to an increase in costs in connection with IDEAYA’s Phase 1/2 clinical trial to evaluate IDE196 in solid tumors, and costs for personnel and consulting in support of our research programs during the year ended December 31, 2019, offset by a decrease in license fees for our IDE196 license agreement with Novartis during the year ended December 31, 2018.

General and administrative expenses for the year ended December 31, 2019 totaled $10.0 million compared to $4.7 million for 2018. The increase was primarily due to an increase in costs for personnel, directors’ and officers’ liability insurance premiums, and professional fees in connection with becoming a publicly traded company.

The net loss for the three months ended December 31, 2019 was $10.8 million compared to $8.6 million for the same period in 2018. Total stock compensation expense for the three months ended December 31, 2019 was $0.7 million compared to $0.3 million for the same period in 2018.

The net loss for the year ended December 31, 2019 was $42.0 million compared to $34.3 million for the same period in 2018. Total stock compensation expense for the year ended December 31, 2019 was $2.2 million compared to $1.0 million for the same period in 2018.

On March 24, 2020 Triumvira Immunologics, Inc. (Triumvira), a private, clinical-stage biopharmaceutical company developing a novel platform for engineering T-cells to attack cancers, and Lonza, a leading cell & gene therapy manufacturer, reported that the companies have an agreement in place (Press release, Triumvira Immunologics, MAR 24, 2020, View Source [SID1234555777]).

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This collaboration, aimed at the development of Triumvira’s TAC01-HER2 T-cell therapy for treating solid tumors, will leverage Lonza’s expertise in process development and the usage of its proprietary Cocoon Platform. This automated patient-scale cell therapy manufacturing platform offers flexibility to execute a wide variety of protocols within a single system is enabled through a single-use, highly customizable cassette and custom programming.

Immunotherapy, particularly T-cell products, belong to the most potent drugs that could overcome the complex barriers encountered in solid tumors. However, current T-cell approaches remain insufficiently effective. The TAC receptor represents a significant advancement, providing a novel mechanism of T-cell activation, leading to improved anti-tumor responses in solid tumor models.

TAC01-HER2 has demonstrated encouraging and repeated safety and efficacy in multiple preclinical models of HER2-overexpressing tumors. Within this collaboration, TAC01-HER2 will initially be tested in a Phase 1/2 clinical trial examining different HER2-overexpressing solid tumors.

On March 24, 2020 Oncopeptides AB (Nasdaq Stockholm: ONCO) reported the publication of results from its O-12-M1 study, melflufen plus dexamethasone in relapsed/refractory multiple myeloma (RRMM), a multicenter, international, open-label, phase 1/2 study in The Lancet Haematology (Press release, Oncopeptides, MAR 24, 2020, View Source [SID1234555761]). Melflufen is a first-in-class anti-cancer peptide-drug conjugate currently in development by Oncopeptides with activity in a variety of cancers. Current clinical development is focused on the treatment of RRMM.

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In the O-12-M1 study, heavily pre-treated RRMM patients with a median of four prior lines of therapy (2-14), were dosed with 40 mg of melflufen administered intravenously once every 28 days in combination with weekly dexamethasone. Patients receiving melflufen plus dexamethasone experienced an overall response rate (ORR) of 31%, a median duration of response (DOR) of 8.4 months, and median overall survival (OS) of 20.7 months.

"We are excited to share with the scientific community these findings from our phase 1/2 study of our peptide-drug conjugate, melflufen. The results published today served as the foundation of our broad clinical development program," said Klaas Bakker, Chief Medical Officer of Oncopeptides. "We recognize the significant unmet needs of patients with RRMM who currently have few available treatment options and are in desperate need of well-tolerated treatments with the potential to overcome cancer resistance patterns. I would like to thank all patients and their care partners for participation in this study."

Additional findings from the study include:

The median progression-free survival was 5.7 months
The most common adverse events were thrombocytopenia (62%) and neutropenia (58%), while grade 3 or higher non-haematological toxicities were infrequent
Melflufen plus dexamethasone showed activity regardless of previous treatment failure, including in patients with disease refractory to alkylators
More information about the O-12-M1 study is available on www.oncopeptides.com.

For more information, please contact:

Rein Piir, Head of Investor Relations at Oncopeptides
E-mail: [email protected]
Cell phone: +46 70 853 72 92

This information was submitted for publication at 00.35 CET March 24, 2020.

About melflufen

Melflufen (INN melphalan flufenamide) is a first-in-class anti-cancer peptide-drug conjugate that rapidly delivers an alkylating payload into tumor cells. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is immediately cleaved by peptidases to deliver an entrapped hydrophilic alkylator payload. Peptidases play a key role in protein homeostasis and feature in cellular processes such as cell-cycle progression and programmed cell death. In vitro, melflufen is 50-fold more potent in myeloma cells than the alkylator payload itself due to the increased intracellular alkylator concentration. Melflufen displays cytotoxic activity against myeloma cell lines resistant to other treatments, including alkylators, and has also demonstrated inhibition of DNA repair induction and angiogenesis in preclinical studies.

On March 23, 2020 Oasmia reported that it will hold an audiocast and telephone conference on Friday 27 March at 10:00 (CET) to present the company’s Q3 report for the financial year 2019/2020 (Press release, Oasmia, MAR 23, 2020, View Source [SID1234556576]).

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The presentation of the Q3 report will be in English.

Presenters will be Francois Martelet, CEO, and Michael af Winklerfelt, CFO.

The audiocast is viewed via the link
View Source
It will be possible to ask texted questions directly via the Audiocast solution.