on November 5, 2020 BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, reported its financial results for the third quarter ended September 30, 2020 and provided an update on the company’s operations (Press release, BridgeBio, NOV 5, 2020, View Source [SID1234570007]).

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BridgeBio announced a merger agreement last month with Eidos Therapeutics, Inc. (Nasdaq: EIDX), which is developing acoramidis (formerly AG10), a potential best-in-class transthyretin (TTR) stabilizer for patients with TTR amyloid (ATTR) cardiomyopathy and polyneuropathy. With this transaction, BridgeBio intends to fully and formally welcome Eidos back into its vibrant ecosystem of innovation and has agreed to acquire all of the outstanding common stock of Eidos it does not already own. The company expects to complete the proposed transaction in the first quarter of 2021, subject to certain conditions, including the receipt of stockholder approvals.

Since the company’s last quarterly update, BridgeBio had its first new drug application (NDA) accepted by the U.S. Food and Drug Administration (FDA) under Priority Review designation and initiated two new clinical trials, including a Phase 2 trial of encaleret (calcium sensing receptor antagonist) for autosomal dominant hypocalcemia type 1 (ADH1), one of BridgeBio’s four core value driver programs. It also entered into collaboration agreements with the Salk Institute and the University of Colorado Anschutz Medical Campus to advance the development of new therapies for genetically driven diseases.

BridgeBio held its first-ever R&D Day on September 29, 2020, which focused on the company’s drug engineering platform, its targeted oncology portfolio, and four highlighted programs where clinical data are anticipated in the next 12 to 18 months – acoramidis for ATTR, low-dose infigratinib (FGFR inhibitor) for achondroplasia, AAV5 gene therapy for congenital adrenal hyperplasia (CAH), and encaleret for ADH1.

"We are nearing a significant inflection point as a company as we approach the start of 2021. Our four key programs have critical data readouts within the next year and a half – in ATTR, achondroplasia, CAH and ADH1. We are progressing 17 ongoing clinical trials and we are preparing for commercialization, to bring our first investigational therapy to patients. There has never been a more exciting moment to be at the forefront of the revolution taking place in genetic medicine," said BridgeBio CEO and founder Neil Kumar, Ph.D.

Recent pipeline progress and corporate updates:

BridgeBio and Eidos Therapeutics enter into merger agreement: BridgeBio to acquire all outstanding shares of common stock of Eidos it does not already own; agreement unanimously approved by special committee of Eidos’ independent directors. Transaction removes the operational complexity of the current ownership structure and allows BridgeBio to fully invest in opportunities around the investigational drug, acoramidis, including subsequent studies to potentially broaden the evidence for its usage, and accelerate its commercial development using BridgeBio’s established infrastructure. Proposed transaction expected to be completed in the first quarter of 2021, subject to certain conditions, including approval by both BridgeBio and Eidos stockholders.

Fosdenopterin (formerly BBP-870/ORGN001) – Synthetic cPMP for molybdenum cofactor deficiency (MoCD) Type A: FDA acceptance of NDA under Priority Review designation with Breakthrough Therapy Designation and Rare Pediatric Disease Designation previously granted. There are currently no approved therapies for the treatment of MoCD Type A, which results in severe and irreversible neurological injury for infants and children. This is BridgeBio’s first NDA acceptance.

New academic partnerships: Established collaboration agreements with the Salk Institute and the University of Colorado Anschutz Medical Campus to advance the discovery of therapies for genetically driven diseases.

BridgeBio Pharma R&D Day: Held a virtual R&D Day on September 29, 2020. Presentation replay can be found on BridgeBio’s investor website here.

Major milestones anticipated over the next 12-18 months for BridgeBio’s four core value drivers:

Acoramidis (AG10) – TTR stabilizer for ATTR: Completed screening in September for pivotal Phase 3 ATTRibute-CM clinical trial of acoramidis in patients with ATTR cardiomyopathy. The study enrolled more than 600 subjects with either wild-type or variant TTR across more than 80 sites in 18 countries. Topline results from Part A of the ATTRibute-CM trial are expected in late 2021 or early 2022 and from Part B in 2023. If Part A is successful, intend to file for regulatory approval of acoramidis in 2022.

Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia: Remain on track to report initial data from the ongoing Phase 2 dose ranging study by end of 2021. Achondroplasia is the most common form of genetic short stature and one of the most commonly known genetic diseases, with 55,000 cases in the United States and European Union. Low-dose infigratinib is the only known therapy in development for achondroplasia that targets the disease at its genetic source and the only orally administered product candidate in clinical stage development.

Encaleret – CaSR antagonist for ADH1: Initiated Phase 2 clinical study and dosed first patients, with topline proof-of-concept results anticipated in 2021. If the development program is successful, encaleret would be the first approved therapy for ADH1, a condition caused by gain of function variants in the CaSR gene estimated to be carried by 12,000 individuals in the United States.

BBP-631 – AAV5 gene therapy candidate for CAH: Investigational New Drug (IND) application-enabling studies for AAV gene therapy proceeding. Remain on track to initiate a first in human Phase 1/2 study and report initial data in 2021. CAH is one of the most prevalent genetic diseases thought to be addressable with AAV gene therapy, with more than 75,000 cases in the United States and European Union.

Third quarter 2020 financial results:

Cash, Cash Equivalents and Marketable Securities

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $710.7 million as of September 30, 2020, compared to $577.1 million at December 31, 2019. The net increase in cash balance of $133.6 million reflects $537.0 million in net proceeds received from the issuance of our 2.50% Convertible Senior Notes due 2027 (2027 Notes), $24.1 million in net proceeds received from Eidos’ at-the-market issuance of shares, offset by payment of $75.0 million to repurchase BridgeBio shares in capped call transactions in connection with the issuance of our 2027 Notes, $49.3 million payment related to capped call option, $13.3 million payments of interest on our debts, and $289.9 million primarily related to operating expenses.

Cash, cash equivalents and marketable securities, excluding restricted cash, decreased by $130.2 million compared to our balance as of June 30, 2020, which was $840.9 million. The decrease in cash reflects $9.2 million payments of interests on our debts and $121.0 million primarily related to operating expenses.

Operating Expenses

Operating expenses for the three and nine months ended September 30, 2020 were $128.1 million and $355.1 million, respectively, as compared to $81.3 million and $214.3 million, respectively, for the same periods in the prior year. The increases in operating expenses of $46.8 million and $140.8 million during the respective periods were attributable to the increase in external-related costs and increase in headcount to support the progression in our research and development programs, including our increasing research pipelines, and overall growth of our operations.

Operating expenses for the three months ended September 30, 2020 increased by $3.5 million when compared to the operating expenses for the three months ended June 30, 2020 of $124.6 million.

Our research and development expenses have not been significantly impacted by the global outbreak of COVID-19 for the periods presented. While we experienced some initial delays in certain of our clinical enrollment and trial commencement activities, we continue to adapt in this unprecedented time to enable alternative site, telehealth and home visits, at home drug delivery, as well as mitigation strategies with our contract manufacturing organizations. The longer-term impact of COVID-19 on our operating expenses is currently unknown.

The condensed consolidated balance sheet as of December 31, 2019 is derived from the audited consolidated financial statements as of that date. Certain reclassifications have been made to conform to the September 30, 2020 condensed consolidated balance sheet presentation.

December 31, 2019 amounts include long-term marketable securities of $31.1 million.

On November 5, 2020 Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO) reported that the company will arrange a virtual capital markets day for investors, analysts and journalists on November 30th, 2020 (Press release, Oncopeptides, NOV 5, 2020, View Source [SID1234570006]). The program will be published on the company´s website during the week of November 23 at: www.oncopeptides.com/en/calendar/

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Oncopeptides Virtual Capital Markets Day
14.00 – 16.00 CET (8:00am-10am ET) November 30th, 2020

The presenters during the Capital Market Day will be:

CEO Marty J Duvall, Oncopeptides,
CSO, Jakob Lindberg, Oncopeptides
CMO Klaas Bakker, Oncopeptides

Professor Paul Richardson, MD, Dana-Farber Cancer Institute, Boston
Associate Professor Maria-Victoria Mateos, MD, PhD, University Hospital, Salamanca
Assistant Professor Joshua Richter, MD, Icahn School of Medicine, Mount Sinai Hospital, New York

The webcast will be accessible at: View Source

The information was submitted for publication at 14.00 CET on November 5, 2020.

On November 5, 2020 Sierra Oncology, Inc. (SRRA), a late-stage biopharmaceutical company focused on the Phase 3 execution, registration and potential commercialization of momelotinib, a novel drug that may address serious unmet needs in myelofibrosis, reported two abstracts have been selected for presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held December 5-8, 2020 (Press release, Sierra Oncology, NOV 5, 2020, View Source [SID1234570005]).

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"Data presented at this year’s ASH (Free ASH Whitepaper) annual meeting may support the potential of momelotinib as a unique treatment option for myelofibrosis patients, capable of improving all three hallmarks of disease: anemia, symptoms and spleen," said Barbara Klencke, M.D., Chief Development Officer at Sierra Oncology. "Further, activity is demonstrated in thrombocytopenic patients, regardless of previous treatment with a JAK inhibitor. Collectively, the additional data presented from the SIMPLIFY-1 and SIMPLIFY-2 clinical trials may demonstrate potential long-term survival, and the possibility that more patients may become transfusion independent. A similar clinical profile has not been seen with other agents at this time."

Robust Overall Survival and Sustained Efficacy Outcomes During Long Term Exposure to Momelotinib in JAK Inhibitor Naïve and Previously JAK Inhibitor Treated Intermediate/High Risk Myelofibrosis Patients

Long-term overall survival data from the previously completed SIMPLIFY-1 and SIMPLIFY-2 Phase 3 trials will be reported in an oral presentation by Srdan Verstovsek, MD, PhD, Chief, Section for Myeloproliferative Neoplasms, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas. The trials evaluated JAK inhibitor-naïve and previously JAK inhibitor-treated patients with myelofibrosis who were either randomized to receive momelotinib or were dosed initially with ruxolitinib or best available therapy followed by momelotinib.

Presentation Details

Abstract: 54
Title: Robust Overall Survival and Sustained Efficacy Outcomes During Long Term Exposure to Momelotinib in JAK Inhibitor Naïve and Previously JAK Inhibitor Treated Intermediate/High Risk Myelofibrosis Patients
Presenter: Srdan Verstovsek, MD, PhD
Session Name: 634. Myeloproliferative Syndromes: Clinical: New Therapies and JAKi-based Combinations for Myelofibrosis
Session Information: Saturday, December 5, 2020; 7:30 AM – 9:00 AM PT
Presentation Time: 8:15 AM PT

Momelotinib’s Spleen, Symptom and Anemia Efficacy is Maintained in Intermediate/High Risk Myelofibrosis Patients with Thrombocytopenia

Comparative efficacy data for momelotinib and ruxolitinib in patients with low platelets from SIMPLIFY-1 and SIMPLIFY-2 will be presented in a poster presentation by Jean-Jacques Kiladjian, MD, PhD, Professor of Clinical Pharmacology, Paris Diderot University; Consultant Hematologist, Head, Clinical Investigation Center, Saint Louis Hospital, Paris, France. The presentation will include post-hoc comparative efficacy analyses for momelotinib and ruxolitinib for spleen, symptom and transfusion independence response in patients with baseline platelet counts of <150 x 109/L versus the ITT populations from the two previously completed global Phase 3 SIMPLIFY studies. A baseline platelet limit of ≥50 × 109/L was required in SIMPLIFY-1 while there was no lower platelet limit for SIMPLIFY-2. In SIMPLIFY-2, most patients randomized to best available therapy (88%) received ruxolitinib during the randomization period.

Presentation Details

Abstract: 3086
Title: Momelotinib’s Spleen, Symptom and Anemia Efficacy is Maintained in Intermediate/High Risk Myelofibrosis Patients with Thrombocytopenia
Presenter: Jean-Jacques Kiladjian, MD, PhD
Session Name: 634. Myeloproliferative Syndromes: Clinical: Poster III
Session Information: Monday, December 7, 2020; 7:00 AM – 3:30 PM PT

On November 5, 2020 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody technology platform, reported the treatment of the first patient in the Phase 2 expansion study of CX-2029, an anti-CD71 Probody drug conjugate (Press release, CytomX Therapeutics, NOV 5, 2020, View Source [SID1234570004]). The study, being conducted under a partnership with AbbVie, is evaluating CX-2029 as monotherapy in four cohorts; squamous non-small cell lung cancer (sqNSCLC), squamous head and neck cancer (sqHNSCC), esophageal cancer, and diffuse large B-cell lymphoma (DLBCL).

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"Our Phase 2 advancement of CX-2029 against the previously undruggable target CD71 marks a major milestone in our broadening clinical pipeline and highlights the progress we continue to make in applying our Probody platform to unique therapeutic opportunities in cancer," said Amy Peterson, MD, chief development officer of CytomX Therapeutics. "These expansion cohorts build on our Phase 1 clinical experience with CX-2029 in which we achieved meaningful therapeutic activity for this first-in-class drug candidate, setting the stage for Phase 2 exploration of its potential."

This open-label, multi-center Phase 2 cohort expansion study (NCT003543813) will enroll approximately 25 evaluable patients in each of the cohorts and assess the efficacy and tolerability of 3 mg/kg of CX-2029 administered every three weeks. The primary objective is overall response rate (ORR) with secondary objectives evaluating safety and tolerability. CytomX anticipates initial data from this study in late 2021.

About the CytomX and AbbVie Collaboration

In April 2016, AbbVie and CytomX entered into a Co-Development and Licensing Agreement under which the two companies are co-developing CX-2029, a Probody drug conjugate against CD71 conjugated to the cytotoxic payload MMAE. CD71, also known as the transferrin receptor 1 ("TfR1"), is a cell surface protein essential for iron uptake in dividing cells. CD71 is highly expressed in a number of solid and hematologic cancers and has attractive molecular properties for efficient delivery of cytotoxic payloads to tumor cells. CD71 has high potential as an anti-cancer target but is widely considered undruggable due to its presence on most dividing healthy cells. CX-2029 is designed to potentially create a therapeutic window for this novel target.

Under the agreement, CytomX is responsible for clinical development up to initial clinical proof of concept. AbbVie will lead late-stage clinical development and global commercial activities with CytomX eligible to receive up to $390 million in development, regulatory and commercial milestone payments, pending the achievement of pre-determined outcomes. In addition, CytomX is eligible to receive a profit share in the U.S. and tiered double-digit royalties on net product sales outside of the U.S. with CytomX retaining an option to co-promote in the U.S. In the first quarter of 2020, CytomX earned a $40 million milestone payment from AbbVie following the achievement of pre-specified criteria for the dose escalation phase of the ongoing Phase 1/2 clinical trial (NCT003543813).

On November 5, 2020 Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, reported recent business highlights and financial results for the third quarter ended September 30, 2020 (Press release, Magenta Therapeutics, NOV 5, 2020, View Source [SID1234570003]).

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"Magenta showed steady progress throughout the third quarter, bringing aboard new scientific leadership in Lisa Olson to broaden our technical expertise across research and discovery. We continued to progress both clinically and preclinically, showcasing findings at the European Society for Blood and Marrow Transplantation annual meeting," said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta Therapeutics. "We are driven by what’s ahead, including presentations at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, and into 2021, and the anticipation of clinical data in our conditioning and mobilization programs."

Recent Program Updates:

Mobilization –

By the end of 2020, Magenta plans to initiate multiple Phase 2 clinical trials of MGTA-145, the Company’s first-line stem cell mobilization agent. These trials, including both allogeneic and autologous transplant settings across multiple diseases, are intended to evaluate mobilization and collection of functional hematopoietic stem cells ("HSCs") and engraftment of these cells in patients after transplant. The MGTA-145 Phase 1 trial in healthy volunteers was completed earlier this year and met all primary and secondary endpoints.
Magenta presented data from its Phase 1 trial of MGTA-145 at the European Society for Blood and Marrow Transplantation ("EBMT") annual meeting, held August 29 to September 1, 2020. These data provide further confirmation that MGTA-145, in combination with plerixafor, enables the same-day mobilization and collection of high numbers of functional HSCs for transplant.
Magenta will present data from the MGTA-145 program at the upcoming American College of Rheumatology Convergence 2020, to be held November 5th through 9th, 2020 and the American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") annual meeting, to be held December 5th through 8th, 2020.
Conditioning –

MGTA-117, Magenta’s clinical candidate for antibody drug conjugate ("ADC")-based conditioning for stem cell transplant and gene therapy and Magenta’s most advanced conditioning program, is on track with IND-enabling studies ongoing and progressing in GMP manufacturing. Magenta expects to generate initial clinical data in 2021.
Magenta has identified a lead antibody for its CD45-ADC program for blood and immune system reset and preclinical work continues to advance.
Magenta presented two sets of preclinical data on its CD45-ADC program at the EBMT annual meeting. The first abstract showed that a single dose of CD45-ADC removed disease-causing T-cells, was well tolerated and enabled successful immune reset to halt disease progression. In the second study, the data demonstrate that a single dose of its CD45-ADC is fully myeloablative and enables complete chimerism in a full mismatch allogeneic hematopoietic stem cell transplant, potently and safely enabling immune reset.
Magenta will also present data on its MGTA-117 and CD45-ADC conditioning programs at the ASH (Free ASH Whitepaper) annual meeting in December 2020.
Recent Business Highlights:

In September 2020, Magenta announced it was named as co-recipient along with the University of Southern California, University of Washington and Fred Hutchinson Cancer Research Center, Harvard University and Massachusetts General Hospital, and the Ragon Institute of a multi-project, interdisciplinary U19 grant from the National Institutes of Health (NIH) to explore the use of novel targeted conditioning agents with gene editing approaches to advance research in a cure for HIV.

Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2020, were $161.7 million, compared to $145.7 million as of December 31, 2019. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations and capital expenditures into the second half of 2022.

Research and Development Expenses: Research and development expenses were $11.8 million in the third quarter of 2020, compared to $16.5 million in the third quarter of 2019. The decrease was driven primarily by lower clinical trial costs for the MGTA-145 Phase 1 clinical trials which were completed in the first quarter of 2020, decreased preclinical costs for manufacturing related to the conditioning programs and lower manufacturing and clinical trial costs due to the discontinuance of enrollment in the Phase 2 trial of MGTA-456 in inherited metabolic diseases in June 2020.

General and Administrative Expenses: General and administrative expenses were $6.6 million for the third quarter of 2020, compared to $6.1 million for the third quarter of 2019. The increase was primarily due to an increase in personnel costs associated with the growth of the Company.

Net Loss: Net loss was $17.7 million for the third quarter of 2020, compared to net loss of $21.0 million for the third quarter of 2019.