On November 5, 2020 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq: RDUS), reported its financial and operating results for the third quarter ended September 30, 2020, and provided a business update (Press release, Radius, NOV 5, 2020, View Source [SID1234570009]).

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"Over the past few months, we have made reasonable progress within the current business," commented Kelly Martin, CEO of Radius. Martin commented further that, "In the months ahead, we will focus on improving the performance of TYMLOS in the U.S. market, completing the three pivotal trials in a high quality manner, and constructing an attractive equity story for current or future shareholders."

Selected Highlights:

Q3 2020 U.S. net sales of TYMLOS were $50.4 million, an 8% year-over-year increase over Q3 2019.

Year-to-date 2020 net product revenue of $148 million vs. year-to-date of $117 million is a growth of 26%.

U.S. TYMLOS "Net New Patients" grew at 7+% in September vs. previous 3-month moving average.
º October Net New Patients showed 10 % growth vs. previous 3-month moving average.
º AACE guidelines include abaloparatide in the treatment recommendations as an initial therapy for postmenopausal osteoporosis patients with a recent fracture.
º Commercial business market segmentation and reengineering remains a central focus.
º Progress on implementation of a streamlined and institutional distribution channel strategy nearly complete.

Clinical Development
º ATOM study, evaluating abaloparatide for use in osteoporotic men at high risk for fracture, completed final recruitment of 228 patients.
º wearABLe study, evaluating the effects on bone mineral density of abaloparatide delivered via a novel transdermal system, completed final recruitment of 511 patients.
º EMERALD study, with our partner, Menarini Group, evaluating use of elacestrant to treat ER+/HER2- advanced or metastatic breast cancer completed final recruitment of 478 patients.
º Histomorphometry Phase 2 Study: data presented at American Society for Bone and Mineral Research (ASBMR) in September. Assessed early effect of abaloparatide at the tissue level. Results demonstrated significant increases in bone formation after three months in postmenopausal women with osteoporosis.
Japan: Pivotal Phase III trial of abaloparatide injection to treat both men and postmenopausal women with high risk of fracture by Radius partner, Teijin Pharma, achieved primary endpoint. Japan market progression remains on track.

Europe: seeking guidance and clarity regarding possible regulatory re-submission.
Financial Metric:

End of Q3 2020 total cash balance at $126 million
Q3 vs. Q2, 2020 cash burn approximately zero
Note: in Q3, Radius received a one-time payment from Menarini of $30M
Third Quarter 2020 Financial Results

Three Months Ended September 30, 2020

For the three months ended September 30, 2020, Radius reported a net loss of $6.3 million, or $0.14 per share, compared to a net loss of $30.0 million, or $0.65 per share, for the three months ended September 30, 2019.

For the three months ended September 30, 2020, non-GAAP adjusted net income, which excludes expenses related to stock-based compensation, restructuring plans, depreciation, non-cash interest obligations under debt obligations, impairment of operating lease right of use assets, and amortization of intangible assets, was $7.0 million, or $0.15 per share, compared to non-GAAP adjusted net loss of $20.4 million, or $0.44 per share, for the three months ended September 30, 2019.

For the three months ended September 30, 2020, TYMLOS net product revenues were $50.4 million compared to approximately $46.8 million for the three months ended September 30, 2019.

For the three months ended September 30, 2020, research and development expense was $39.5 million compared to $31.8 million for the three months ended September 30, 2019, an increase of $7.7 million, or 24%. This increase was primarily driven by a $11.5 million increase in abaloparatide transdermal system program costs. This increase was primarily offset by a $2.2 million decrease in elacestrant program costs, which is comprised of a $13.2 million increase in gross program expenses offset by $15.4 million of billed reimbursable expenses. We will be reimbursed for the costs incurred in connection with the elacestrant project pursuant to the terms of the TSA with Berlin-Chemie, under which the Company will perform certain services for Berlin-Chemie related to the EMERALD Phase 3 monotherapy study until the earlier of the completion of the contemplated services or the filing with the FDA of a NDA for elacestrant.

For the three months ended September 30, 2020, selling, general and administrative expenses were $33.7 million compared to $35.6 million for the three months ended September 30, 2019, a decrease of $1.9 million, or 5%. This decrease was primarily the result of a $0.8 million decrease in travel and entertainment expenses, a $2.3 million decrease in professional support costs, a $0.5 million decrease in compensation cost, and a $0.1 million decrease in other operating costs. These decreases were partially offset by a $1.8 million increase in occupancy and depreciation costs.

Nine Months Ended September 30, 2020

For the nine months ended September 30, 2020, Radius reported a net loss of $87.8 million, or $1.89 per share, compared to a net loss of $108.3 million, or $2.36 per share, for the nine months ended September 30, 2019.

For the nine months ended September 30, 2020, non-GAAP adjusted net loss, which excludes expenses related to stock-based compensation, restructuring plans, depreciation, non-cash interest obligations under debt obligations, impairment of operating lease right of use assets, and amortization of intangible assets, was $51.6 million, or $1.11 per share, compared to non-GAAP adjusted net loss of $77.6 million, or $1.69 per share, for the nine months ended September 30, 2019.

For the nine months ended September 30, 2020, TYMLOS net product revenues were $148.5 million compared to approximately $117.7 million for the nine months ended September 30, 2019.

For the nine months ended September 30, 2020, research and development expense was $123.3 million compared to $82.2 million for the nine months ended September 30, 2019, an increase of $41.1 million, or 50%. This increase was primarily driven by a $36.5 million increase in abaloparatide transdermal system project costs, and a $6.4 million increase in project costs for elacestrant. These increases were partially offset by a $1.0 million decrease in RAD140 project costs. We will be reimbursed for the costs incurred in connection with the elacestrant project pursuant to the terms of the TSA with Berlin-Chemie, under which the Company will perform certain services for Berlin-Chemie related to the EMERALD Phase 3 monotherapy study until the earlier of the completion of the contemplated services or the filing with the FDA of a NDA for elacestrant.

For the nine months ended September 30, 2020, selling, general and administrative expenses were $108.4 million compared to $116.9 million for the nine months ended September 30, 2019, a decrease of $8.6 million, or 7%. This decrease was primarily the result of a $6.3 million decrease in professional fees, a $3.3 million decrease in travel and entertainment expenses and a $0.3 million decrease in other operating expenses. These decreases were offset by a $0.3 million increase in compensation related expenses and an $1.0 million increase in occupancy and depreciation.

As of September 30, 2020, Radius had $126.3 million in cash, cash equivalents, restricted cash, and marketable securities. Based upon our cash, cash equivalents and marketable securities balance as of September 30, 2020, we believe that, prior to the consideration of potential proceeds from partnering and/or collaboration activities, we have sufficient capital to fund our development plans, U.S. commercial and other operational activities for at least twelve months from the date of this press release.

Webcast and Conference Call

In connection with today’s reporting of Third Quarter 2020 Financial Results, Radius will host a conference call and live audio webcast at 8:30 a.m. ET today, November 5, 2020, to review the commercial, research and development, and financial highlights and provide a Company update.

A live audio webcast of the call can be accessed from the Investors section of the Company’s website, www.radiuspharm.com. The full text of the announcement and financial results will also be available on the Company’s website.

For those unable to participate in the conference call or webcast, a replay will be available on Thursday, November 5, 2020 at 11:30 a.m. ET and will be archived on the Company’s website for 90 days. To access the replay, dial (855) 859-2056 for U.S. or (404) 537-3406 for International, using conference ID number 9147422.

On November 5, 2020 Nimbus Therapeutics, a biotechnology company designing breakthrough medicines through structure-based drug discovery and development, reported that it will host its second in a series of virtual seminars highlighting the latest data from its HPK1 program (Press release, Nimbus Therapeutics, NOV 5, 2020, View Source [SID1234570008]). The seminar, titled "Novel, Potent, Selective HPK1 Inhibitors Enhance Immune Activation to Inhibit Tumor Growth," will take place on Tuesday, Nov. 17, from 11 a.m. to 12 p.m. ET.

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Nimbus’ scientific leadership will present new preclinical data on the anti-tumor immune activity of its small-molecule HPK1 inhibitors. These data are featured in recent poster presentations at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium and the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting.

If you wish to attend the live seminar, please pre-register at: Nimbus HPK1 Seminar II. A replay of the webcast will be available at this link after the event.

A recording of Nimbus’ previous HPK1 seminar can be accessed at https://bit.ly/NimbusHPK1Seminar

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