On April 26, 2023 Orbital Therapeutics, a company focused on enhancing global health by unleashing the full potential of RNA medicines to treat human disease, reported the successful closing of $270 million Series A financing. The Series A raise was led by ARCH Venture Partners, with participation from initial investors a16z Bio + Health and Newpath Partners, and new investors Abu Dhabi Growth Fund (ADG), Redmile Group, Exor N.V., Invus, Moore Strategic Ventures, iGlobe Platinum Fund Group, Casdin Capital, Agent Capital, Alexandria Venture Investments, Rellim Capital Management, Heritage Medical Systems, and other undisclosed investors (Press release, Orbital Therapeutics, APR 26, 2023, View Source [SID1234630515]).

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Orbital Therapeutics aims to expand the applicability of RNA-based medicines across a range of human diseases, including in the areas of next-generation vaccines, immunomodulation, and protein replacement. The company is building a first-in-kind RNA platform that integrates both established and emerging RNA technologies and delivery mechanisms. This platform is designed to extend the durability of Orbital’s novel RNA therapeutics, while also expanding their delivery to a wide range of cell types and tissues.

"In recent years, we have only just started to see the expansive potential RNA-based therapeutics may offer to the future of medicine and how we treat disease," said Giuseppe Ciaramella, Ph.D., Orbital co-founder and CEO. "Since our founding, we have been diligently focused on advancing our platform of RNA technologies to elucidate the most promising opportunities with our initial programs so we may reach patients as efficiently as possible. We are thrilled to have the backing of such an exceptional group of investors, whose support further enables the growth of our organization and the meaningful expansion of our platform and portfolio."

"Few companies can possess the know-how, technology and biologic insights to deliver on the full breadth of opportunity that RNA medicines could provide for patients. Orbital was founded and has been built to be that special company," said Kristina Burow, managing partner at ARCH Venture Partners, co-founder and board director of Orbital. "We are pleased to partner with Orbital’s team of industry leaders and visionaries in drug development to help them achieve a bold mission of bringing forward the next generation of RNA-based medicines."

In addition to the financing, Orbital announced the expansion of its leadership team with the appointments of Niru Subramanian as chief operating officer and Jonathan Piazza as chief financial officer, who join Gilles Besin, Ph.D., chief scientific officer, and Dr. Ciaramella on the company’s executive leadership team.

Dr. Ciaramella added, "Key to our long-term success is having a team that not only brings experience, but also passion and drive for the work we are doing. Niru has an extensive background in driving corporate strategy and portfolio development for successful biotech and pharmaceutical companies, which is complemented by Jonathan’s successful history of leading financial organizations within the industry and guiding several strategic transactions while at top investment banks. Their collective expertise and dedication to create a company that can advance important new medicines for patients makes them ideal additions to our team, and they already have made tremendous early contributions to our company’s growth and capital raise. We are thrilled to have them on board."

Niru Subramanian, Chief Operating Officer

Ms. Subramanian brings 25 years of business and corporate development experience, including building new strategies and capabilities for high-growth organizations, leading multifunctional teams, managing partnerships and M&A transactions, and directing portfolio planning, alliance management, finance and operations. She most recently served as chief operating officer at Rheos Medicines, and prior to that, was senior vice president, corporate development & business planning at Agios Pharmaceuticals, where she led several key initiatives including the $2 billion sale of Agios’ oncology business in December 2020. Earlier, she held several roles at Novartis Vaccines, including as vice president, head of Influenza Global Programs and at Novartis as vice president, Vaccines Business transition lead following the divestment of the business unit. Earlier in her career, she worked at The Boston Consulting Group in their healthcare practice area advising large and small biopharma companies on a variety of strategic and operational issues. Ms. Subramanian received a BS in computer science and engineering from the University of Pennsylvania and an MBA from the Harvard Business School.

Jonathan Piazza, Chief Financial Officer

Mr. Piazza is a seasoned biotech executive and finance professional with more than 20 years of experience in biotech, healthcare investment banking, finance, and large pharma roles. He most recently served as CFO of Silverback Therapeutics and helped orchestrate its IPO, and later its reverse merger with ARS Pharma. As a healthcare banker at Goldman Sachs, Barclays Capital, and Lehman Brothers, he has advised on approximately $100 billion in biopharma and life sciences financing and strategic transactions across both large and small companies. Previously, he was a financial advisor at Smith Barney (now Morgan Stanley Smith Barney), and prior to that, he worked in engineering, development, and international marketing disciplines at Abbott Laboratories across multiple divisions of the company. Mr. Piazza earned a BS in chemical engineering from The Ohio State University and an MBA from the Haas School of Business at the University of California at Berkeley.

On April 26, 2023 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the first quarter ended March 31, 2023 (Press release, BioMarin, APR 26, 2023, View Source,-Including-15-Year-over-year-Growth-of-Total-Revenues [SID1234630514]).

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"We begin 2023 by delivering double-digit revenue growth in the first quarter driven by continued strong, global uptake of VOXZOGO, solid growth of our enzyme business and ongoing focus on operational excellence. In addition to record-breaking revenue, we were also pleased with continued profitability in the quarter," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin.

Mr. Bienaimé continued, "During the quarter, with respect to ROCTAVIAN, we were pleased with progress made in Germany with physicians and those with severe hemophilia A interested in treatment. Specifically, we were encouraged by feedback from German hematologists interested in ROCTAVIAN, as well as the number of people pursuing antibody testing to determine their eligibility for treatment. To further support the hemophilia A community in Germany, today we announce the decision to work directly with the primary, public health insurer to facilitate access to ROCTAVIAN, rather than pursue additional Outcomes Based Agreements with sub-insurers, following months of complex negotiations. In the United States, we are actively preparing for the launch of ROCTAVIAN and are working with multiple key treatment centers to prepare for potential approval in June. Total revenue growth is tracking to plan, as supported by VOXZOGO accessibility to more families seeking treatment, our solid enzyme franchise, potential U.S. approval of ROCTAVIAN on the horizon and good progress on the commercial launch of ROCTAVIAN in Europe. 2023 is off to a great start and we are optimistic about the outlook ahead."

Financial Highlights:

Total Revenues for the first quarter of 2023 were $596.4 million, an increase of 15% compared to the same period in 2022. The increase in Total Revenues was primarily attributed to the following:
Higher VOXZOGO commercial sales due to continued global market expansion and rapid patient uptake following regulatory approvals in late 2021 and early 2022, and
Higher ALDURAZYME product revenues primarily due to the timing of order fulfillment to Sanofi. BioMarin ALDURAZYME revenues are driven by the timing of when the product is released and control is transferred to Sanofi.
GAAP Net Income decreased to $50.9 million for the first quarter of 2023 compared to $120.8 million for the same period in 2022. The decreased net income was primarily due to the absence of the $89.0 million gain, net of tax, recognized in the first quarter of 2022 upon the sale to a third party the PRV the Company received in connection with FDA approval of VOXZOGO partially offset by an increase in gross profit.
Non-GAAP Income increased to $115.8 million for the first quarter of 2023 compared to Non-GAAP Income of $98.8 million for the same period in 2022 driven by higher gross profit due to increased sales volume partially offset by increased selling, general and administrative (SG&A) expenses to support the anticipated commercial launch of ROCTAVIAN, employee-related costs and other strategic initiatives.
Recent Product Approvals and Launches (VOXZOGO and ROCTAVIAN)

As of the end of March 2023, approximately 1,500 children with achondroplasia were being treated with VOXZOGO across 35 active markets. In the first quarter, VOXZOGO growth accelerated in Japan and Brazil, followed by other markets, respectively. Based on these trends, today BioMarin updated full-year 2023 VOXZOGO guidance to between $380 million and $430 million. VOXZOGO is currently approved for the treatment of children 2 years old and older in Europe, for children 5 years old and older in the U.S., and approved for all ages from birth in Japan.
In Europe, BioMarin is making progress in Germany, France and Italy to facilitate access to ROCTAVIAN. In Germany, 18 people with severe hemophilia A have completed antibody testing to determine their seroprevalence to AAV5. On reimbursement in Germany, BioMarin is now working directly with the National Association of Statuary Health Insurance Funds (GKV) to finalize access to ROCTAVIAN, due to the complexity to reach final terms on the remaining Outcomes Based Agreements (OBAs) with sub-insurers. GKV is the primary provider of public health insurance, supplying coverage to approximately 90% of the German population. At present, people in Germany with severe hemophilia A, who are eligible for treatment with ROCTAVIAN, can access treatment through either Named Patient authorizations or previously secured OBAs. In France and Italy, BioMarin is working directly with the single public insurance funds in each country to secure reimbursement and access to ROCTAVIAN, which is expected later in 2023.
In March, the FDA extended review of the Biologics License Application (BLA) for ROCTAVIAN gene therapy for adults with severe hemophilia A. The FDA determined that the submission in the first quarter of the three-year data analysis from the ongoing Phase 3 GENEr8-1 study, as requested by the FDA, constituted a Major Amendment due to the substantial amount of additional data and set a new PDUFA target action date of June 30, 2023.
Based on the updated PDUFA target action date in the U.S., as well as the transition to working directly with GKV, BioMarin today lowered full-year 2023 ROCTAVIAN guidance to between $50 million and $150 million. This updated ROCTAVIAN estimated revenue range assumes contributions from Europe, the U.S., and other markets, including Named Patient authorizations, in 2023.
Mid-stage Product Life Cycle Expansion Opportunities (VOXZOGO and ROCTAVIAN)

In the coming months in the U.S. and Europe, the Company expects to learn the outcome of its request to expand VOXZOGO access to younger age groups, based on favorable results from a Phase 2 study in infants and young children. If age expansions are accepted, more than 1,000 additional children will be eligible for VOXZOGO treatment in the U.S. and Europe.
BioMarin is engaged in discussions with health authorities concerning the opportunity to leverage VOXZOGO, a natural regulator of bone growth, in other conditions characterized by impaired bone growth.
Product expansion opportunities with ROCTAVIAN continue, including a clinical study investigating ROCTAVIAN treatment in those with active or prior inhibitors and continued exploration of methods of administering ROCTAVIAN in people with pre-existing antibodies against AAV5.
Earlier-stage Development Portfolio (BMN 255, BMN 331, BMN 351, BMN 349, BMN 293 (DiNA-001))

BioMarin plans to showcase progress across its earlier-stage development pipeline at R&D Day in New York City on September 12, 2023. Invitations to the event will be circulated in June.
BMN 255 for hyperoxaluria in chronic liver disease: The Company has concluded the multi-ascending dose study with BMN 255 in healthy human volunteers. In January 2023, BioMarin shared early data that demonstrated a rapid and potent increase in plasma glycolate following treatment with BMN 255. Oral daily dosing at all tested levels for 14 days was safe and showed sustained elevations of plasma glycolate, which is predicted to have a profound reduction in oxalate excretion in patients. BioMarin now plans to initiate and enroll an expanded study in patients with chronic liver disease and hyperoxaluria in 2023. The Company believes the availability of a potent, orally bioavailable, small molecule like BMN 255 may be able to significantly reduce disease and treatment burden in a patient population with significant unmet need.
BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): Dosing continues in the Phase 1/2 HAERMONY study to evaluate BMN 331, an investigational AAV5-mediated gene therapy for people living with HAE. In January 2023, BioMarin shared that the first participant treated with the 6e13vg/kg dose demonstrated C1-Inhibitor levels that were approaching the therapeutically relevant range. In March, the second sentinel participant was safely dosed at 6e13vg/kg.
BMN 351 for Duchenne Muscular Dystrophy (DMD): Investigational New Drug application (IND)-enabling studies continue with BMN 351, an antisense oligonucleotide therapy for individuals with exon 51-skip-amenable DMD. BMN 351 was developed using familiar chemistry and superior biology, by targeting a novel, splice enhancer site demonstrating improved binding affinity and tolerability in preclinical models. Preclinical data suggest that restored expression of near-full-length dystrophin protein at levels of up to 40% will convert phenotypes from rapid loss to durable preservation of strength and ambulation. BioMarin is working towards initiating clinical studies with BMN 351 in 2023.
BMN 349 for alpha-1 antitrypsin deficiency: Preclinical studies have demonstrated that BMN 349 is an orally bioavailable, small molecule that preferentially sequesters mutant protein, preventing polymerization in liver cells that drive the progressive liver disease form of the illness. In preclinical studies BMN 349 is titratable to effect, with rapid onset and high potency. Preclinical results have strong implications for potential improvement of current management, particularly for severe liver disease requiring rapid action. IND enabling studies are underway and BioMarin’s goal is to submit an IND for BMN 349 in the second half of 2023.
BMN 293 (formerly DiNA-001) for MYBPC3 hypertrophic cardiomyopathy (HCM): Preclinical studies are underway with BMN 293 following a collaboration announced in 2020 with DiNAQOR, a platform company that develops organ specific delivery of novel gene therapies to treat rare genetic cardiac and renal diseases. Mutations in the MYBPC3 gene are the most common cause of inherited HCM. Early investigations suggest that gene therapy-mediated gene transfer can lead to widespread expression of the gene product, cardiac myosin-binding protein C (MyBP-C), in cardiac tissue, which can normalize cardiac hypertrophy, improve relaxation kinetics and potentially alleviate functional deficits in individuals suffering from cardiomyopathy. BioMarin’s goal is to submit an IND for BMN 293 in the second half of 2023.

2023 Full-Year Financial Guidance (in millions, except % and EPS amounts) (Updated)

Item

2023 Guidance

Updated April 26, 2023

Total Revenues

$2,375

to

$2,500

Unchanged

Enzyme Product Revenues(1)

$1,700

to

$1,850

Unchanged

ROCTAVIAN Revenues

$100

to

$200

$50

to

$150

VOXZOGO Revenues

$330

to

$380

$380

to

$430

Unchanged

Gross Profit %

77.5 %

to

79 %

Unchanged

R&D % of Revenue

30 %

to

32 %

Unchanged

SG&A % of Revenue

36 %

to

38 %

Unchanged

GAAP Net Income

$155

to

$205

Unchanged

GAAP Diluted EPS

$0.78

to

$1.03

Unchanged

Non-GAAP Income

$360

to

$410

Unchanged

Non-GAAP Diluted EPS

$1.80

to

$2.05

Unchanged

(1)

Enzyme-based Products include ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ and VIMIZIM.

The full-year 2023 ROCTAVIAN revenue guidance range, provided above, represents global revenue estimates and assumes a U.S. approval in June 2023.

BioMarin will host a conference call and webcast to discuss first quarter 2023 financial results today, Wednesday, April 26, 2023 at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.

On April 26, 2023 Bicara Therapeutics, a clinical-stage biotechnology company developing dual-action biologics to elicit a potent and durable immune response, reported that it will present dose expansion data from the ongoing Phase 1/1b clinical trial of its lead investigational candidate, BCA101, in combination with pembrolizumab in patients with recurrent, metastatic head and neck squamous cell carcinoma (Press release, Bicara Therapeutics, APR 26, 2023, View Source;utm_medium=rss&utm_campaign=bicara-therapeutics-announces-oral-presentation-of-head-and-neck-cancer-dose-expansion-data-from-phase-1-1b-study-of-bca101-at-upcoming-2023-asco-annual-meeting [SID1234630513]). BCA101 is a first in class dual-action bifunctional antibody designed to inhibit EGFR and disable TGF-β directly at the tumor site. These data will be presented in an oral presentation at the upcoming 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held from June 2-6, 2023 in Chicago, IL.

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Details of the oral presentations are as follows:

Title: Dose expansion results of the bifunctional EGFR/TGF-β inhibitor BCA101 with pembrolizumab in patients with recurrent, metastatic head and neck squamous cell carcinoma.
Presenter: Glenn J. Hanna, M.D.
Abstract Number: 6005
Session Type/Title: Oral Abstract Session – Head and Neck Cancer
Date/Time: June 5, 2023 from 8:00-11:00 a.m. CT
Location: McCormick Place Convention Center

This presentation described here will be made available on the Bicara website following the conference.

On April 26, 2023 BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global biotechnology company, reported that it will present data from its broad solid tumor and hematology portfolio at the upcoming American Society of Cancer Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, June 2-6, 2023 (Press release, BeiGene, APR 26, 2023, View Source [SID1234630512]).

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"Our accepted data presentations at ASCO (Free ASCO Whitepaper) demonstrate the resolve and productivity of BeiGene’s R&D team in our drive to improve treatment outcomes and access for patients worldwide," said Lai Wang, Ph.D., Global Head of R&D at BeiGene. "We look forward to joining the oncology community in Chicago and sharing progress as we continue to advance our rich pipeline of first-in-class and potentially best-in-class programs."

Data to be presented at ASCO (Free ASCO Whitepaper) include:

Abstract Title

Abstract #

Presentation Details

Lead Author

Solid Tumors

Results from the Pivotal Phase 2b HERIZON-BTC-01 Study: Zanidatamab in Previously-treated HER2 amplified Biliary Tract Cancer

4008

Oral presentation: Gastrointestinal Cancer— Gastroesophageal, Pancreatic, and Hepatobiliary

Fri., June 2,

2:45-5:45 p.m. CT

Shubham Pant

A phase 1 study of the OX40 agonist, BGB-A445, with or without tislelizumab, an anti-PD-1 monoclonal antibody, in patients with advanced solid tumors

2574

Poster presentation: Developmental Therapeutics—Immunotherapy

Sat., June 3,

8:00-11:00 a.m. CT

Jayesh Desai

Zanidatamab, a HER2-targeted bispecific antibody, in combination with docetaxel as first-line therapy for patients with advanced HER2-positive breast cancer: updated results from a Phase Ib/II study

1044

Poster presentation: Breast Cancer—Metastatic

Sun., June 4, 8:00-11:00 a.m. CT

Xiaojia Wang

Tislelizumab versus sorafenib in first-line treatment of unresectable hepatocellular carcinoma: the RATIONALE-301 European/North American subgroup

4082

Poster presentation: Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary

Mon., June 5, 8:00-11:00 a.m. CT

Arndt Vogel

Impact of risk factors on overall survival in patients with unresectable hepatocellular carcinoma treated with first-line tislelizumab

4083

Poster presentation: Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary

Mon., June 5, 8:00-11:00 a.m. CT

Masatoshi Kudo

AdvanTIG-105: phase 1b dose-expansion study of ociperlimab + tislelizumab with chemotherapy in patients with stage IV gastric/gastroesophageal adenocarcinoma

4028

Poster presentation: Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary

Mon., June 5, 8:00-11:00 a.m. CT

Se Hyun Kim

Hematology

A phase 1 study evaluating the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of Bcl-2 inhibitor BGB-11417 in adult patients with mature B-cell malignancies

7558

Poster presentation: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia

Mon., June 5, 8:00-11:00 a.m. CT

Caixia Li

First interim analysis of a phase 1 study of zanubrutinib plus lenalidomide in patients with relapsed/refractory diffuse large B-cell lymphoma

7557

Poster presentation: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia

Mon., June 5, 8:00-11:00 a.m. CT

Huilai Zhang

Zanubrutinib plus obinutuzumab versus obinutuzumab in patients with relapsed/refractory follicular lymphoma: Updated analysis of the ROSEWOOD study

7545

Poster presentation: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia

Mon., June 5, 8:00-11:00 a.m. CT

Christopher Flowers

MAHOGANY: A phase 3 trial of zanubrutinib plus anti-CD20 antibodies vs lenalidomide plus rituximab in patients with relapsed or refractory follicular or marginal zone lymphoma

TPS7590

Poster presentation: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia

Mon., June 5, 8:00-11:00 a.m. CT

Loretta Nastoupil

Matching-adjusted indirect comparison of zanubrutinib versus ibrutinib in relapsed/refractory marginal zone lymphoma

e19527

Publication only

Catherine Thieblemont

Comparative efficacy of zanubrutinib versus rituximab in relapsed marginal zone lymphoma: matching-adjusted indirect comparison

e19526

Publication only

Catherine Thieblemont

Real-world treatment patterns of Bruton tyrosine kinase inhibitors in patients with mantle cell lymphoma in community oncology practices in the United States

e19525

Publication only

Bijal Shah

On April 26, 2023 Artiva Biotherapeutics, Inc., a clinical stage company whose mission is to deliver highly effective, off-the-shelf, allogeneic natural killer (NK) cell-based therapies, reported that a poster describing initial data from the Phase 1/2 trial of AB-101 will be presented at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place June 2-6 in Chicago (Press release, Artiva Biotherapeutics, APR 26, 2023, View Source [SID1234630511]). AB-101 is a non-genetically modified, cord blood-derived, allogeneic, cryopreserved, ADCC-enhancing NK cell being investigated in combination with rituximab for the treatment of relapsed/refractory B-cell non-Hodgkin lymphoma.

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Details of the presentation are as follows:

Title: AB-101, an allogeneic, non–genetically modified, natural killer (NK) cell therapy, evaluated as monotherapy or in combination with rituximab in R/R non-Hodgkin lymphoma.
Abstract: 7529
Session: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia
Session Day and Time: Monday, June 5, 8 am to 11 am CT
Presenter: Thorsten Graef, M.D., Chief Medical Officer, Artiva Biotherapeutics
About AB-101

AB-101 is a non-genetically modified, cord blood-derived, allogeneic, cryopreserved, ADCC-enhancing NK cell therapy candidate for use in combination with monoclonal antibodies or innate-cell engagers in the out-patient setting. Artiva selects cord blood units with the high affinity variant of the receptor CD16 and a KIR-B haplotype for enhanced product activity. Using the Company’s AlloNKTM platform, Artiva can generate thousands of doses of pure, cryopreserved, infusion-ready NK cells from a single umbilical cord blood unit while retaining the high and consistent expression of CD16 and other tumor-engaging receptors, without the need for engineering. We believe this makes AB-101 an optimal adjunct therapy to targeted, ADCC-mechanistic biologics.

Artiva is conducting a Phase 1/2 multicenter clinical trial (ClinicalTrials.gov Identifier: NCT04673617) to assess the safety and clinical activity of AB-101 alone and in combination with the anti-CD20 monoclonal antibody, rituximab, in patients with relapsed or refractory B-cell-non-Hodgkin lymphoma (NHL) who have progressed beyond two or more prior lines of therapy including CAR-T therapy. This study is progressing at multiple clinical sites across the U.S., and AB-101 is administered weekly in the out-patient setting over one-month cycles and with up to four cycles to assess therapeutic efficacy and durability. Artiva is also collaborating with Affimed N.V. in developing a combination therapy, comprised of AB-101 and the Innate Cell Engager AFM13, for the treatment of patients with relapsed/refractory CD30-positive lymphomas.