On October 23, 2019 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported a business update that will be discussed in more detail on the company’s investor and analyst call to be held today at 10am Eastern Time / 7am Pacific Time (Press release, Rigel, OCT 23, 2019, View Source [SID1234542441]).

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"This is an exciting time for Rigel with significant advancements in all segments of our business," said Raul Rodriguez, Rigel’s president and CEO. "We continue to grow sales of TAVALISSE in the U.S. and are making substantial strides in expanding our pipeline. Our clinical development efforts will be led by our newly appointed chief medical officer, Dr. Wolfgang Dummer. We are excited to leverage his experience and depth of knowledge as we continue to pursue our clinical development goals."

Business Update Highlights

TAVALISSE Revenues Increase

Preliminary estimates indicate that TAVALISSE (fostamatinib disodium hexahydrate) net product sales continued to achieve double digit quarter over quarter growth, increasing 15% to $11.7 million from $10.2 million in the second quarter of 2019. This information is preliminary, has not been audited and is subject to change upon completion of Rigel’s closing procedures.

IRAK1/4 Program Shows Proof-of-Mechanism in Humans

Rigel completed a Phase 1 clinical trial of R835, an interleukin-1 receptor-associated kinase 1/4 (IRAK 1/4) inhibitor. In addition to positive tolerability and pharmacokinetic data, R835 showed consistent inhibition of cytokine production in an LPS (lipopolysaccharide) challenge which was designed to gauge the molecule’s impact on inflammatory stimulation.

New RIP1 Inhibitor Program

For the first time today, Rigel announced its new receptor-interacting protein kinase (RIP1) inhibitor program. The lead molecule, R552, has initiated a Phase 1 clinical trial. RIP1 is believed to be a key driver of necroptosis which is implicated in a broad range of key inflammatory cellular processes including cell death and cytokine production.

Appointment of Wolfgang Dummer, MD, PhD as CMO

The Company is pleased to announce the appointment of Wolfgang Dummer, MD, PhD to the role of Chief Medical Officer. Dr. Dummer has more than 20 years of clinical and drug development experience at world class institutions, as well as an extensive academic history. Most recently, he served as Chief Medical Officer at Aridis Pharmaceuticals, Inc. where he was responsible for overseeing all aspects of drug development in the field of antimicrobial immunotherapy. Prior to that, he served as Vice President of Clinical Development at BioMarin Pharmaceutical Inc., where he led the development of a deep rare disease pipeline, including the company’s leading marketed product, Vimizim (elosulfase alpha). Prior to Biomarin, Dr. Dummer served for 11 years in capacities of increasing importance in Clinical Research and Development at Genentech, Inc. (now part of Roche), overseeing numerous programs, including Rituximab. Dr. Dummer is a board-certified clinical dermatologist and allergist/immunologist. Over the course of his career, he has published more than 50 peer reviewed journal articles and has more than 40 abstracts, presentations, and book contributions.

Business Update Conference Call

As previously announced, Rigel will host a conference call today to provide a business update. Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call and accompanying slides will also be webcast live and can be accessed from Rigel’s website at www.rigel.com. The webcast will be archived and available for replay for 90 days after the call via the Rigel website.

About ITP

In patients with ITP (immune thrombocytopenia), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs) and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About R8351

R835 is an oral investigational candidate that is a potent and selective inhibitor of IRAK1 and IRAK4 shown preclinically to block inflammatory cytokine production in response to toll-like receptor (TLR) and the interleukin-1 receptor (IL-1R) family signaling. TLRs and IL-1Rs play a critical role in the innate immune response and dysregulation of these pathways can lead to a variety of inflammatory conditions. Dysregulation of the TLR and IL-1R pathways may be associated with a variety of inflammatory conditions including psoriasis, rheumatoid arthritis, lupus and gout (among others).

About R5521

R552 is an investigational candidate that is a receptor-interacting protein kinase (RIP1) inhibitor. RIP1 is believed to play a critical role in necroptosis, a type of regulated cell death. In necroptosis, cells rupture leading to the dispersion of cell contents, which signals an immune response and enhances inflammation. It is implicated in a broad range of key inflammatory cellular processes including cell death and cytokine production. In preclinical studies, R552 showed prevention of joint and skin inflammation in a RIP1-mediated murine model of inflammation and tissue damage.

About TAVALISSE
Indication

TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information
Warnings and Precautions

Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.

Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to >3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.

Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (>Grade 3), interrupt, reduce dose or discontinue TAVALISSE.

Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.

TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.

Drug Interactions

Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.

It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.

Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.

Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.

Adverse Reactions

Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).

Common adverse reactions (>5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.

On October 23, 2019 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that it will host a conference call at 1:30 p.m. PST/4:30 p.m. EST on Wednesday, November 6, 2019 following release of its third quarter 2019 financial results (Press release, Puma Biotechnology, OCT 23, 2019, https://investor.pumabiotechnology.com/press-release/featured-release/puma-biotechnology-host-conference-call-discuss-third-quarter-financi [SID1234542440]).

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The call may be accessed by dialing 1-877-709-8150 (domestic) or 1-201-689-8354 (international). Please dial in at least ten minutes in advance and inform the operator that you would like to join the "Puma Biotechnology Conference Call." A live webcast of the conference call and presentation slides may be accessed on the Investors section of the Puma Biotechnology website at View Source A replay of the call will be available approximately one hour after completion of the call and will be archived on Puma’s website for 90 days.

On October 23, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for the treatment of cancer, reported that an abstract containing data from the Company’s Phase 2 clinical trial of tipifarnib in HRAS mutant head and neck squamous cell carcinomas (HNSCC) has been selected for oral presentation at the upcoming AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), which will be held October 26-30, 2019 at the Hynes Convention Center in Boston (Press release, Kura Oncology, OCT 23, 2019, View Source [SID1234542439]).

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Nearly 500 abstracts were considered for this year’s program and just 10 were selected as short-talks by the Scientific Committee Cochairs. As a result of the selection, the previously announced poster presentation related to the Phase 2 clinical trial of tipifarnib in HRAS mutant HNSCC has been rescheduled to take place following the oral presentation. Details of the oral and poster presentations, entitled "Preliminary results from a phase 2 trial of tipifarnib in squamous cell carcinomas (SCCs) with HRAS mutations" (abstract A087), are as follows:

Oral Presentation
Session Title: Spotlight on Proffered Papers 3: Targeting RAS Mutant Cancers
Session Date: Tuesday, October 29, 2019
Session Start Time: 11:50 a.m. ET
Session End Time: 12:30 p.m. ET
Location: Level 3 – Ballroom AB

Poster Presentation
Session Title: Poster Session C: Clinical Trials 2
Session Date: Tuesday, October 29, 2019
Session Start Time: 12:30 p.m. ET
Session End Time: 4:00 p.m. ET
Location: Level 2 – Hall D

The data are embargoed until the beginning of the Spotlight on Proffered Papers 3: Targeting RAS Mutant Cancers session at 11:50 a.m. ET on Tuesday, October 29, 2019. A copy of the oral and poster presentations will be available at www.kuraoncology.com following presentation at the meeting.

On October 23, 2019 Elicio Therapeutics, a next generation immuno-oncology company, reported that Christopher Haqq M.D., Ph.D. has joined Elicio as Executive Vice President, Head of Research and Development, and Chief Medical Officer (Press release, Elicio Therapeutics, OCT 23, 2019, View Source [SID1234542438]).

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"Chris is an accomplished physician-scientist who brings to Elicio over 20 years of translational drug development and leadership in large and small biotechnology companies across T cell immunotherapy, biologic, and small molecule modalities," commented Elicio CEO Robert Connelly. "We expect Chris’ knowledge of the impact of T-cell expansion, persistence and trafficking in immuno-oncology will dovetail with the unique lymph node targeting ability of Elicio’s Amphiphile platform."

Before joining Elicio, Dr. Haqq was the first employee and Chief Medical Officer of Atara Biotherapeutics, and later its Chief Scientific Officer, where he was the architect for an innovative pipeline including EBV specific T-cell product candidates for oncology and autoimmune disease, and a next generation off the shelf CAR-T cells for solid tumors. Earlier at Cougar Biotechnology and Janssen, Dr. Haqq was the lead clinician for a pivotal prostate cancer study leading to market approval for Zytiga (abiraterone acetate) and he led early development studies at Amgen.

Dr. Haqq has served as medical monitor for numerous oncology clinical trials working closely with global regulatory agencies. His teams have made successful filings for investigational new drug applications, breakthrough therapy, priority medicines, special protocol assessments and marketing approval and he has authored multiple patents and publications. Dr. Haqq initially practiced as a medical oncologist and led a translational science laboratory as an Assistant Professor in the Division of Hematology/Oncology at the University of California, San Francisco following post-graduate training as an Intern and Resident in Internal Medicine, Fellow in Medical Oncology and Fellow in Molecular Medicine. Dr. Haqq completed his M.D. and Ph.D. in Genetics at Harvard Medical School and his undergraduate training at Stanford University and the University of British Columbia.

"I am excited to lead Elicio’s strong R&D team during the transition from the lab bench to the bedside for its promising lymph node targeted immunotherapies where preclinical monotherapy and CAR-T combination show unique potential," said Dr. Haqq. "And as my family has experienced the unmet need for an effective mKRAS therapy, I am looking forward to evaluate ELI-002’s safety and efficacy in mKRAS bearing pancreatic, colorectal and lung cancers."

About the Amphiphile Platform

The Elicio Amphiphile platform enables precise targeting and delivery of immunogens and cell-therapy activators directly to the lymphatic system, the "brain center" of the immune response, to significantly amplify and enhance the body’s own system of defenses, defeat solid and hematologic cancers, and prevent their recurrence. Once in the lymph nodes, Amphiphile immunotherapies are taken up by antigen presenting cells (APC’s) to orchestrate signaling to natural or engineered immune cells in order to maximize therapeutic immune responses to disease. This strategy has been used to improve the activity of immunostimulatory agents, antigens, adjuvants, and cell-therapies that generate little to no response when used in the conventional forms. By precisely targeting these immunotherapies to the lymph nodes, Amphiphiles can unlock their full potential to generate and amplify anti-tumor immune responses. This substantially enhanced anti-tumor functionality and long-term protective memory may someday unlock the full potential of the immune response to eliminate cancer.

On October 23, 2019 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the third quarter ended September 30, 2019 (Press release, BioMarin, OCT 23, 2019, View Source [SID1234542437]).

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Total Net Product Revenues for the third of quarter 2019 increased to $450.9 million, compared to $386.3 million for the third quarter of 2018. The increase in Net Product Revenues was attributed to the following:

Vimizim Net Product Revenues increased by $40.2 million, or 33%, driven primarily by increased sales volume driven by government orders in certain Middle Eastern countries, a large order from Brazil as well as smaller orders from other Latin American countries;

Palynziq Net Product Revenues increased by $20.0 million, driven by a combination of revenue from patients achieving maintenance dosing and new patients initiating therapy in the U.S. Palynziq received approval from the U.S. Food and Drug Administration (FDA) in May 2018 and launched in the third quarter of that year. Palynziq received European Medicines Agency (EMA) approval in May 2019 and commercial sales in Europe are expected to commence in the fourth quarter of 2019;

Brineura Net Product Revenues increased by $9.9 million, or 100%, due in large part by growth in the number of patients across all regions; and

Kuvan Net Product Revenues increased by $7.3 million, or 6%, primarily driven by an increase in the number of patients in North America; partially offset by

Naglazyme Net Product Revenues decreased by $8.7 million, or 8%, primarily due to decreased sales volume driven by government ordering patterns from certain Latin American and European countries; and

Aldurazyme Net Product Revenues decreased $4.8 million, due to the timing of customer acceptance for product shipped to Genzyme in the third quarter for which no revenue was recognized as of September 30, 2019.
The increase in GAAP Net Income for the third quarter of 2019, compared to GAAP Net Loss the same period in 2018 was primarily due to the following:

increased gross profits of $51.3 million driven by increased product sales;

increased tax benefit, which is primarily attributed to quarterly fluctuations in the mix and timing of our profits and losses on a territorial basis and reversals of certain tax reserves that were no longer required, partially offset by

higher selling, general and administrative (SG&A) expense related to pre-commercialization activities for valoctocogene roxaparvovec, support of the EU commercial launch and continued U.S. expansion of Palynziq, and increased general and administrative expense primarily attributed to personnel-related costs resulting from increased headcount to support our growth; and

higher research and development (R&D) expense related to preclinical activities for BMN 307 and clinical activities for the Company’s vosoritide and valoctocogene roxaparvovec development programs, partially offset by decreased R&D expense related to Palynziq for which we began capitalizing manufacturing costs upon FDA approval in May 2018, and a decrease in tralesinidase alfa clinical manufacturing costs. R&D expenses in the quarter were consistent with 2019 guidance despite the acceleration of the valoctocogene roxaparvovec development program and subsequent activities implemented to pursue an expedited regulatory path forward.

Key Program Highlights

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Valoctocogene roxaparvovec gene therapy for hemophilia A: The European Medicines Agency (EMA) recently granted BioMarin’s request for accelerated assessment of valoctocogene roxaparvovec, for adults with severe hemophilia A. Accelerated assessment reduces the time-frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a Marketing Authorization Application (MAA) for an Advanced Therapy Medicinal Product (ATMP). Applications are eligible for accelerated assessment if the CHMP and CAT decide the product is of major interest for public health, particularly from the point of view of therapeutic innovation. Evaluating a MAA under the EMA centralized procedure can take up to 210 days, not counting clock stops when applicants are requested to provide additional information. On request, the CHMP and CAT can reduce the time-frame to 150 days if the applicant provides sufficient justification for an accelerated assessment. The decision to grant accelerated assessment has no impact on the eventual CHMP and CAT opinion on whether a marketing authorization should be granted.

On July 8, the Company announced that based on recent meetings with health authorities in the U.S. and Europe, it plans to submit marketing applications to both the FDA and the EMA in the fourth quarter of 2019 for valoctocogene roxaparvovec with the 6e13 vg/kg dose. The submissions will be based on the recently completed Phase 3 interim analysis and the updated three-year Phase 1/2 data of patients treated with valoctocogene roxaparvovec. Both submissions are expected to represent the first time a gene therapy product for any type of hemophilia indication will be reviewed for marketing authorization by health authorities.

Enrollment in the GENEr8-1 Phase 3 study is expected to be complete by R&D Day with 52-week results from the 130 subjects expected at the end of 2020. Although the trial is open label, BioMarin has implemented a data access plan designed to significantly mirror a blinded trial. This restricts the release of any ongoing data to a small group of medical personnel monitoring and managing the trial, and then, only to the extent necessary to perform their monitoring responsibilities.

The Company has chosen to cease development of the 4e13 vg/kg dose of valoctocogene roxaparvovec given the overwhelming preference by patients to be treated with the 6e13 vg/kg dose.

BioMarin intends to provide a 4 year update with the 6e13 vg/kg dose subjects and a 3 year update with the 4e13 vg/kg dose subjects from the ongoing Phase 2 study in mid-2020.

Palynziq for PKU: Palynziq, an injection to reduce blood Phe concentrations in adult patients with PKU, was added to BioMarin’s commercial product portfolio upon its U.S. approval May 2018. As of September 30, 2019, 670 patients were on reimbursed Palynziq, with an additional 153 naïve patients enrolled and awaiting their first treatment with commercial Palynziq. Of the 670 patients on therapy at the end of the third quarter, 528 were formerly naïve patients and 142 had transitioned from clinical studies. Of the 125 PKU clinics in the U.S., 97 unique clinics had at least one complete patient enrollment in the REMS program as of September 30, 2019.
On May 6, 2019, the European Commission (EC) granted marketing authorization for Palynziq at doses of up to 60 milligrams once daily, to reduce blood Phe concentrations in patients with PKU aged 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options. The Company is in the process of securing reimbursement on a country-by-country basis across the European Union and anticipates meaningful revenue contributions from this region in 2020.

Vosoritide for children with achondroplasia: The vosoritide development program includes four distinct areas of focus to support global approval, including a large contemporaneous natural history study which is underway. The global Phase 3 study is a randomized, double-blind placebo-controlled study of vosoritide in approximately 110 children with achondroplasia between the ages of 5 to 14 years. Data from this study is expected by year-end 2019.
The Company plans to share 54-month results from the ongoing Phase 2 study with vosoritide in children ages 5 to 14 years at the upcoming R&D Day planned for November 14. These data are expected to corroborate maintenance of effect at the time of anticipated marketing application submissions.

The fourth component of the Company’s global development program with vosoritide, includes a large Phase 2 study in infants and young children (newborn to 60 months old) with achondroplasia, to determine the impact of treatment in this age group. Three cohorts, segmented by age, are at various stages of enrollment in this study. Cohort 1 includes children ages 24 to 60 months old and has completed enrollment. Cohort 2 includes children ages 6 to 24 months old and will complete enrollment by year-end. Cohort 3, includes children ages 0 to 6 months old and began enrolling earlier this month.

BMN 307 gene therapy product candidate for phenylketonuria (PKU): On October 21, 2019, BioMarin was granted Orphan drug designation from the FDA for BMN 307 for the treatment of phenylketonuria. On September 26, 2019, the Company submitted a CTA with the medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K.
Preclinical data with BMN 307 demonstrated a lifetime Phe correction sustained at 80 weeks in mouse models. BMN 307 is an AAV vector containing the DNA sequence that codes for the phenylalanine hydroxylase enzyme that is deficient in people with PKU. Product to support clinical evaluation is being produced at BioMarin’s gene therapy manufacturing facility, where valoctocogene roxaparvovec is currently made, using a commercial scale manufacturing process to facilitate rapid clinical development.

Tralesinidase alfa (formerly referred to as BMN 250) for Mucopolysaccharidosis IIIB (MPS IIIB) or Sanfilippo Syndrome, Type B: On October 23, 2019, the Company announced that it had entered into a licensing agreement with Allievex Corp. (Allievex) for tralesinidase alfa, an investigational Enzyme Replacement Therapy (ERT) for MPS IIIB or Sanfilippo Syndrome Type B. Under the terms of the agreement, Allievex will receive a worldwide, exclusive license to tralesinidase alfa. BioMarin is entitled to receive a minority equity stake in Allievex, milestone payments if certain development, regulatory and sales milestones are met by Allievex and royalties on net sales of tralesinidase alfa. Upon closing of the transaction, Allievex will assume all financial obligations associated with the development and commercialization of tralesinidase alfa other than certain continued manufacturing activities that will be paid by BioMarin. BioMarin will transfer all tralesinidase alfa-related clinical and regulatory activity and responsibilities to Allievex during a transition period following closing of the transaction. Tralesinidase alfa is currently being evaluated in ongoing natural history and clinical trials.

R&D Day to be held in New York November 14, 2019: The Company plans to hold an investor event to discuss potential new product candidates, vosoritide, valoctocogene roxaparvovec status and other general corporate updates. Please email [email protected] for more information.
BioMarin will host a conference call and webcast to discuss third quarter 2019 financial results today, Wednesday, October 23, 2019 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.
U.S. / Canada Dial-in Number: 866.502.9859
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Conference ID: 6989536

Conference ID: 6989536