As we enter the next decade, our pace of scientific innovation continues to increase. In 2020, we celebrated one of our earliest breakthroughs, the 15th anniversary of the approval of Naglazyme® (galsulfase), which laid the groundwork for our leadership in rare diseases. Our approved products, our R&D engine, our manufacturing expertise, and the employees supporting BioMarin create a solid base for growth that we believe will support us in continuing our success into the new decade.
As I write this letter, we face an unprecedented pandemic that is heart wrenching and will profoundly impact our society. During this time, we gain a greater appreciation of the value of the life sciences industry as a force for good. Never before has there been so much hope and need in our industry’s ability to change the course of disease.
We have met the adversity of 2020 with versatility and agility. In what has been the most unpredictable year we have experienced at BioMarin, the resilience of our business and fortitude of our employees has consistently shone through.
As a result, we remain well-positioned for substantial growth as we focus on the execution of our global opportunities with valoctocogene roxaparvovec and vosoritide, as well as other key pipeline products. And while economic uncertainty related to the pandemic will touch every company, including ours, we are monitoring the situation very closely, adjusting where necessary and remaining focused on the long-term as we manage through this health crisis. BioMarin is an “essential business” based on the importance of our medicines, and as such, we are making every effort to ensure continued access to our therapies for our patients no matter where they are located. As scientific pioneers, we are comfortable navigating uncharted territory, an ability that serves us in this health crisis, as well as in our business overall.
We begin the new decade with an established base business that is foundational to our financial strength. Our revenue growth and improvement in profitability also increased our operating cash flows. We have two potential blockbusters on the horizon that are all expected to drive meaningful growth.
With valoctocogene roxaparvovec, we did not expect the CRL in August, but we have refocused on productive next steps toward approval. We remain encouraged and committed to valoctocogene roxaparvovec based on the continued support and anecdotes from our investigators who report the life-changing effects of valoctocogene roxaparvovec. And with the complete, one-year Phase 3 data from our 134 subject study the next inflection point in the program, we excitedly await those results in early 2021. In the meantime, we continue to make progress in discussions with health authorities and understanding what it takes to register valoctocogene roxaparvovec.
We want to thank the hemophilia community for providing BioMarin the opportunity and support to develop valoctocogene roxaparvovec. A major part of this support comes from the dedication and commitment of key opinion leaders in the field.
With vosoritide for the treatment of achondroplasia, applications in the US and Europe are under review by health authorities and are in process for potential approvals in 2021, with the CHMP opinion expected in the second half of 2021 and the US PDUFA Action date planned for August 20, 2021. Based on the highly statistically significant results observed in children with achondroplasia treated with vosoritide, we are optimistic that vosoritide will be the first pharmacologic therapy approved in this indication. As with valoctocogene roxaparvovec, we anticipate the highly innovative attributes of vosoritide will drive uptake from patients and meaningful contributions to the business upon potential approval and launch. There is tremendous unmet medical need in this patient population, as demonstrated by the level of interest from families seeking treatment for their very young children.
We believe the next two years present an enormous opportunity for value creation based on the following: potential launches in valoctocogene roxaparvovec and vosoritide, if approved, continued expansion of our PKU franchise with global Palynziq® (pegvaliase-pqpz) penetration and the development of PKU gene therapy, initial clinical development of a gene therapy for Hereditary Angioedema (HAE), our abundant cash reserves, our wholly owned manufacturing facilities and our global footprint.
As we begin a new decade having already built a strong base business, transitioned our pipeline to address larger rare indications, and laid the foundation for significant profitability if valoctocogene roxaparvovec and vosoritide are approved, we believe we have diversified risk and positioned ourselves for substantial success in both the near-term and long-term.
Promising Early Stage Pipeline
On the heels of valoctocogene roxaparvovec and vosoritide reviews and potential approvals, and continued commercial expansion of Palynziq for phenylketonuria, each of which represent very large global revenue opportunities, our earlier-stage pipeline is also moving forward. In July of this year, we began enrolling patients in the PHEarless Phase 2 study for BMN 307, our investigational gene therapy for PKU, with a goal of achieving normalized Phe. This study could potentially be registration enabling as we are conducting it with material manufactured with a commercial-ready process to de-risk the program and facilitate rapid clinical development. We are excited about the prospect of BMN 307, as it represents a potential third PKU treatment option in our PKU franchise and a second gene therapy development program, leveraging our learnings and capabilities from valoctocogene roxaparvovec.
Finally, our earlier-stage pipeline includes our BMN 331 gene therapy product candidate for HAE, a rare and potentially life-threatening genetic disease that causes swelling throughout the body, which could include choking because of swelling of the airway. Last quarter, we began IND-enabling studies with BMN 331, which should benefit from an even more efficient development program based on our increasing experience.
We also recently announced the expansion of our clinical program for vosoritide with two new Phase 2 studies. The first study is sponsored by BioMarin to investigate the safety of vosoritide in infants with achondroplasia at risk of life-threatening foramen magnum compression. The second study is an investigator-initiated study sponsored by Children’s National Hospital in Washington, D.C. to investigate vosoritide in children with selected genetic forms of short stature, which together represent addressable patient populations of approximately 275,000.
Our early pipeline employs our development strategy to build on our technical expertise of gene therapy in PKU and HAE, to explore developing one treatment for multiple indications as we are doing by investigating vosoritide in GSS, and to develop multiple treatments for one disease area like PKU with a potential third product candidate in development.
Exciting Research Opportunities
We have entered into a number of strategic partnerships with the goal of growing our pipeline long-term. In May, we announced a preclinical collaboration and license agreement with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies, extending our gene therapy leadership. The license initially covers DiNAQOR’s lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM). Additionally, the companies will collaborate on several of DiNAQOR’s other pipeline programs, and BioMarin has the option to extend the license to include these additional programs on similar terms.
Recently, we announced another preclinical collaboration with a Company called Deep Genomics artificial intelligence drug discovery platform to identify oligonucleotide drug candidates in four rare disease indications with high unmet need. Under the collaboration, we hope to identify and validate target mechanisms and lead candidates, that BioMarin will advance into preclinical and clinical development.
Driving Growth, Entering the Next Decade
As described earlier, we believe we have developed a clear and achievable strategy that is designed to position BioMarin for long-term success.
With 2020 coming to a close, it is important to have perspective on what has been accomplished to this point, as well as the tremendous opportunities that lie ahead. It has been an unpredictable year, but the underlying fundamentals of our business are strong, and we are resolute in our mission. BioMarin employees have demonstrated extraordinary resilience in the face of uncertainty and we have confidence in our ability to navigate any new challenges that may arise as we forge ahead.
We are grateful to our employees who work tirelessly to pioneer treatments that advance the standard of care for patients with rare genetic diseases, the patients and their families who inspire us to deliver scientific breakthroughs, the physicians and nurses who treat our patients, academic researchers, regulatory authorities, government officials, payers, and the local communities where we live and operate.
Thank you for your continued support and stay safe.
Chairman and Chief Executive Officer
BioMarin Pharmaceutical Inc.
– November 20, 2020
This letter contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about BioMarin’s continued revenue growth, financial success and opportunities for value creation; BioMarin’s ability to reach expected milestones for products and product candidates in its pipeline; the continued clinical development and commercialization of BioMarin’s commercial products and product candidates and the timing of such development and commercialization; the possible approval and commercialization of BioMarin’s product candidates, the expected contributions of these product candidates to top-line growth, if approved, and the potential for these product candidates to provide meaningful cost-savings to the healthcare system and change treatment paradigms, if approved; actions by regulatory authorities and the timing of such actions; BioMarin’s ability to navigate through and operate during the COVID-19 pandemic, including it being well-positioned to limit disruptions to its operations; BioMarin’s diversification of risk and position for substantial success in both the near-term and long-term; BioMarin’s strategy for long-term success and the clarity and achievability of that strategy; and other clinical development, regulatory interactions, manufacturing and commercial operations in 2020 and beyond. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin’s ability to successfully commercialize its current or future commercial products; the results and timing of current and planned preclinical studies and clinical trials, as well as the potential impact of the COVID-19 pandemic on BioMarin’s ability to continue such preclinical studies and clinical trials and the timing of such preclinical studies and clinical trials; BioMarin’s ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities concerning BioMarin’s products and product candidates, including the potential impact of the COVID-19 pandemic on the regulatory authorities’ abilities to issue such decisions and the timing of such decisions; the market for BioMarin’s products and product candidates; actual sales of BioMarin’s commercial products; and those factors detailed in BioMarin’s filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption “Risk Factors” in BioMarin’s Annual Report on Form 10-K for the year ended December 31, 2019, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.