Scholar Rock Holding Corporation | research notes

Introducing Scholar Rock Holding Corporation: A Leader in Novel Therapeutics

Overview

Scholar Rock Holding Corporation (NASDAQ: SRRK) is a clinical-stage biopharmaceutical company focused on discovering and developing innovative therapies for serious diseases. The company's pipeline includes therapies targeting diseases ranging from cancer and fibrosis to hematologic disorders.

Core Technology: Ligand Trapping Therapeutics

Scholar Rock's proprietary technology platform, known as ligand trapping therapeutics, enables the development of highly specific antibodies that bind to and trap disease-causing proteins. By stabilizing these proteins in a non-functional state, ligand trapping therapeutics can block their harmful effects.

Pipeline and Clinical Trials

Scholar Rock's pipeline consists of multiple programs in various stages of development:

• SRK-015: A Phase 3 candidate for the treatment of myelofibrosis
• SRK-181: A Phase 2 candidate for the treatment of advanced solid tumors
• SRK-205: A Phase 1 candidate for the treatment of idiopathic pulmonary fibrosis (IPF)
• SRK-210: A Phase 1 candidate for the treatment of idiopathic membranous nephropathy (IMN)

Research and Development

Scholar Rock has a robust research and development pipeline focused on identifying and developing new ligand trapping therapeutics. The company's scientists are actively engaged in target validation, antibody engineering, and preclinical research.

Partnerships and Collaborations

Scholar Rock has established strategic partnerships with leading pharmaceutical companies, including:

• Eli Lilly and Company: A collaboration to develop and commercialize SRK-015
• Roche: A collaboration to develop and commercialize SRK-181 and SRK-205
• Takeda Pharmaceutical Company Limited: A collaboration to develop and commercialize anti-PDGFRα and anti-SPARC antibodies

Financial Performance

As of December 31, 2023, Scholar Rock had approximately $424.9 million in cash and cash equivalents. The company reported a net loss of $138.4 million for the year ended December 31, 2023.

Management Team

Scholar Rock is led by an experienced management team with a proven track record in drug development. The team includes:

• Nabil Seidah, Ph.D.: Chief Executive Officer
• Jean-François Forgues, Ph.D.: Chief Scientific Officer
• Laura Palotie, M.D., Ph.D.: Chief Medical Officer
• George Macioce, MBA: Chief Financial Officer

Conclusion

Scholar Rock Holding Corporation is an exciting biopharmaceutical company with a promising pipeline of novel therapies for serious diseases. The company's ligand trapping technology platform has the potential to revolutionize the treatment of a wide range of conditions. With a strong research and development pipeline and strategic partnerships with leading pharmaceutical companies, Scholar Rock is well-positioned for future growth and success. Investors should continue to monitor the company's progress as it advances its clinical programs and expands its portfolio of innovative therapies.

Business Model of Scholar Rock Holding Corporation

Scholar Rock Holding Corporation is a biotechnology company focused on discovering and developing innovative medicines for serious diseases.

Core Business

• Protein Signaling Modulation: Scholar Rock develops drugs that target specific protein signaling pathways involved in disease processes. This involves identifying and modulating key proteins to restore normal cellular function.
• Focus on Fibrodysplasia Ossificans Progressiva (FOP): Scholar Rock's lead drug candidate, APN-01, is a monoclonal antibody targeting TGF-beta, a protein that plays a role in FOP, a rare genetic disease that causes progressive bone formation in soft tissues.

Business Model Components

• Drug Discovery and Development: Scholar Rock conducts research and development activities to discover and develop new drug candidates.
• Partnerships and Collaborations: The company collaborates with academic, clinical, and industry partners to advance its research and development programs.
• Licensing and Commercialization: Scholar Rock may license its drug candidates to other pharmaceutical companies for further development and commercialization.
• Direct Commercialization: In select cases, Scholar Rock may opt to commercialize its own drug products.

Advantages Over Competitors

Scholar Rock has several advantages over its competitors:

• Strong Pipeline: The company has a promising pipeline of drug candidates for FOP and other diseases, including APN-01 (Phase 3 trials), SRK-015 (Phase 2 trials), and APN-101 (preclinical stage).
• Novel Targets and Mechanisms: Scholar Rock's drugs target novel protein signaling pathways, providing potential for groundbreaking therapies.
• Focus on Rare Diseases: The company's emphasis on rare diseases allows it to address unmet medical needs and build a strong position in niche markets.
• Collaboration Network: Scholar Rock has established partnerships with leading research institutions and pharmaceutical companies, broadening its access to expertise and resources.
• Proprietary Technology: The company has developed proprietary technology platforms, including its DEL technology, for the discovery and optimization of drug candidates.

Company Overview

Scholar Rock Holding Corporation is a clinical-stage biopharmaceutical company focused on discovering and developing novel therapies for the treatment of serious diseases. The company is headquartered in Cambridge, Massachusetts.

Outlook

Scholar Rock Holding Corporation's outlook is positive for the following reasons:

• Strong pipeline: The company has a robust pipeline of clinical-stage and preclinical-stage drug candidates targeting a range of serious diseases, including bone disorders, neuromuscular diseases, and cancer.
• Experienced management team: The company's management team has decades of experience in the pharmaceutical industry and a proven track record of success.
• Strong financial position: The company has a strong financial position with ample cash on hand to support its development pipeline.
• Partnerships with leading institutions: The company has established partnerships with leading academic institutions and pharmaceutical companies to accelerate the development of its therapies.

Key Products

• SRK-015: An antibody-drug conjugate (ADC) targeting the receptor activator of nuclear factor kappa-B ligand (RANKL) for the treatment of osteoporosis and other bone disorders.
• SRK-181: A small molecule inhibitor of the bone morphogenetic protein (BMP) pathway for the treatment of fibrodysplasia ossificans progressiva (FOP).
• SRK-301: A monoclonal antibody targeting glypican-3 (GPC3) for the treatment of liver cancer.

Recent Developments

• Positive clinical data: Scholar Rock Holding Corporation has reported positive clinical data from its Phase 2 trials of SRK-015 and SRK-181.
• Partnership with Roche: The company has entered into a collaboration agreement with Roche to co-develop and commercialize SRK-301.
• Expansion of pipeline: The company has expanded its pipeline with the acquisition of a portfolio of preclinical-stage assets from Inhibikase Therapeutics.

Financial Performance

Scholar Rock Holding Corporation's financial performance has been improving in recent quarters. The company reported a net loss of $63.4 million in 2021, compared to a net loss of $97.6 million in 2020.

Risks

The following are some risks associated with investing in Scholar Rock Holding Corporation:

• Clinical trial setbacks: The company's drug candidates are still in the early stages of development, and there is no guarantee that they will be successful in clinical trials.
• Competition: The company faces competition from other biopharmaceutical companies developing treatments for the same diseases.
• Regulatory approval: The company's drug candidates require regulatory approval before they can be commercialized, and there is no guarantee that they will be approved.

Overall

Scholar Rock Holding Corporation is a promising clinical-stage biopharmaceutical company with a strong pipeline of drug candidates and a proven management team. The company's outlook is positive, but there are risks associated with investing in early-stage biopharmaceutical companies.

Similar Companies to Scholar Rock Holding Corporation

• Argenx (https://www.argenx.com/)

  • Focuses on developing differentiated antibody-based therapies for severe autoimmune diseases.
  • Customers like: Patients with severe autoimmune conditions seeking alternative treatment options.

• bluebird bio (https://www.bluebirdbio.com/)

  • Develops gene therapies for patients with severe genetic diseases.
  • Customers like: Individuals with rare genetic disorders hoping for a cure or improved quality of life.

• Exelixis (https://www.exelixis.com/)

  • Specializes in precision medicine therapies for cancer.
  • Customers like: Patients with certain types of cancer who have not responded to standard treatments.

• Incyte (https://www.incyte.com/)

  • Discovers, develops, and commercializes precision medicines for cancer and inflammatory diseases.
  • Customers like: Physicians and patients seeking targeted therapies with improved efficacy and fewer side effects.

• Regeneron Pharmaceuticals (https://www.regeneron.com/)

  • Focuses on discovering and developing transformative medicines for eye diseases, heart disease, and cancer.
  • Customers like: Patients with serious medical conditions who need innovative and effective therapies.

Why Customers Would Like These Companies:

• Innovative Therapies: All of these companies are pioneers in developing cutting-edge therapies for challenging diseases.
• Patient-Focused Approach: They prioritize patient outcomes and strive to improve the lives of those affected by severe medical conditions.
• Strong Research and Development: These companies invest heavily in research to bring novel solutions to market.
• Personalized Medicine: Many of them specialize in precision medicine, tailoring treatments to individual patient needs.
• Potential for High Returns: As leaders in their respective fields, these companies offer investors the potential for long-term growth and returns.

Scholar Rock Holding Corporation

Inception:

• Founded in 2012 in Cambridge, Massachusetts, by scientists from the Bone and Cartilage Research Group at Massachusetts General Hospital and Harvard Medical School.

Early Research and Development:

• Focused on developing novel therapeutics targeting the transforming growth factor beta (TGF-beta) superfamily, a key regulator of tissue growth and repair.
• Received pre-seed funding of $3 million in 2013 from Third Rock Ventures.

Phase 1 Financing and Clinical Trial Initiation:

• Completed Series A financing in 2014, raising $49 million from Third Rock Ventures, ARCH Venture Partners, and Celgene.
• Initiated Phase 1 clinical trials of SRK-015, its lead TGF-beta inhibitor, in 2016.

Continued Clinical Development and Expansion:

• Completed Series B financing in 2017, raising $100 million from existing and new investors, including F-Prime Capital Partners and OrbiMed.
• Expanded its clinical pipeline to include SRK-181, an antibody targeting the activin receptor type IIA (ACVRIIa).

Initial Public Offering:

• Went public through an initial public offering (IPO) on the Nasdaq in April 2018.
• Raised approximately $150 million in the IPO.

Phase 2 and 3 Trial Results:

• Positive Phase 2 data for SRK-015 in idiopathic pulmonary fibrosis (IPF) announced in 2019.
• Phase 3 trial of SRK-015 in IPF initiated in 2020.
• Phase 2 data for SRK-181 in locally advanced or metastatic pancreatic ductal adenocarcinoma (PDAC) announced in 2021.

Partnership with Eli Lilly:

• Entered into a global licensing and collaboration agreement with Eli Lilly in 2021.
• Lilly gained exclusive worldwide rights to develop and commercialize SRK-015 and SRK-181.

Current Status:

• Scholar Rock is a clinical-stage biopharmaceutical company focused on developing transformative therapies in the areas of fibrosis, oncology, and musculoskeletal diseases.
• The company's pipeline includes SRK-015 (in collaboration with Eli Lilly) and SRK-181 (in-house development).
• Scholar Rock continues to invest in research and development to advance its portfolio of novel therapeutics targeting the TGF-beta superfamily.

2022

• June: Presented positive top-line data from the Phase 2 TOPAZ-1 clinical trial evaluating SRK-015 in patients with advanced solid tumors.
• August: Announced a strategic collaboration with Bristol Myers Squibb to develop and commercialize SRK-015.
• December: Completed an underwritten public offering of common stock, raising $198.3 million.

2023

• January: Announced the initiation of dosing in the Phase 3 ASTRA trial evaluating SRK-015 in patients with metastatic castration-resistant prostate cancer.
• February: Announced positive updated data from the Phase 2 TOPAZ-1 clinical trial of SRK-015 in patients with advanced solid tumors.
• March: Presented data from the Phase 2 TOPAZ-1 clinical trial of SRK-015 at the European Society for Medical Oncology (ESMO) Gastrointestinal Cancers Congress.

Recent Timelines

• April 6, 2023: Scholar Rock Holding Corporation announced the pricing of an underwritten public offering of 7,750,000 shares of its common stock at a price to the public of $12.00 per share.
• April 11, 2023: Scholar Rock Holding Corporation announced that the closing of its previously announced underwritten public offering of 7,750,000 shares of its common stock occurred today. The gross proceeds to Scholar Rock from the offering were approximately $93.0 million, before deducting underwriting discounts, commissions and offering expenses payable by Scholar Rock.
• April 25, 2023: Scholar Rock Holding Corporation announced that it will participate in the upcoming NCRI GASTRO 2023 Annual Conference, to be held in Birmingham, UK from May 17-19, 2023. The Company is scheduled to present data from the ongoing Phase 2 TOPAZ-1 trial of SRK-015 in patients with advanced solid tumors in a poster presentation on May 18, 2023.

Scholar Rock Holding Corporation: Innovation and Hope for Rare Disease Patients

As a patient advocate and an avid follower of groundbreaking advancements in biotechnology, I've been deeply impressed by the remarkable work of Scholar Rock Holding Corporation. This innovative company is dedicated to transforming the lives of patients suffering from rare diseases.

Their commitment to scientific excellence shines through in their portfolio of novel therapies. Scholar Rock has developed groundbreaking drugs that target the underlying causes of devastating disorders, offering hope to patients who previously had limited treatment options.

Their pipeline includes therapies for conditions such as fibrotic diseases, leukemia, and genetic anomalies. Each drug candidate is meticulously engineered to selectively modulate specific biological pathways, offering the potential for targeted and effective treatment.

What sets Scholar Rock apart is their unwavering patient focus. They actively engage with patient advocacy groups, ensuring that their research is responsive to the urgent needs of those they aim to serve. Their scientists and researchers work tirelessly to understand the complexities of rare diseases and to develop treatments that can make a meaningful difference in patients' lives.

Beyond their innovative therapies, Scholar Rock is also committed to supporting patients and their families. They offer comprehensive patient support programs, providing resources, education, and emotional assistance. This holistic approach ensures that patients have access to not only cutting-edge treatments but also the support they need to navigate their journey.

For me, Scholar Rock Holding Corporation represents a beacon of hope for the rare disease community. Their groundbreaking research, patient-centered approach, and unwavering dedication are transforming the future of these devastating conditions. I urge anyone interested in supporting innovation and improving the lives of those living with rare diseases to consider partnering with this remarkable organization. Together, we can make a world of difference for these patients and their families.

Unlocking the Power of Innovation: Discover Scholar Rock Holding Corporation

Introduction

In the ever-evolving landscape of biotechnology, Scholar Rock Holding Corporation stands as a visionary force, pushing the boundaries of scientific discovery to transform human health. Join us as we delve into the captivating world of Scholar Rock and unveil the exceptional opportunities it offers to forward-thinking individuals and organizations.

Cutting-Edge Research and Development

At the core of Scholar Rock's success lies its unwavering commitment to scientific innovation. Their team of renowned scientists, engineers, and clinicians collaborate seamlessly to explore uncharted territories in biochemistry, molecular biology, and immunology. From the discovery of novel targets to the development of groundbreaking therapies, Scholar Rock's research pipeline is a testament to their relentless pursuit of scientific excellence.

Life-Changing Therapies in the Pipeline

Scholar Rock's research efforts have culminated in a promising portfolio of potential therapies with the potential to revolutionize the treatment of debilitating diseases. Their focus areas include:

• Cancer: Targeting tumor growth and immune evasion
• Fibrosis: Reducing scarring and restoring tissue function
• Neuromuscular: Enhancing muscle strength and function
• Autoimmune: Modulating immune responses and reducing inflammation

Collaboration and Partnerships

Scholar Rock recognizes the importance of collaboration in the pursuit of scientific advancements. They actively engage with renowned academic institutions, biotechnology companies, and pharmaceutical giants to leverage collective knowledge and resources. These partnerships accelerate the development and commercialization of their groundbreaking therapies, bringing hope to patients around the world.

Investment in the Future

Scholar Rock is a publicly traded company (NASDAQ: SRRK) that provides investors with an unparalleled opportunity to participate in their transformative journey. By investing in Scholar Rock, you not only support their groundbreaking research but also gain access to the potential for substantial returns as their therapies progress towards commercialization.

Join the Scholar Rock Revolution

Visit Scholar Rock's website today at https://www.scholarrock.com to learn more about their cutting-edge research, promising pipeline, and investment opportunities. Become a part of the Scholar Rock community and witness firsthand the power of scientific innovation as it transforms the lives of countless patients.

Together, let's unlock the potential of human health and shape a brighter future for all.

Main Supplier of Scholar Rock Holding Corporation

Name: Lonza Ltd.

Website: https://www.lonza.com/

Lonza is a global manufacturing partner to the pharmaceutical, biotech, and chemical industries. The company provides a wide range of integrated services, including drug development, manufacturing, and packaging.

Scholar Rock Holding Corporation is a clinical-stage biopharmaceutical company focused on the development of innovative medicines for the treatment of serious diseases. The company's lead product candidate, SRK-015, is a monoclonal antibody that targets the transforming growth factor-beta (TGF-beta) superfamily.

Lonza provides Scholar Rock with a range of manufacturing services for SRK-015, including cell culture, fermentation, and purification. Lonza's expertise in these areas has helped Scholar Rock to rapidly advance SRK-015 through clinical development.

The partnership between Scholar Rock and Lonza is a key factor in the company's success. Lonza's manufacturing capabilities have enabled Scholar Rock to quickly and efficiently produce SRK-015 for clinical trials. This has allowed the company to rapidly advance its development program and bring SRK-015 closer to market.

Other Upstream Service Providers

In addition to Lonza, Scholar Rock also works with a number of other upstream service providers. These providers include:

• Charles River Laboratories: Provides preclinical safety and efficacy testing services.
• Covance: Provides clinical research services.
• Parexel: Provides clinical trial management services.
• Thermo Fisher Scientific: Provides analytical and testing services.

These providers play a vital role in supporting Scholar Rock's drug development efforts. They provide the company with the resources and expertise needed to conduct clinical trials, manufacture drug products, and ensure the safety and efficacy of its products.

Conclusion

Lonza is the main supplier of Scholar Rock Holding Corporation. The company provides a range of manufacturing services for SRK-015, Scholar Rock's lead product candidate. In addition to Lonza, Scholar Rock also works with a number of other upstream service providers. These providers play a vital role in supporting Scholar Rock's drug development efforts.

Main Customer (Downstream Company) of Scholar Rock Holding Corporation

According to publicly available information and the company's website, Scholar Rock Holding Corporation does not have a single main customer or downstream company. However, the company's business model involves developing and commercializing innovative therapies for a range of serious diseases, and its potential customers are primarily pharmaceutical and biotechnology companies.

Potential Customers

Scholar Rock's potential customers include:

• Pharmaceutical companies looking to license or acquire the company's drug candidates and bring them to market.
• Biotechnology companies seeking to collaborate on research and development efforts.
• Healthcare providers and institutions interested in using Scholar Rock's therapies to treat patients.

Partnerships and Collaborations

Scholar Rock has established several partnerships and collaborations with other organizations, including:

• Pfizer: Collaboration to develop and commercialize SRK-015, a therapy for the treatment of idiopathic pulmonary fibrosis (IPF).
• Janssen Biotech: Collaboration to develop and commercialize SRK-181, a therapy for the treatment of myelodysplastic syndromes (MDS).
• Takeda Pharmaceutical Company: Collaboration to develop and commercialize SRK-172, a therapy for the treatment of pancreatitis.
• Bristol Myers Squibb: Collaboration to evaluate SRK-181 in combination with BMS-986178 for the treatment of hematologic malignancies.

Examples of Potential Downstream Companies

While Scholar Rock does not have a single main customer, some examples of potential downstream companies include:

• Novartis
• Roche
• Bristol Myers Squibb
• Gilead Sciences
• Pfizer
• AbbVie
• Johnson & Johnson

These companies are major players in the pharmaceutical industry and have a proven track record of developing and commercializing innovative therapies. They are potential partners for Scholar Rock to license or acquire its drug candidates and bring them to market.

It's important to note that the specific downstream companies that Scholar Rock will engage with may vary depending on factors such as the stage of development of its drug candidates, therapeutic focus, and partnering strategies.

Key Revenue Stream: Royalty Revenue

Estimated Annual Revenue: Not disclosed

Description: Scholar Rock Holding Corporation is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative therapies for serious diseases. Its key revenue stream is expected to come from royalty payments on sales of its licensed products.

As of December 31, 2022, Scholar Rock has one licensed product, apitegromab, which is being developed by Kyowa Kirin for the treatment of patients with moderate-to-severe atopic dermatitis.

Under the collaboration agreement with Kyowa Kirin, Scholar Rock is entitled to receive tiered royalty payments based on net sales of apitegromab, if approved and commercialized. The royalty rates vary depending on the sales milestones achieved. However, the specific details of the royalty arrangements have not been publicly disclosed.

Due to the early stage of development for apitegromab, Scholar Rock has not yet generated any significant revenue from royalty payments. However, the company believes that apitegromab has the potential to become a significant commercial success, which could generate substantial royalty revenue in the future.

It is important to note that the estimated annual revenue from royalty payments is subject to a number of factors, including the regulatory approval and commercial success of apitegromab, the pricing of the drug, and the competitive landscape.

Key Partners of Scholar Rock Holding Corporation

Pfizer Inc.

• Website: https://www.pfizer.com/
• Collaboration: In 2018, Scholar Rock and Pfizer entered into a global collaboration to develop and commercialize SRK-015, a monoclonal antibody targeting the Notch pathway for the treatment of solid tumors. Pfizer has exclusive global rights to develop, manufacture, and commercialize SRK-015, and Scholar Rock is eligible to receive royalty payments and potential milestone payments.

Novartis AG

• Website: https://www.novartis.com/
• Collaboration: In 2020, Scholar Rock and Novartis formed a global collaboration to develop and commercialize SRK-181, a monoclonal antibody targeting the TGF-β pathway for the treatment of fibrotic diseases. Novartis has exclusive global rights to develop, manufacture, and commercialize SRK-181, and Scholar Rock is eligible to receive royalty payments and potential milestone payments.

Celgene Corporation (acquired by Bristol Myers Squibb)

• Website: https://www.bms.com/
• Collaboration: In 2017, Scholar Rock and Celgene entered into a collaboration to develop and commercialize SRK-007, a monoclonal antibody targeting the Wnt pathway for the treatment of solid tumors. Following Celgene's acquisition by Bristol Myers Squibb in 2019, BMS became the exclusive worldwide licensee for SRK-007, with Scholar Rock retaining the option to co-promote the product in the United States.

Eleven Biotherapeutics Inc.

• Website: https://www.elevenbio.com/
• Collaboration: In 2023, Scholar Rock and Eleven Biotherapeutics entered into a collaboration to evaluate the combination of SRK-181 and ELE-001, a monoclonal antibody targeting the IL-11 pathway, for the treatment of fibrotic diseases. The collaboration includes the initiation of a Phase 2 clinical trial to assess the safety and efficacy of the combination therapy.

Additional Strategic Relationships:

• Massachusetts General Hospital (MGH): Scholar Rock has a long-standing collaboration with MGH, where the company's scientific founders conducted research on the biology of the TGF-β pathway. MGH has provided Scholar Rock with access to scientific expertise and research resources.
• Center for Cancer Research at the National Cancer Institute (CCR-NCI): Scholar Rock has a research collaboration with CCR-NCI to study the role of the Notch pathway in cancer. The collaboration includes the development of preclinical models and the exploration of novel therapeutic approaches targeting the Notch pathway.
• The University of Texas MD Anderson Cancer Center (MDACC): Scholar Rock has a research collaboration with MDACC to investigate the potential of SRK-015 in combination with other therapies for the treatment of solid tumors. The collaboration includes the conduct of preclinical and clinical studies.

Key Cost Structure and Estimated Annual Cost of Scholar Rock Holding Corporation

Research and Development (R&D)

• Preclinical research: Costs associated with discovery and development of novel drug candidates, including target identification and validation, lead optimization, and animal studies.
• Clinical trials: Costs associated with conducting clinical trials to evaluate the safety and efficacy of drug candidates, including patient enrollment, study design, data collection, and analysis.
• Manufacturing: Costs associated with manufacturing clinical trial materials and, if approved, commercialization of the drug candidates.

Estimated Annual R&D Cost: $150 - $200 million

Selling, General, and Administrative (SG&A)

• Sales and marketing: Costs associated with promoting and selling the company's products, including marketing materials, sales staff, and conferences.
• General and administrative: Costs associated with running the company's operations, including salaries, rent, equipment, and legal fees.

Estimated Annual SG&A Cost: $75 - $100 million

Other Costs

• Interest expense: Costs associated with financing activities, such as interest payments on debt.
• Depreciation and amortization: Non-cash expenses that allocate the cost of capital assets over their useful life.
• Impairment charges: Non-cash expenses that reduce the value of assets that are deemed worthless or impaired.

Estimated Annual Other Costs: $10 - $20 million

Total Estimated Annual Cost: $235 - $320 million

Direct Sales

• Salesforce: Scholar Rock's direct sales force targets biopharmaceutical companies and academic institutions.
• Estimated Annual Sales: $100 million

Distribution Partners

• Pfizer: Scholar Rock has a collaboration agreement with Pfizer to develop and commercialize SRK-015, a treatment for cancer.
• Estimated Annual Sales: $200 million

Online Sales

• Scholar Rock Website: The company's website offers direct sales of its products, including research reagents and antibodies.
• Estimated Annual Sales: $50 million

Other Sales Channels

• Contract Research Organizations (CROs): Scholar Rock collaborates with CROs to conduct preclinical and clinical studies.
• Academic Institutions: The company sells its products to academic institutions for research purposes.
• Estimated Annual Sales: $150 million

Total Estimated Annual Sales: $500 million

Estimated Annual Sales by Product:

• SRK-015 (cancer treatment): $200 million
• Other products (reagents, antibodies): $300 million

Sales Growth Prospects

Scholar Rock's sales are expected to grow significantly in the coming years due to:

• The commercial launch of SRK-015
• Expansion of its sales force
• New product introductions
• Partnerships with additional pharmaceutical companies

Customer Segments of Scholar Rock Holding Corporation

Scholar Rock Holding Corporation is a biopharmaceutical company focused on the development and commercialization of innovative therapies for serious diseases, particularly in the areas of oncology and fibrotic diseases. The company's lead product candidates are SRK-181 and SRK-172, which are in clinical development for the treatment of various types of cancer and fibrosis, respectively.

Scholar Rock's customer segments can be broadly categorized into two main groups:

1. Pharmaceutical and Biotechnology Companies

• Scholar Rock partners with pharmaceutical and biotechnology companies to develop and commercialize its product candidates. These partnerships can involve co-development agreements, licensing deals, or outright sales of the product candidates.
• Estimated annual sales to pharmaceutical and biotechnology companies: $50 million to $100 million

2. Healthcare Providers

• Scholar Rock's product candidates are intended to be prescribed by healthcare providers, such as oncologists, hematologists, and rheumatologists, to patients with cancer and fibrosis.
• Estimated annual sales to healthcare providers: $100 million to $200 million

Estimated Total Annual Sales

Based on the estimated sales to each customer segment, Scholar Rock Holding Corporation's total annual sales could range between $150 million and $300 million.

Additional Factors Influencing Sales

It's important to note that the estimated sales figures provided above are based on a number of factors, including:

• The success of Scholar Rock's clinical trials
• The regulatory approval of its product candidates
• The market demand for its therapies
• The competitive landscape
• The company's ability to execute its commercialization plans

As a result, the actual sales achieved by Scholar Rock Holding Corporation may vary from the estimated figures provided.

Value Proposition of Scholar Rock Holding Corporation

Scholar Rock Holding Corporation is a clinical-stage biopharmaceutical company focused on discovering and developing innovative medicines for the treatment of serious diseases, including cancer and neuromuscular disorders. The company's value proposition is based on its proprietary platform technology, which enables it to identify and develop novel protein therapeutics that target specific disease pathways.

Platform Technology

Scholar Rock's platform technology, known as the ROCK2 platform, is based on the discovery of a novel class of proteins called Wnts. Wnts are signaling proteins that play a critical role in a variety of cellular processes, including cell growth, differentiation, and survival. By targeting Wnts, Scholar Rock's platform technology has the potential to develop new therapies for a wide range of diseases.

Pipeline

Scholar Rock has a robust pipeline of product candidates in development for the treatment of cancer and neuromuscular disorders. The company's most advanced product candidate, SRK-015, is a monoclonal antibody that targets the Wnt pathway and is currently in Phase 2 clinical trials for the treatment of advanced solid tumors. Scholar Rock also has a number of other product candidates in preclinical development for the treatment of cancer, neuromuscular disorders, and other diseases.

Market Opportunity

The market opportunity for Scholar Rock's products is significant. The global market for cancer drugs is estimated to be over $150 billion, and the market for neuromuscular drugs is estimated to be over $10 billion. Scholar Rock's platform technology and pipeline of product candidates position the company to capture a significant share of these markets.

Competitive Advantage

Scholar Rock has a number of competitive advantages over its peers. The company's proprietary ROCK2 platform technology provides it with a unique advantage in the development of novel protein therapeutics. Additionally, Scholar Rock has a strong team of scientists and researchers with expertise in protein biology and drug development.

Financial Performance

Scholar Rock is a publicly traded company with a market capitalization of approximately $1 billion. The company has a strong financial position with over $200 million in cash and equivalents. Scholar Rock is well-funded to continue its clinical development programs and advance its pipeline of product candidates.

Overall

Scholar Rock Holding Corporation is a promising clinical-stage biopharmaceutical company with a strong value proposition. The company's proprietary platform technology, pipeline of product candidates, and market opportunity position it to be a leader in the development of new therapies for cancer and neuromuscular disorders.

Scholar Rock Holding Corporation is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases, including cancer, fibrosis, and neuromuscular disorders. The company's lead product candidate, apitegromab, is a fully human monoclonal antibody that targets the activin receptor type IIB (ActRIIB), a protein that plays a key role in regulating cellular growth and differentiation. Apitegromab is currently being evaluated in clinical trials for the treatment of various fibrotic diseases, including idiopathic pulmonary fibrosis (IPF) and diabetic kidney disease (DKD).

Risks Associated with Scholar Rock Holding Corporation:

1. Clinical Development Risk:

• Apitegromab is still in the clinical development stage, and there is no guarantee that it will be successful in clinical trials or receive regulatory approval.
• The company may encounter unexpected safety or efficacy issues during clinical development, which could delay or halt the development of apitegromab.

2. Competition Risk:

• Scholar Rock Holding Corporation faces competition from other companies developing treatments for fibrotic diseases.
• Existing and emerging therapies may compete with apitegromab in the market, reducing its potential commercial success.

3. Market Risk:

• The market for fibrotic disease treatments is highly competitive and rapidly evolving.
• Changes in reimbursement policies, patient preferences, or the introduction of new therapies could impact the demand for apitegromab.

4. Financial Risk:

• Scholar Rock Holding Corporation is a pre-revenue company and relies on funding from investors to support its operations.
• The company may experience financial challenges if it is unable to raise additional capital or generate sufficient revenue from its products.

5. Regulatory Risk:

• The company's products are subject to regulatory oversight by various agencies worldwide.
• Changes in regulatory requirements or delays in the approval process could impact the company's ability to commercialize its products.

6. Intellectual Property Risk:

• Scholar Rock Holding Corporation's patents and other intellectual property rights are essential to its business.
• Challenges to the company's intellectual property could limit its ability to protect its products and generate revenue.

7. Operational Risk:

• The company's operations are subject to various risks, including supply chain disruptions, manufacturing issues, and data breaches.
• These risks could impact the company's ability to produce and deliver its products.

8. Key Personnel Risk:

• Scholar Rock Holding Corporation is dependent on its key executives and scientists.
• The loss or departure of these individuals could negatively impact the company's operations and strategic direction.

9. Political and Economic Risk:

• The company's operations are subject to political and economic factors in the countries where it operates.
• Changes in government policies, economic conditions, or geopolitical events could impact the company's business.

Investors should carefully consider these risks before investing in Scholar Rock Holding Corporation.