INZY Inozyme Pharma Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
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INZ-701
ABCC6 deficiency
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Phase 1/2
Phase 1/2
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INZ-701
ENPP1 deficiency
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Phase 1/2
Phase 1/2
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Latest News
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– Submitted CTA for INZ-701 for the treatment of ENPP1 deficiency to United Kingdom regulatory agency –
– Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency –
– Expect to initiate INZ-701 Phase 1/2 clinical trials for ENPP1 and ABCC6 deficiencies in first half of 2021 –
BOSTON, Nov. 12, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today reported financial results for the third quarter ended September 30, 2020 and provided recent business highlights.
"ENPP1 deficiency is a systemic, progressive and continuous disease occurring over the course of a patient's lifetime, starting as early as fetal development and spanning into adulthood. The fact that INZ-701 had previously received orphan drug designation and now rare pediatric disease and fast track designations underscores the significant unmet medical need for a treatment for this disease," said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. "I'm pleased with the progress we have made with U.S. and European regulatory authorities, and we remain on track to initiate our planned Phase 1/2 clinical trials in the first half of 2021, subject to clearance of our regulatory applications."
Recent Business Highlights
- Submitted Clinical Trial Application (CTA) for INZ-701 for the treatment of ENPP1 deficiency – Inozyme recently submitted its first CTA to initiate a Phase 1/2 clinical trial of INZ-701 for the treatment of ENPP1 deficiency to the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA).
- Received Rare Pediatric Disease Designation and Fast Track Designation from the U.S. Food and Drug Administration (FDA) for INZ-701 for the treatment of ENPP1 deficiency – The FDA grants rare pediatric disease designation to drugs for serious and life-threatening diseases in which the serious or life-threatening manifestations primarily affect children aged from birth through 18 years and affect fewer than 200,000 people in the U.S. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives approval of a biologics license application (BLA) for a rare pediatric disease product application may be eligible for a voucher which can be redeemed to obtain priority review for a subsequent marketing application for a different product. Separately, Fast Track Designation facilitates the potential expedited development and review of a drug for the treatment of a serious or life-threatening disease and that has demonstrated the potential to address unmet medical needs. Benefits of this designation include frequent engagements with the FDA to discuss the drug's clinical development plan, eligibility for priority review, and a rolling review of a BLA. Previously, the FDA and the European Medicines Agency (EMA) had granted orphan drug designation to INZ-701 for the treatment of ENPP1 deficiency.
- Completed disease burden study in ENPP1 deficiency and ABCC6 deficiency – Inozyme and GACI Global, a patient advocacy organization dedicated to bettering the lives of families affected by Generalized Arterial Calcification of Infancy and/or Autosomal Recessive Hypophosphatemic Rickets Type 2 (GACI/ARHR2), completed a study to characterize the burden of disease and understand the systemic progression of disease for the rare genetic diseases of both ENPP1 deficiency and ABCC6 deficiency from the perspective of a patient and/or parent. Inozyme expects to share data from this study in 2021.
Upcoming Anticipated Milestones, Subject to COVID-19 Dynamics
- INZ-701 for ENPP1 deficiency
- Early 2021: Clearance of IND and CTAs
- H1 2021: Initiation of Phase 1/2 clinical trial
- H1 2021: Initiation of prospective natural history study
- H2 2021: Preliminary safety and biomarker data from Phase 1/2 clinical trial
- INZ-701 for ABCC6 deficiency
- Early 2021: Clearance of CTAs
- H1 2021: Initiation of Phase 1/2 clinical trial
- H2 2021: Preliminary safety and biomarker data from Phase 1/2 clinical trial
Upcoming Investor Conference
- Piper Sandler 32nd Annual Healthcare Conference, November 30 – December 3, 2020
Third Quarter 2020 Financial Results
- Cash Position and Financial Guidance – Cash, cash equivalents and investments were $171.7 million as of September 30, 2020. Based on its current plans, the Company expects that its existing cash, cash equivalents and investments will be sufficient to enable it to fund its operating expenses and capital expenditure requirements at least into the second half of 2022.
- Research and Development (R&D) Expenses– R&D expenses were $25.2 million for the third quarter ended September 30, 2020, compared to $3.3 million for the same period in 2019. The increase was primarily due to an increase of $17.8 million resulting from the non-recurring, non-cash purchase of in-process research and development intellectual property assets from Alexion in exchange for stock of the Company in July 2020, costs associated with preclinical studies and clinical preparation activities with the Company's CRO, and growth in the number of R&D employees.
- General and Administrative (G&A) Expenses – G&A expenses were $3.1 million for the third quarter ended September 30, 2020, compared to $1.0 million for the same period in 2019. The increase was primarily due to the growth in the number of G&A employees, an increase in legal fees related to patents, new contracts and operations as a public company, and generally higher fees in areas such as audit, tax and information technology to support the Company's growth.
- Net Loss – Net loss was $28.1 million, or $1.55 loss per share, for the third quarter ended September 30, 2020, compared to $4.0 million, or $3.38 loss per share, for the same period in 2019.
About Inozyme Pharma
Inozyme Pharma, Inc. (NASDAQ:INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.
Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our future clinical trials, our research and development programs, the availability of preclinical study and clinical trial data, the timing of our regulatory applications and the period over which we believe that our existing cash, cash equivalents and investments will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to successfully resolve the clinical hold with regard to its planned Phase 1/2 clinical trial of INZ-701 for ENPP1 deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.
Condensed Consolidated Balance Sheet Data
(Unaudited)(in thousands)
September 30,
2020December 31,
2019Cash, cash equivalents and investments $ 171,709 $ 47,132 Total assets 178,993 47,944 Total liabilities 11,077 3,236 Convertible preferred stock — 77,927 Additional paid-in-capital 247,872 1,428 Accumulated deficit (79,958 ) (34,652 ) Total stockholders' equity (deficit) 167,916 (33,219 ) Condensed Consolidated Statements of Operations and Comprehensive Loss
(Unaudited)(in thousands, except share and per share data)
Three Months Ended September 30, Nine Months Ended September 30, 2020 2019 2020 2019 Operating expenses: Research and development $ 25,174 $ 3,317 $ 39,457 $ 10,941 General and administrative 3,142 1,003 6,313 3,097 Total operating expenses 28,316 4,320 45,770 14,038 Loss from operations (28,316 ) (4,320 ) (45,770 ) (14,038 ) Other income (expense): Interest income 64 288 306 892 Other income (expense), net 157 (3 ) 158 (34 ) Other income (expense), net 221 285 464 858 Net loss $ (28,095 ) $ (4,035 ) $ (45,306 ) $ (13,180 ) Other comprehensive (loss) income: Unrealized (losses) gains on available-for-sale securities (13 ) (2 ) (5 ) 8 Total other comprehensive (loss) income (13 ) (2 ) (5 ) 8 Comprehensive loss $ (28,108 ) $ (4,037 ) $ (45,311 ) $ (13,172 ) Net loss attributable to common stockholders—basic and diluted $ (28,095 ) $ (4,035 ) $ (45,306 ) $ (13,180 ) Net loss per share attributable to common stockholders—basic and diluted $ (1.55 ) $ (3.38 ) $ (6.57 ) $ (11.20 ) Weighted-average common shares outstanding—basic and diluted 18,101,496 1,195,309 6,893,745 1,176,769 Investors:
Brian Luque, Director, Investor Relations
(951) 206-1200
[email protected]Media:
Alex Van Rees, SmithSolve
(973) 442-1555 ext. 111
[email protected]
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BOSTON, Nov. 09, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced the appointment of Kevin B. Johnson, Ph.D., MBA, as senior vice president, regulatory affairs, effective immediately.
Dr. Johnson brings to Inozyme more than 25 years of experience in developing and implementing global regulatory and clinical development strategies for rare diseases across entire product development lifecycles from preclinical through clinical development and ultimately to product approval for a variety of drugs, biologics, combination products and cell/gene therapies. Dr. Johnson will be responsible for leading Inozyme's global regulatory strategy.
"We are thrilled to have Kevin join the Inozyme leadership team during this period of steady execution and as we prepare to enter clinical development," said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. "Kevin is a talented regulatory affairs executive with deep experience in rare diseases and I look forward to Kevin's contributions in helping Inozyme meet the needs of underserved patient communities."
Dr. Johnson joins Inozyme from Magenta Therapeutics, Inc., where he served as the senior vice president, head of regulatory and quality, and led global strategy for a portfolio of biologics. Prior to that, he served as senior vice president, head of regulatory affairs, quality and pharmacovigilance at IMARA Inc., during which time the company received several regulatory designations for orphan diseases such as sickle cell disease and beta-thalassemia. Before IMARA, Dr. Johnson led global regulatory strategies at Vtesse (later acquired by Sucampo), addressing ultra-rare diseases such as Niemann-Pick disease type C under Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) and Promising Innovative Medicine designation from the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA). Dr. Johnson also served as the director, global regulatory affairs for rare diseases and gene therapies at GlaxoSmithKline, working on the international regulatory team for the European approval of the gene therapy Strimvelis® for ADA-SCID, and which team subsequently received Regenerative Medicine Advanced Therapy (RMAT) designation for a retinal gene therapy product.
"Based on the compelling science and preclinical research conducted with INZ-701, Inozyme has the potential to help patients with devastating and debilitating rare metabolic diseases who currently lack effective treatment options," said Dr. Johnson. "I am excited to join Inozyme at such an important time in the company's growth trajectory, and I look forward to contributing to Inozyme's success."
Dr. Johnson holds a Ph.D. in Neurobiology from the University of North Carolina School of Medicine, an MBA from the Kenan-Flagler School of Business at the University of North Carolina, and a B.S. in Chemistry from the University of South Florida. Dr. Johnson also holds a Regulatory Affairs Certification (RAC) credential from the Regulatory Affairs Professional Society.
About Inozyme Pharma
Inozyme Pharma is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.
Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to successfully resolve the clinical hold with regard to its planned Phase 1/2 clinical trial of INZ-701 for ENPP1 deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.
Investors:
Brian Luque, Director, Investor Relations
(951) 206-1200
[email protected]Media:
Alex Van Rees, SmithSolve
(973) 442-1555 ext. 111
[email protected] -
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– Dr. Sabbagh Brings More Than 20 Years of Experience in Rare Genetic Disorders and Mineral Metabolism Research Development Programs –
– Company Announces Retirement of David Thompson, Ph.D. –
– Company to Open New Laboratory Space to Increase R&D Capabilities –
BOSTON, Oct. 13, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced the appointment of Yves Sabbagh, Ph.D., as senior vice president and chief scientific officer, and the retirement of David Thompson, Ph.D., Inozyme's former chief scientific officer.
Dr. Sabbagh brings to Inozyme more than 20 years of experience in rare genetic disorders and mineral metabolism with responsibilities leading to the identification and evaluation of novel therapeutic approaches and translating them into clinical candidates. Dr. Sabbagh will be responsible for expanding Inozyme's proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization.
"Yves is an accomplished and well-regarded scientist in the areas of renal, bone and mineral research and brings to Inozyme extensive management experience leading teams developing highly innovative drug candidates," said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. "I want to express my deep appreciation for David's extensive contributions over the last several years. He has been instrumental in establishing the research group at Inozyme and our portfolio of indications for INZ-701. We look forward to maintaining an active relationship with David in his new role as senior advisor to the Company and wish him the very best in his retirement."
Prior to joining Inozyme, Dr. Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi. Prior to that executive role, he held scientific roles of increasing responsibility at Sanofi and Genzyme Corporation spanning endocrine, renal and rare bone diseases including driving the strategy for bone indications. Prior to his corporate experience, he was an instructor at the Harvard Medical School in the Endocrine unit.
"Based on the compelling science and the quality of translational research conducted in mineralization disorders with an initial focus on ENPP1 and ABCC6 deficiencies, Inozyme has the potential to help patients with devastating and debilitating rare diseases that currently lack effective treatment options," said Dr. Sabbagh. "I am excited to be joining Inozyme at such an important time in its growth trajectory. And I look forward to leveraging the company's new lab infrastructure in Boston's innovative Seaport District to contribute to Inozyme's continued success and bring transformative therapies to patients."
Dr. Sabbagh has co-authored more than 30 peer-reviewed publications and book chapters and is a member of several scientific societies. Dr. Sabbagh received a B.Sc. in biochemistry from McGill University, an MSc in microbiology from Université Laval and a Ph.D. in biology from McGill University.
In conjunction with Dr. Sabbagh's appointment, Dr. Thompson announced his retirement as senior vice president and chief scientific officer. Dr. Thompson will step down from his role following a transition period and will remain associated with the Company in his role as senior advisor.
About Inozyme Pharma
Inozyme Pharma is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of potentially first-in-class therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.
Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to successfully resolve the clinical hold with regard to its planned Phase 1/2 clinical trial of INZ-701 for ENPP1 deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.
Investors:
Brian Luque, Director, Investor Relations
(951) 206-1200
[email protected]Media:
Alex Van Rees, SmithSolve
(973) 442-1555 ext. 111
[email protected] -
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- Longitude Venture Partners IV will build on the Firm's commitment to improve clinical outcomes, enhance patient quality of life, and drive efficiency in healthcare delivery -
- In 2020 to date, Longitude portfolio companies announced 4 IPOs and 3 M&A transactions -
MENLO PARK, Calif. and GREENWICH, Conn. and BOSTON, Sept. 23, 2020 (GLOBE NEWSWIRE) -- Longitude Capital, a leading healthcare venture capital firm, today announced the closing of Longitude Venture Partners IV, L.P. ("LVP4"), with $585 million in capital commitments. LVP4, the largest fund that Longitude Capital has raised to date, will build on the proven strategy of its predecessor funds by investing in biotechnology, medical technology, and health solutions companies that seek to transform the healthcare industry.
"We are in the golden era of medical and biological innovation. The significant unmet clinical needs and the inefficiencies of our current healthcare system are driving meaningful scientific breakthroughs and creative solutions," said Patrick Enright, co-founder and Managing Director of Longitude Capital. Since inception in 2006, Longitude Capital has raised nearly $2 billion in cumulative capital commitments and managed over 30 company exits. "We are fortunate to have helped build many successful healthcare companies and look forward to working alongside the next wave of entrepreneurs and scientists to advance critical medicines, devices, and health solutions to the marketplace."
Juliet Bakker, co-founder and Managing Director of Longitude Capital, added, "We are thankful for the continued support of, and partnerships with, our investors who value our differentiated venture growth investing approach, experienced team, and passion for forging new frontiers in healthcare."
LVP4 will invest opportunistically across all stages of a company's development through a variety of approaches that include traditional venture capital investing and special situations such as spin-outs, equity-linked transactions, and private investments in public equities. Many of these investments stem from Longitude's proprietary research of targeted healthcare sectors, therapeutic areas, and technologies of interest.
Longitude Capital's recent Initial Public Offerings (IPOs) and exits include 89bio (NASDAQ:ETNB), Aimmune (NASDAQ:AIMT, recently agreed to be acquired by Nestlé Health Science))), Axonics Modulation Technologies (NASDAQ:AXNX), Checkmate Pharmaceuticals (NASDAQ:CMPI), Inflazome (acquired by Roche), Inozyme Pharma (NASDAQ:INZY), KaNDy Therapeutics (acquired by Bayer), Molecular Templates (NASDAQ:MTEM), Poseida Therapeutics (NASDAQ:PSTX), and Vaxcyte (NASDAQ:PCVX).
About Longitude Capital
Longitude Capital is a leading healthcare venture capital firm, that invests in transformative biotechnology, medical technology, and health solutions companies seeking to improve clinical outcomes, enhance quality of life, and drive efficiency of healthcare delivery. Founded in 2006, Longitude Capital invests in both privately held and publicly traded companies through a variety of investment approaches. Longitude Capital has offices in Menlo Park, CA, Greenwich, CT, and Boston, MA. For more information, including a complete listing of investments, please visit www.longitudecapital.com.
About Longitude Capital Recent IPOs and Exits
- 89bio is a biopharmaceutical company developing and commercializing innovative therapies for the treatment of liver and cardio-metabolic diseases. 89bio was originally spun out of Teva Pharmaceuticals in 2018 by founding investors Longitude Capital and OrbiMed, and completed its IPO in November 2019. www.89bio.com.
- Aimmune is a biopharmaceutical company developing and commercializing treatments for potentially life-threatening food allergies. Aimmune's PALFORZIA® is the world's first approved treatment for peanut allergy. Longitude Capital was the founding institutional investor of Aimmune. In 2020, Aimmune agreed to be acquired by Nestlé Health Science for $34.50 per share in cash, representing a total equity value of $2.6 billion. www.aimmune.com.
- Axonics Modulation Technologies is a medical technology company developing and commercializing novel implantable rechargeable sacral neuromodulation (SNM) devices for patients with urinary and bowel dysfunction. In 2018, Longitude Capital led a $40 million financing that preceded the company's IPO later that year. www.axonics.com.
- Checkmate Pharmaceuticals is a clinical-stage biopharmaceutical company developing proprietary technology to harness the power of the immune system to combat cancer. In 2020, Longitude Capital and Novo Holdings led the company's $85 million Series C financing that preceded the company's IPO later in the year. www.checkmatepharma.com.
- Inflazome is a biotechnology company developing small molecules that block harmful inflammation by targeting inflammasomes, protein complexes that generate signals in order to activate an immune response. In 2020, Inflazome announced its acquisition by Roche for an upfront payment of €380 million, and potential predetermined milestone payments. www.inflazome.com.
- Inozyme Pharma is a rare disease pharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization. Longitude Capital led Inozyme's $49 million Series A financing in 2017, and was joined by New Enterprise Associates, Novo Holdings, and Sanofi Ventures. Inozyme completed its initial public offering in 2020. www.inozyme.com.
- KaNDy Therapeutics was a UK-based clinical-stage company focused on optimizing the potential of its unique NK-1,3 receptor antagonist NT-814 to treat common, chronic debilitating conditions related to menopause. In 2020, KaNDy Therapeutics was acquired by Bayer Pharmaceuticals for an upfront payment of $425 million, and a potential $450 million in R&D, regulatory milestones and additional commercial milestones.
- Molecular Templates is a clinical-stage company focused on the discovery and development of targeted biologic therapeutics. In 2017, Longitude Capital led Molecular Template's $40 million PIPE (private investment into public equity) financing. Molecular Templates now has strategic collaborations with Takeda Pharmaceuticals and Vertex Pharmaceuticals. www.mtem.com.
- Poseida Therapeutics is a clinical-stage biopharmaceutical company dedicated to utilizing its proprietary gene engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure various cancers. Longitude Capital led Poseida's $30 million Series B financing in 2018. Poseida completed its IPO in 2020. www.poseida.com.
- Vaxcyte is a next-generation vaccine company seeking to improve global health by developing superior and novel vaccines designed to prevent or treat some of the most common and deadly infectious diseases worldwide. Longitude Capital invested in Vaxcyte's $22 million Series A financing and served on the Board of the company through its IPO in 2020. www.vaxcyte.com.
About Longitude Capital Recent Investments
- Eargo is a medical device company dedicated to improving the quality of life of people with hearing loss. Longitude Capital and Gilde Healthcare led Eargo's $81 million Series E financing in 2020. www.eargo.com.
- Epirium Bio is a clinical-stage biopharmaceutical company that uses insights related to the biology of mitochondrial function and tissue regeneration to pursue novel and clinically significant therapeutic approaches for neuromuscular, neurodegenerative, and mitochondrial disorders. Longitude Capital and ARCH Venture Partners led Epirium's $85 million Series A financing in 2019. www.epirium.com.
- Polares Medical is a clinical-stage medical technology company focused on the development of a unique trans-catheter mitral valve hemi-replacement system to treat patients suffering from mitral regurgitation (MR). Longitude Capital led Polares Medical's $40 million Series B financing in 2020. www.polaresmedical.com.
- WelbeHealth is a services company dedicated to unlocking the full potential of vulnerable seniors through PACE (Program of All-Inclusive Care for the Elderly), a comprehensive medical and social care model. Longitude Capital and .406 Ventures led WelbeHealth's $30 million Series C in 2020. www.welbehealth.com.
Source: Longitude Capital
Contact Information Maggie Jamison Longitude Capital 650-854-5700 [email protected]
- 89bio is a biopharmaceutical company developing and commercializing innovative therapies for the treatment of liver and cardio-metabolic diseases. 89bio was originally spun out of Teva Pharmaceuticals in 2018 by founding investors Longitude Capital and OrbiMed, and completed its IPO in November 2019. www.89bio.com.
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Upsized IPO in July 2020 raised $128.8 million in gross proceeds
Submitted IND for INZ-701 for the treatment of ENPP1 deficiency; currently on FDA clinical hold pending completion of ongoing GLP toxicology studies
Initiation of INZ-701 Phase 1/2 clinical trials anticipated in early 2021, as previously planned
BOSTON, Sept. 03, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today reported financial results for the second quarter ended June 30, 2020 and provided recent business highlights.
"We have made substantial progress over the past quarter and in July, including completing our upsized initial public offering in July, acquiring additional ENPP1 deficiency program assets, and submitting our first investigational new drug application (IND) for INZ-701 for the treatment of ENPP1 deficiency," said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. "FDA has requested final study reports for our ongoing three-month GLP toxicology studies in mice and non-human primates and has placed our planned Phase 1/2 clinical trial of INZ-701 in adult patients with ENPP1 deficiency in the United States on clinical hold pending submission of the reports. We expect to be able to submit these reports in the fourth quarter of this year and initiate our clinical program, as we had planned, in early 2021."
Recent Business Highlights
- Completed upsized Initial Public Offering – In July 2020, Inozyme completed its initial public offering of 8,050,000 shares of common stock at a public offering price of $16.00 per share, including the full exercise of the underwriter's option to purchase additional shares. Gross proceeds from the IPO were $128.8 million and net proceeds from the offering, after deducting underwriting discounts, commissions and offering expenses, were approximately $116.5 million.
- Submitted an IND for INZ-701 - On July 30, 2020, Inozyme submitted its first IND for INZ-701 for the treatment of ENPP1 deficiency to the U.S. Food and Drug Administration (FDA). At the end of the 30-day FDA review period, the Company was notified that the IND was placed on clinical hold, pending submission of the final study report for its ongoing three-month toxicology studies in mice and non-human primates (NHPs) being performed in accordance with Good Laboratory Practices (GLP) regulations. Inozyme initiated these GLP toxicology studies prior to submission of the IND and expects to complete the studies and have reports available in the fourth quarter of 2020. The FDA did not request any additional preclinical data to resolve the clinical hold other than the submission of the final study report for the three-month GLP toxicology studies in mice and NHPs. Subject to the submission of the final study reports for the three-month toxicology studies as recommended by the FDA and the successful resolution of the clinical hold, the Company expects to initiate its Phase 1/2 trials in early 2021 and report initial safety and biomarker data in 2021, as it had planned.
- Expanded and strengthened Board of Directors with appointment of Doug Treco, Ph.D., as Chairman and Lynne Sullivan, MST as an Independent Director – In May 2020, Doug Treco joined Inozyme's Board as chairman and Lynne Sullivan joined the Board as an independent director. Dr. Treco co-founded Ra Pharmaceuticals, Inc., where he was chief executive officer and a member of the Board of Directors from its inception until the company was acquired in April 2020.
Ms. Sullivan is currently the chief financial officer for UNITY Biotechnology, and she previously served as the chief financial officer of Compass Therapeutics and as a senior vice president of finance at Biogen, where she spent 11 years, with global responsibility for Corporate Finance, Financial Planning and Analysis and Corporate Tax.
- Acquired ENPP1 program assets from Alexion Pharmaceuticals – In July 2020, Inozyme announced the acquisition of intellectual property and assets from Alexion Pharmaceuticals focusing on ENPP1 gene deficiencies. The acquisition complements Inozyme's ongoing development of INZ-701 and expands the Company's intellectual property portfolio.
- Initiated disease burden study in ENPP1 deficiency and ABCC6 deficiency – In May 2020, Inozyme and GACI Global, a patient advocacy organization dedicated to bettering the lives of families affected by Generalized Arterial Calcification of Infancy and/or Autosomal Recessive Hypophosphatemic Rickets Type 2 (GACI/ARHR2), announced the initiation of a study to characterize the burden of disease and understand the systemic progression of disease for the rare genetic diseases of both ENPP1 deficiency and ABCC6 deficiency from the perspective of a patient and/or parent.
- Expanded physical footprint with move to new office – To meet the demands of anticipated growth, Inozyme expanded and enhanced its physical office space in August 2020.
Second Quarter 2020 Financial Results
- Cash Position – Cash, cash equivalents and short-term investments were $63.9 million as of June 30, 2020, as compared to $40.8 million as of March 31, 2020. Total cash, cash equivalents and short-term investments on June 30, 2020 does not include total net proceeds of approximately $116.5 million from the Company's IPO in July 2020. Based on its current plans, the Company expects that its existing cash, cash equivalents and short-term investments, including the proceeds from its July 2020 IPO, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the second half of 2022.
- Research and Development (R&D) Expenses – R&D expenses were $7.9 million for the second quarter ended June 30, 2020, compared to $3.5 million for the same period in 2019. The increase was primarily due to higher consulting and professional fees related to the planned filing of an IND for INZ-701, preclinical studies and clinical preparation activities with the Company's CRO, and growth in the number of R&D employees.
- General and Administrative (G&A) Expenses – G&A expenses were $1.7 million for the second quarter ended June 30, 2020, compared to $1.1 million for the same period in 2019. The increase was primarily due to the growth in the number of G&A employees, an increase in legal fees related to patents and new contracts, and generally higher fees in areas such as audit, tax and information technology to support the Company's growth.
- Net Loss – Net loss was $9.5 million, or $7.57 loss per share, for the second quarter ended June 30, 2020, compared to $4.2 million, or $3.57 loss per share, for the same period in 2019.
About Inozyme Pharma
Inozyme Pharma is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of potentially first-in-class therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.
Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, and timing, of our future clinical trials and our research and development programs, the availability of preclinical study and clinical trial data and the period over which we believe that our existing cash and cash equivalents will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to successfully resolve the clinical hold with regard to its planned Phase 1/2 clinical trial of INZ-701 for ENPP1 deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.
Condensed Consolidated Balance Sheet Data
(Unaudited)(in thousands)
June 30,
2020December 31,
2019Cash, cash equivalents and short-term investments $ 63,867 $ 47,132 Total assets 68,511 47,944 Total liabilities 6,965 3,236 Convertible preferred stock 111,565 77,927 Accumulated deficit (51,863 ) (34,652 ) Total stockholders' deficit (50,019 ) (33,219 ) Condensed Consolidated Statements of Operations and Comprehensive Loss
(Unaudited)(in thousands, except share and per share data)
Three Months Ended June 30, Six Months Ended June 30, 2020 2019 2020 2019 Operating expenses: Research and development $ 7,877 $ 3,489 $ 14,283 $ 7,623 General and administrative 1,671 1,064 3,171 2,094 Total operating expenses 9,548 4,553 17,454 9,717 Loss from operations (9,548 ) (4,553 ) (17,454 ) (9,717 ) Other income (expense): Interest income 71 394 242 604 Other income (expense), net 4 (14 ) 1 (31 ) Other income (expense), net 75 380 243 573 Net loss $ (9,473 ) $ (4,173 ) $ (17,211 ) $ (9,144 ) Other comprehensive (loss) income: Unrealized (losses) gains on available-for-sale securities (15 ) 10 8 12 Total other comprehensive (loss) income (15 ) 10 8 12 Comprehensive loss $ (9,488 ) $ (4,163 ) $ (17,203 ) $ (9,132 ) Net loss attributable to common stockholders—basic
and diluted$ (9,473 ) $ (4,173 ) $ (17,211 ) $ (9,144 ) Net loss per share attributable to common
stockholders—basic and diluted$ (7.57 ) $ (3.57 ) $ (14.01 ) $ (7.83 ) Weighted-average common shares outstanding—basic
and diluted1,251,244 1,170,480 1,228,296 1,167,346 Contacts
Investors:
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Mike Biega
(617) 221 9660
[email protected]Media:
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Alex Van Rees
(973) 442-1555 ext. 111
[email protected] - Completed upsized Initial Public Offering – In July 2020, Inozyme completed its initial public offering of 8,050,000 shares of common stock at a public offering price of $16.00 per share, including the full exercise of the underwriter's option to purchase additional shares. Gross proceeds from the IPO were $128.8 million and net proceeds from the offering, after deducting underwriting discounts, commissions and offering expenses, were approximately $116.5 million.