– Interim information from SEAPORT-1, a Part 1 trial of INZ-701 in sufferers with end-stage kidney illness receiving hemodialysis, on monitor for fourth quarter of 2024 –
– Interim information from ENERGY-1, a Part 1b trial of INZ-701 in infants with ENPP1 Deficiency, on monitor for second half of 2024 –
– Money, money equivalents, and short-term investments as of March 31, 2024, anticipated to fund operations into the fourth quarter of 2025 –
BOSTON, Could 07, 2024 (GLOBE NEWSWIRE) — Inozyme Pharma, Inc. INZY (“the Firm” or “Inozyme”), a clinical-stage uncommon illness biopharmaceutical firm creating novel therapeutics for the therapy of pathologic mineralization and intimal proliferation, in the present day reported monetary outcomes for the primary quarter ended March 31, 2024, and supplied enterprise highlights.
“We have been extraordinarily happy to see preliminary proof of improved vascular well being with INZ-701 therapy in adults with ABCC6 Deficiency, offering sturdy assist for additional scientific improvement on this illness,” mentioned Douglas A. Treco, Ph.D., CEO and Chairman of Inozyme. “We discovered that youngsters with ABCC6 Deficiency are at excessive threat for neurological and visible impairment and characterize probably the most urgent unmet want on this illness, considerably increasing the addressable inhabitants past impacted adults. We stay up for working with regulators to determine a path to approval for our ABCC6 Deficiency program, in addition to presenting extra information from present trials in our calciphylaxis and ENPP1 Deficiency applications.”
Latest Highlights
- Part 1/2 Scientific Trial of INZ-701 in Adults with ABCC6 Deficiency. In April 2024, the Firm announced optimistic topline security and immunogenicity information, with scientific enhancements in vascular pathology, visible perform, and affected person reported outcomes (PROs).
- Pure Historical past Research and Improvement Plans for INZ-701 in Pediatric ABCC6 Deficiency. In April 2024, the Firm reported preliminary findings from pure historical past research which point out a considerable illness burden amongst pediatric sufferers with ABCC6 Deficiency, manifesting as a excessive incidence of main scientific occasions, notably stroke, extreme neurological illness, and extreme heart problems, occurring early in life. Topic to regulatory evaluate and enough funding, the Firm expects to provoke a pivotal trial in pediatric sufferers with ABCC6 Deficiency in Q1 2025.
- Part 1/2 Scientific Trial of INZ-701 in Adults with ENPP1 Deficiency. In April 2024, the Firm announced optimistic topline information indicating that the previously-reported favorable security, immunogenicity, and scientific end result information have been maintained via 48 weeks in Cohorts 1-3. Knowledge from Cohort 4 assist as soon as weekly dosing in ongoing and future scientific trials.
Anticipated Milestones
- ENPP1 Deficiency
- Initiation of the ENERGY-2 pivotal trial in infants, Ex-U.S. – 2H 2024
- Interim information from the ENERGY-1 Part 1b trial in infants – 2H 2024
- Topline information from the ENERGY-3 pivotal trial in pediatric sufferers – Mid-2025
- ABCC6 Deficiency
- Initiation of pivotal scientific trial in pediatric sufferers, topic to regulatory evaluate and enough funding – Q1 2025
- Calciphylaxis
- Interim information from SEAPORT-1 Part 1 trial in sufferers with end-stage kidney illness (ESKD) receiving hemodialysis – This fall 2024
First Quarter 2024 Monetary Outcomes
- Money Place and Monetary Steerage. Money, money equivalents, and short-term investments have been $166.2 million as of March 31, 2024. Primarily based on its present plans, the Firm anticipates its money, money equivalents, and short-term investments as of March 31, 2024, will allow the Firm to fund money circulate necessities into This fall 2025.
- Analysis and Improvement (R&D) Bills. R&D bills have been $19.1 million for the quarter ended March 31, 2024, in comparison with $11.9 million for the prior-year interval.
- Common and Administrative (G&A) Bills. G&A bills have been $5.2 million for the quarter ended March 31, 2024, in comparison with $6.5 million for the prior-year interval.
- Web Loss. Web loss was $23.3 million, or $0.38 loss per share, for the quarter ended March 31, 2024, in comparison with $17.4 million or $0.40 loss per share for the prior-year interval.
About ABCC6 Deficiency
ABCC6 Deficiency is a progressively debilitating situation of the vasculature and comfortable tissue that’s estimated to have an effect on roughly 1 in 25,000 to 1 in 50,000 people worldwide. Infants with ABCC6 Deficiency are identified with generalized arterial calcification of infancy (GACI Sort 2), a situation that resembles GACI Sort 1, the toddler type of ENPP1 Deficiency. Pediatric sufferers who survive the primary yr of life might develop neurological illness, together with stroke, and heart problems secondary to ongoing vascular calcification and stenosis. In older people, ABCC6 Deficiency presents as pseudoxanthoma elasticum (PXE), which is characterised by pathologic mineralization in blood vessels and comfortable tissues clinically affecting the pores and skin, eyes, and vascular system. There are not any authorized therapies for ABCC6 Deficiency.
About ENPP1 Deficiency
ENPP1 Deficiency is a progressively debilitating situation of the vasculature, comfortable tissue, and skeleton with a prevalence of roughly 1 in 64,000 pregnancies worldwide. Though ENPP1 Deficiency was initially described in sufferers with biallelic ENPP1 Deficiency (homozygous or compound heterozygous mutations), many sufferers with monoallelic ENPP1 Deficiency (heterozygous mutations) have scientific signs, probably rising the worldwide prevalence. People who current in utero or in infancy are sometimes identified with generalized arterial calcification of infancy (GACI Sort 1) and roughly 50% of infants die inside six months of delivery. Kids with ENPP1 Deficiency sometimes develop rickets, a situation identified as autosomal-recessive hypophosphatemic rickets kind 2 (ARHR2), whereas adolescents and adults can develop osteomalacia (softened bones). ARHR2 and osteomalacia result in ache and mobility points. Sufferers also can exhibit indicators and signs of listening to loss, arterial and joint calcification, and cardiovascular problems. There are not any authorized therapies for ENPP1 Deficiency.
About Calciphylaxis
Calciphylaxis is a uncommon dysfunction with a excessive mortality charge that largely impacts sufferers with end-stage kidney illness (ESKD). The illness is related to low ranges of pyrophosphate (PPi) and is characterised by pathologic mineralization (i.e., calcification) and intimal proliferation (the overgrowth of easy muscle cells inside blood vessels) of the vasculature within the pores and skin and fatty tissue resulting in poor blood circulate, blood clots, painful pores and skin ulcers, critical infections, and dying. Sufferers with calciphylaxis have a reported one-year survival charge of roughly 50%. The estimated incidence of calciphylaxis is roughly 3.5 per 1,000 sufferers with ESKD with roughly 5,000 new sufferers presenting yearly throughout main geographies. There are not any authorized therapies for calciphylaxis.
About INZ-701
INZ-701, a recombinant Fc fusion protein, is an ENPP1 enzyme substitute remedy (ERT) in improvement for the therapy of uncommon problems of the vasculature, comfortable tissue, and skeleton. INZ-701 metabolizes adenosine triphosphate (ATP) to generate PPi, a pure inhibitor of mineralization, and AMP, which may be processed to phosphate and adenosine, the latter being a pure inhibitor of intimal proliferation. In preclinical research, the experimental remedy has proven potential to forestall pathologic mineralization and intimal proliferation, which may drive morbidity and mortality in devastating problems reminiscent of, ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis. Scientific information to this point have demonstrated that INZ-701 was usually nicely tolerated, exhibited a positive security profile, and meaningfully elevated PPi ranges in a number of scientific trials.
About Inozyme Pharma
Inozyme Pharma, Inc. is a clinical-stage uncommon illness biopharmaceutical firm creating novel therapeutics for the therapy of illnesses impacting the vasculature, comfortable tissue, and skeleton. Inozyme is creating INZ-701, an enzyme substitute remedy, to deal with pathologic mineralization and intimal proliferation, which may drive morbidity and mortality in these extreme illnesses. INZ-701 is at present in scientific improvement for the therapy of ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis.
For extra data, please go to https://www.inozyme.com/ or observe Inozyme on LinkedIn, X, and Facebook.
Cautionary Observe Relating to Ahead-Trying Statements
Statements on this press launch about future expectations, plans, and prospects, in addition to another statements relating to issues that aren’t historic information, might represent “forward- wanting statements” throughout the that means of The Personal Securities Litigation Reform Act of 1995.
These statements embody, however aren’t restricted to, statements regarding the initiation, timing, and design of our deliberate scientific trials, the provision and timing of information from scientific trials, the potential advantages of INZ-701, our regulatory technique, together with our plan to work with regulators to determine a path to approval for our ABCC6 Deficiency program, and the interval over which we imagine that our current money, money equivalents, and short-term investments can be enough to fund our money circulate necessities. The phrases “anticipate,” “imagine,” “proceed,” “might,” “estimate,” “anticipate,” “intend,” “might,” “plan,” “potential,” “predict,” “challenge,” “ought to,” “goal,” “will,” “would,” and related expressions are supposed to determine forward-looking statements, though not all forward-looking statements comprise these figuring out phrases. Any forward-looking statements are based mostly on administration’s present expectations of future occasions and are topic to various dangers and uncertainties that would trigger precise outcomes to vary materially and adversely from these set forth in, or implied by, such forward-looking statements. These dangers and uncertainties embody, however aren’t restricted to, dangers related to the Firm’s skill to conduct its ongoing scientific trials of INZ-701 for ABCC6 Deficiency, ENPP1 Deficiency, and calciphylaxis; enroll sufferers in ongoing and deliberate trials; receive and preserve crucial approvals from the Meals and Drug Administration and different regulatory authorities; proceed to advance its product candidates in preclinical research and scientific trials; replicate in later scientific trials optimistic outcomes present in preclinical research and early-stage scientific trials of its product candidates; advance the event of its product candidates beneath the timelines it anticipates in deliberate and future scientific trials; receive, preserve, and shield mental property rights associated to its product candidates; handle bills; adjust to the covenants beneath its excellent mortgage settlement; and lift the substantial extra capital wanted to attain its enterprise goals. For a dialogue of different dangers and uncertainties, and different vital components, any of which might trigger the Firm’s precise outcomes to vary from these contained within the forward-looking statements, see the “Threat Components” part within the Firm’s most up-to-date Annual Report on Type 10-Okay and Quarterly Report on Type 10-Q filed with the Securities and Trade Fee, in addition to discussions of potential dangers, uncertainties, and different vital components, within the Firm’s most up-to-date filings with the Securities and Trade Fee. As well as, the forward-looking statements included on this press launch characterize the Firm’s views as of the date hereof and shouldn’t be relied upon as representing the Firm’s views as of any date subsequent to the date hereof. The Firm anticipates that subsequent occasions and developments will trigger the Firm’s views to alter. Nonetheless, whereas the Firm might elect to replace these forward-looking statements sooner or later sooner or later, the Firm particularly disclaims any obligation to take action.
Condensed Consolidated Stability Sheet Knowledge
(Unaudited)
| March 31, 2024 | December 31, 2023 | ||||||
| Money, money equivalents, and investments | $ | 166,153 | $ | 188,589 | |||
| Whole Belongings | $ | 176,943 | $ | 200,847 | |||
| Whole Liabilities | $ | 58,107 | $ | 60,368 | |||
| Further paid-in-capital | $ | 428,212 | $ | 426,362 | |||
| Accrued deficit | $ | (309,277 | ) | $ | (285,930 | ) | |
| Whole Stockholders’ Fairness | $ | 118,836 | $ | 140,479 | |||
Condensed Consolidated Statements of Operations and Complete Loss
(Unaudited)
| Three Months Ended March 31, | ||||||||
| 2024 | 2023 | |||||||
| Working bills: | ||||||||
| Analysis and improvement | $ | 19,111 | $ | 11,857 | ||||
| Common and administrative | 5,234 | 6,512 | ||||||
| Whole working bills | 24,345 | 18,369 | ||||||
| Loss from operations | (24,345 | ) | (18,369 | ) | ||||
| Different earnings (expense): | ||||||||
| Curiosity earnings | 2,374 | 1,327 | ||||||
| Curiosity expense | (1,325 | ) | (328 | ) | ||||
| Different bills | (51 | ) | (34 | ) | ||||
| Different earnings (expense), web | 998 | 965 | ||||||
| Web loss | $ | (23,347 | ) | $ | (17,404 | ) | ||
| Different complete (loss) earnings: | ||||||||
| Unrealized (losses) beneficial properties on available-for-sale securities | (156 | ) | 150 | |||||
| International forex translation adjustment | 10 | 19 | ||||||
| Whole different complete (loss) earnings | (146 | ) | 169 | |||||
| Complete loss | $ | (23,493 | ) | $ | (17,235 | ) | ||
| Web loss attributable to widespread stockholders—primary and diluted |
$ | (23,347 | ) | $ | (17,404 | ) | ||
| Web loss per share attributable to widespread stockholders—primary and diluted |
$ | (0.38 | ) | $ | (0.40 | ) | ||
| Weighted-average widespread shares excellent—primary and diluted |
61,772,279 | 43,720,578 | ||||||
Contacts
Traders:
Inozyme Pharma
Stefan Riley, Senior Director of IR and Company Communications
(857) 330-8871
[email protected]
Media:
SmithSolve
Matt Pera
(973) 886-9150
[email protected]
