For years, market watchers have speculated about some biopharma giant or other buying out BioMarin Pharmaceutical (NASDAQ: BMRN), which is why the rare disease drug developer has appeared in every GEN A-List of Top 10 Takeover Targets, from 2013 through the latest list in February 2025.
This past week, however, BioMarin took the position of buyer, by agreeing to shell out approximately $270 million for Inozyme Pharma (NASDAQ: INZY), a developer whose treatments target the PPi-adenosine pathway, a key regulator of bone health and blood vessel function.
Inozyme investors roared their approval of the deal with a buying surge that sent the company’s shares nearly tripling, rocketing 178% on Friday from $1.42 to $3.95. BioMarin investors, however, were far more blasé about the deal, with their stock rising nearly 2%, from $58.27 to y$59.27—even after BioMarin ballyhooed the deal as a “strong strategic fit.”
BioMarin reasons that Inozyme’s Phase III lead candidate, INZ-701, is a near-perfect complement to the BioMarin pipeline of 10 programs across six rare disease treatment candidates.
“Like five of our six enzyme therapies, it would be a first-in-disease treatment,” BioMarin president and CEO Alexander Hardy told analysts Friday on a conference call about the deal. “Adding this investigational therapy to our enzyme therapies portfolio is a natural addition to our late-stage pipeline, and, if data supported, we will leverage our regulatory and commercialization know-how in countries across the world to provide access to INZ-701 to patients across our global footprint.”
Combining the active part of the ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme with a fragment of a human antibody (Fc), INZ-701 is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) designed to treat ENPP1 deficiency in both children and adults.
INZ-701 converts adenosine triphosphate (ATP) into inorganic pyrophosphate (PPi) and adenosine monophosphate (AMP). AMP is further broken down into adenosine and phosphate. By targeting ENPP1, INZ-701 can treat multiple rare diseases driven by disruptions in the PPi-Adenosine Pathway and thus restore balance across the pathway.
“Bigger picture, we see this deal offering a clear strategic fit within BMRN’s rare disease portfolio with a purchase price that leaves firepower leftover for additional deals,” Jessica Fye, a managing director and senior equity research analyst for J.P. Morgan, wrote Friday in a research note.
Fye, who specializes in small- and mid-cap biotech stocks, noted astutely that BioMarin characterized as its first deal of the year the planned purchase of Inozyme, “suggesting they don’t see it as the only one.”
On the call with analysts to discuss the Inozyme acquisition, BioMarin executives discussed the potential for additional mergers and acquisitions (M&A).
“Management continues to believe the current environment remains favorable for further pipeline expansion through business development and anticipates more deal flow in the future,” William Blair analyst Sami Corwin, PhD, reported Friday in a research note.
Hardy communicated BioMarin’s interest in growth through acquisitions two weeks earlier on the company’s most recent earnings call.
“What we’re looking at is things which are completely lined up with what BioMarin is really good at. So, genetically defined conditions, we’re looking at clinical stage assets in particular as well as obviously earlier assets, but we’ve always done that,” Hardy told analysts on May 1. “We continue to focus on doing at least one business development deal this year.”
BioMarin finished the first quarter with net income of $185.686 million, up more than double (109%) from $88.662 million in Q1 2024, on revenue that jumped about 15% year-over-year to $745.145 million from $648.833 million. Inozyme announced first quarter results on Wednesday, reporting a $28.039 million net loss, up 20% from its $23.347 million net loss of a year earlier, with no reported revenue.
On Wednesday, Inozyme stock climbed 24% from $1.09 to $1.35 after combining its first quarter results with a positive update on INZ-701. The company released new interim data from its pivotal Phase III ENERGY 3 trial (NCT06046820) that it said highlighted INZ-701’s potential to modify disease course in ENPP1 deficiency by addressing its underlying biology.
Effectiveness was seen consistently across safety, immunogenicity, and phosphate level increases, Inozyme said. At week 13, for example, mean serum phosphate levels increased from baseline by +8.2% in the 17-patient INZ-701 arm vs. an 0.04% dip in the conventional treatment arm, with 35% (6 of the 17 patients) achieving normal phosphate levels at least once (vs. 0% with standard care)—an outcome suggesting potential improvement of rickets.
ENERGY 3 is set to release topline data in the first quarter of 2026. If positive, that data is expected to support an FDA filing seeking approval for INZ-701 in pediatric ENPP1 deficiency, with Inozyme anticipating launching the drug in that indication in 2027.
H.C. Wainwright analyst Edward White has projected first-year sales for INZ-701 of $46.6 million, with Inozyme forecasting peak-year revenue of between $400 and $600 million by the mid-2030s.
To reach that revenue range—let alone the ~$2.5 billion in peak-year revenue forecasted by Jefferies equity analyst Maury Raycroft, PhD—INZ-701 will need its approval expanded beyond children, toward infants and especially adolescents and adults. Inozyme is enrolling infants in a second Phase III trial of INZ-701 in infants, ENERGY 2, set to conclude in February 2028, and plans a supportive study of the drug for adolescents and adults. The label expansion to adults poses a risk, since Inozyme does not expect to read out pivotal data in adults until about 2030.
“While technically, INZY could contribute to [BioMarin’s] revenues starting in 2027, the more meaningful contribution sounds like it will come in 2030+, more supplementing BMRN’s long-term revenue growth,” Fye observed.
As a result, she added, “We see BMRN looking for assets that may contribute more revenue a bit sooner than that.”
Another risk, according to Corwin of William Blair, is the size of potential patient population for INZ-701. Inozyme has identified 670 patients to date, but BioMarin has estimated a total addressable ENPP1 deficiency population that is likely between 2,000–2,500 patients, most of them expected to be outside the United States.
“We believe additional work on patient-finding activities is necessary to give us confidence that the company can build significantly on the 670 patients already identified by Inozyme,” Corwin wrote.
In addition to its pipeline, BioMarin also markets eight approved therapies. They range in net product revenue from Voxzogo® (vosoritide), a treatment for children with achondroplasia, which generated $214 million in the first quarter, to Roctavian® (valoctocogene roxaparvovec-rvox), a gene therapy for adults with severe hemophilia A that racked up $11 million during Q1.
At $4 a share, BioMarin’s purchase of Inozyme marks a 182% premium from its last pre-announcement closing price. BioMarin said it will “promptly” launch a cash tender offer to acquire all outstanding shares of Inozyme. The offer is subject to the tender of at least a majority of the outstanding Inozyme shares, expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, and other customary conditions.
Inozyme’s board has unanimously recommended that its stockholders tender their shares. Upon successful completion of the tender offer, a wholly owned subsidiary of BioMarin will merge with Inozyme, and any outstanding Inozyme shares not tendered in the offer will be converted into the right to receive the same $4 per share in cash paid in the tender offer.
The post StockWatch: With Inozyme Buyout, BioMarin Pursues Growth via M&A appeared first on GEN - Genetic Engineering and Biotechnology News.
This past week, however, BioMarin took the position of buyer, by agreeing to shell out approximately $270 million for Inozyme Pharma (NASDAQ: INZY), a developer whose treatments target the PPi-adenosine pathway, a key regulator of bone health and blood vessel function.
Inozyme investors roared their approval of the deal with a buying surge that sent the company’s shares nearly tripling, rocketing 178% on Friday from $1.42 to $3.95. BioMarin investors, however, were far more blasé about the deal, with their stock rising nearly 2%, from $58.27 to y$59.27—even after BioMarin ballyhooed the deal as a “strong strategic fit.”
BioMarin reasons that Inozyme’s Phase III lead candidate, INZ-701, is a near-perfect complement to the BioMarin pipeline of 10 programs across six rare disease treatment candidates.
“Like five of our six enzyme therapies, it would be a first-in-disease treatment,” BioMarin president and CEO Alexander Hardy told analysts Friday on a conference call about the deal. “Adding this investigational therapy to our enzyme therapies portfolio is a natural addition to our late-stage pipeline, and, if data supported, we will leverage our regulatory and commercialization know-how in countries across the world to provide access to INZ-701 to patients across our global footprint.”
Combining the active part of the ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme with a fragment of a human antibody (Fc), INZ-701 is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) designed to treat ENPP1 deficiency in both children and adults.
INZ-701 converts adenosine triphosphate (ATP) into inorganic pyrophosphate (PPi) and adenosine monophosphate (AMP). AMP is further broken down into adenosine and phosphate. By targeting ENPP1, INZ-701 can treat multiple rare diseases driven by disruptions in the PPi-Adenosine Pathway and thus restore balance across the pathway.
“Clear strategic fit”
“Bigger picture, we see this deal offering a clear strategic fit within BMRN’s rare disease portfolio with a purchase price that leaves firepower leftover for additional deals,” Jessica Fye, a managing director and senior equity research analyst for J.P. Morgan, wrote Friday in a research note.
Fye, who specializes in small- and mid-cap biotech stocks, noted astutely that BioMarin characterized as its first deal of the year the planned purchase of Inozyme, “suggesting they don’t see it as the only one.”
On the call with analysts to discuss the Inozyme acquisition, BioMarin executives discussed the potential for additional mergers and acquisitions (M&A).
“Management continues to believe the current environment remains favorable for further pipeline expansion through business development and anticipates more deal flow in the future,” William Blair analyst Sami Corwin, PhD, reported Friday in a research note.
Hardy communicated BioMarin’s interest in growth through acquisitions two weeks earlier on the company’s most recent earnings call.
“What we’re looking at is things which are completely lined up with what BioMarin is really good at. So, genetically defined conditions, we’re looking at clinical stage assets in particular as well as obviously earlier assets, but we’ve always done that,” Hardy told analysts on May 1. “We continue to focus on doing at least one business development deal this year.”
BioMarin finished the first quarter with net income of $185.686 million, up more than double (109%) from $88.662 million in Q1 2024, on revenue that jumped about 15% year-over-year to $745.145 million from $648.833 million. Inozyme announced first quarter results on Wednesday, reporting a $28.039 million net loss, up 20% from its $23.347 million net loss of a year earlier, with no reported revenue.
On Wednesday, Inozyme stock climbed 24% from $1.09 to $1.35 after combining its first quarter results with a positive update on INZ-701. The company released new interim data from its pivotal Phase III ENERGY 3 trial (NCT06046820) that it said highlighted INZ-701’s potential to modify disease course in ENPP1 deficiency by addressing its underlying biology.
Effectiveness was seen consistently across safety, immunogenicity, and phosphate level increases, Inozyme said. At week 13, for example, mean serum phosphate levels increased from baseline by +8.2% in the 17-patient INZ-701 arm vs. an 0.04% dip in the conventional treatment arm, with 35% (6 of the 17 patients) achieving normal phosphate levels at least once (vs. 0% with standard care)—an outcome suggesting potential improvement of rickets.
ENERGY 3 is set to release topline data in the first quarter of 2026. If positive, that data is expected to support an FDA filing seeking approval for INZ-701 in pediatric ENPP1 deficiency, with Inozyme anticipating launching the drug in that indication in 2027.
Peak sales forecasts for 2027
H.C. Wainwright analyst Edward White has projected first-year sales for INZ-701 of $46.6 million, with Inozyme forecasting peak-year revenue of between $400 and $600 million by the mid-2030s.
To reach that revenue range—let alone the ~$2.5 billion in peak-year revenue forecasted by Jefferies equity analyst Maury Raycroft, PhD—INZ-701 will need its approval expanded beyond children, toward infants and especially adolescents and adults. Inozyme is enrolling infants in a second Phase III trial of INZ-701 in infants, ENERGY 2, set to conclude in February 2028, and plans a supportive study of the drug for adolescents and adults. The label expansion to adults poses a risk, since Inozyme does not expect to read out pivotal data in adults until about 2030.
“While technically, INZY could contribute to [BioMarin’s] revenues starting in 2027, the more meaningful contribution sounds like it will come in 2030+, more supplementing BMRN’s long-term revenue growth,” Fye observed.
As a result, she added, “We see BMRN looking for assets that may contribute more revenue a bit sooner than that.”
Another risk, according to Corwin of William Blair, is the size of potential patient population for INZ-701. Inozyme has identified 670 patients to date, but BioMarin has estimated a total addressable ENPP1 deficiency population that is likely between 2,000–2,500 patients, most of them expected to be outside the United States.
“We believe additional work on patient-finding activities is necessary to give us confidence that the company can build significantly on the 670 patients already identified by Inozyme,” Corwin wrote.
In addition to its pipeline, BioMarin also markets eight approved therapies. They range in net product revenue from Voxzogo® (vosoritide), a treatment for children with achondroplasia, which generated $214 million in the first quarter, to Roctavian® (valoctocogene roxaparvovec-rvox), a gene therapy for adults with severe hemophilia A that racked up $11 million during Q1.
At $4 a share, BioMarin’s purchase of Inozyme marks a 182% premium from its last pre-announcement closing price. BioMarin said it will “promptly” launch a cash tender offer to acquire all outstanding shares of Inozyme. The offer is subject to the tender of at least a majority of the outstanding Inozyme shares, expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, and other customary conditions.
Inozyme’s board has unanimously recommended that its stockholders tender their shares. Upon successful completion of the tender offer, a wholly owned subsidiary of BioMarin will merge with Inozyme, and any outstanding Inozyme shares not tendered in the offer will be converted into the right to receive the same $4 per share in cash paid in the tender offer.
Leaders and laggards
- Bluebird Bio (BLUE) shares jumped 50% from $3.31 to $4.98 Wednesday after the gene therapy developer announced that its two would-be buyers, privately-held Carlyle Group and SK Capital Partners, are offering shareholders the option of receiving either $3 per share plus a contingent value right (CVR) of $6.84 per share cash payable upon achievement of a net sales milestone; or $5 per share with no CVR. Bluebird’s board has unanimously approved the amended agreement and recommended that all stockholders immediately tender their shares in support of the transaction. Should a majority not do so, Bluebird is at significant risk of defaulting on its loan agreements with Hercules Capital, the company said. Bluebird has extended the expiration date of the tender offer to one minute after 11:59 p.m. ET on May 29.
- Septerna (SEPN) shares leaped 51% from $6.73 to $10.16 Wednesday after the company joined Novo Nordisk (Nasdaq Copenhagen: NOVOB) to announce an exclusive, up-to-$2.2 billion-plus global collaboration and license agreement to discover, develop, and commercialize oral small molecule therapies for obesity, type 2 diabetes and other cardiometabolic diseases. The companies said they will initially begin four development programs for potential small molecule therapies directed to one or more select G protein-coupled receptor (GPCR) targets, including the glucagon-like peptide 1 (GLP-1), glucose-dependent insulinotropic polypeptide (GIP), and glucagon receptors. Potential payments to Septerna include more than $200 million in upfront and near-term milestone payments. Septerna’s pipeline is initially focusing on oral small molecules for endocrinology, immunology, and inflammation, and metabolic diseases. Novo Nordisk shares dipped 1.3% from DKK 438.60 ($65.63) to DKK 432.85 ($64.77).
- UroGen Pharma (NASDAQ: URGN) shares tumbled 26% from $9.85 to $7.31 Friday after an FDA staff briefing document issued in advance of the May 20–21 meeting of the Oncologic Drugs Advisory Committee took issue with the lack of a control arm in the Phase III ENVISION trial (NCT05243550), which supports the company’s New Drug Application (NDA) for UGN-102, a sustained-release hydrogel formulation of mitomycin C being developed to treat adults with recurrent low-grade, intermediate-risk non-muscle invasive bladder cancer. “Given that ENVISION lacked a concurrent control arm, the primary endpoints of complete response (CR) and duration of response (DOR) are difficult to interpret,” according to the document. “It is unclear whether the observed DOR can be attributed to the investigational product or instead reflects the natural history of the disease,” FDA staff added.
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