Amylyx Pharmaceuticals joins the public markets with a $190 million IPO that will support the company as it steers its amyotrophic lateral sclerosis drug through regulatory scrutiny and, if all goes well, for a commercial launch as a new treatment for the neuromuscular disease.
Cambridge, Massachusetts-based Amylyx expanded the offering by 1.25 million shares.Originally planned to issue 8.75 million shares of Amylyx offerings 10 million shares at $19 a share, the midpoint of a target range of $18 to $20. The shares began trading on the Nasdaq on Friday under the ticker “AMLX.” Shares of Amylyx did not rise, closing the first day at $18.07.
Amylyx aims to treat ALS by addressing neuronal deathThe company’s drug, AMX0035, consists of two small molecules, each of which takes a different approach to pathways involved in neuronal survival. The company believes that the combination of these two mechanisms offers the potential to help neurons live longer. In a placebo-controlled Phase 2 study, patients treated with the drug Amylyx showed improvements in measures of physical function in patients with ALS. Based on these results, Amylyx submitted a New Drug Application to the FDA. Last week, the agency accepted the application for priority review and set a target date of June 29 for a regulatory decision.
Pending the FDA’s decision, it will convene an independent panel of experts to discuss the safety and efficacy of AMX0035 and any scientific concerns about the drug. A date for the advisory committee meeting has not been set. Meanwhile, a larger Phase 3 study is underway.This FDA backtracks on need for larger study Support for drug applications.Although the agency ultimately concluded that Amylyx may seek approval based on Phase 2 data, the company is conducting late-stage studies to generate more data on the drug’s safety and efficacy as it seeks regulatory approval in other markets around the world.
according to IPO application, Amylyx’s origins can be traced back to a Brown University dorm room, and in 2013, co-founders Josh Cohen and Justin Klee set out to find answers to the question of why neurons die. The duo’s research led to the development of AMX0035, the company’s only drug candidate to date. Although ALS is the drug’s first disease target, Amylyx noted that improving neuronal survival has potential applications in many neurodegenerative diseases. A clinical trial is already underway to test AMX0035 in Alzheimer’s disease, and testing in Wolfram’s disease is also planned.
Amylyx has raised approximately $234 million since its inception, most recently $135 million in Series C financing in July last year. The prospectus shows that Morningside Ventures is Amylyx’s largest shareholder, with an 18.9% post-IPO stake. ALS Invest 1 owns 10.8%, while Viking Global Investors owns 8.8%.
At the end of the third quarter of 2021, Amylyx reported a cash position of $125.7 million. Combined with the IPO proceeds, the company plans to use about $100 million in capital for the regulatory review process of AMX0035 in ALS, as well as in preparation for the drug’s potential launch if it is approved. Another $15 million is being used to fund ongoing Phase 3 clinical trials until completion; $10 million is earmarked for expanding the company’s drug pipeline into other neurodegenerative diseases.
In the first week of the new year, two more biotech companies joined the public markets. Below are the IPOs of CinCor and Vigil Neuroscience.
CinCor’s mega IPO brings $194 million to hypertension drug trials
Patients with high blood pressure can choose from a variety of high blood pressure medications, many of which are old and some new. In many cases, these drugs are not enough to help patients manage their condition. CinCor is developing a drug that provides a new treatment for hypertension and other cardiovascular and kidney diseases, and has raised $193.6 million for clinical development of its most advanced program.
Late Thursday, Boston-based CinCor priced its IPO at $16 a share, the midpoint of the company’s projected price range. CinCor is able to scale up transactions, providing 12.1 million shares, higher than the company’s planned 11 million shares. CinCor began trading on the Nasdaq on Friday under the ticker symbol “CINC.”
The standard of care for hypertension medications includes angiotensin-converting enzyme (ACE) inhibitors and angiotensin receptor blockers (ARBs), both of which work by lowering levels of the blood pressure-regulating hormone aldosterone. CinCor in its IPO application Long-term treatment with these drugs can lead to an “aldosterone breakthrough,” in which the hormone levels in the blood return or exceed baseline levels. Mineralocorticoid receptor antagonists (MRAs) can lead to increased aldosterone synthesis, which then requires higher doses of the drug to compensate. But MRAs introduce multiple risks of side effects.
CinCor’s lead drug candidate, CIN-107, is a small molecule designed to block aldosterone production by targeting an enzyme responsible for hormone synthesis in the adrenal gland. Despite prior treatment with three antihypertensive drugs, the drug has been tested in phase 2 in patients with uncontrolled blood pressure. A separate phase 2 study was recently initiated in patients with uncontrolled hypertension despite receiving an antihypertensive drug. CinCor said in the filing that it is also developing drugs to treat primary aldosteronism, a hormonal disorder that occurs when too much hormone is produced. Another potential application of the drug is in the relief of complications associated with chronic kidney disease.
Founded in 2018, CinCor is a subsidiary of CinRx Pharma, a Cincinnati-based biotechnology company with a business model of acquiring drug assets and advancing them to exit points, such as licensing deals, joint ventures or IPOs. In 2019, CinCor spun off as an independent company and licensed its lead drug from Roche. According to the prospectus, CinCor raised $185 million ahead of its IPO.Most of the cash is in a company closing in October $143 million Series B financing. The company’s largest shareholder is Sofinnova Investments, with a 14.8% stake after the IPO. Sofinnova Partners and 5AM Ventures each own 10.9%, while CinRx owns 8.1%.
At the end of the third quarter of 2021, the company reported a cash position of $141.7 million. CinCor said in its prospectus that it plans to spend about $90 million to advance CIN-107 by completing Phase 2 testing in patients with uncontrolled hypertension. Another $37 million was budgeted to advance the drug through a separate interim test in primary aldosteronism. CinCor also plans to begin a Phase 2 trial in chronic kidney disease patients with uncontrolled blood pressure. The study is expected to begin in the first half of this year.
Vigil gets $98M to test new treatment for rare neurological disease
Vigil Neuroscience, a company developing treatments for rare inherited neurological diseases with no FDA-approved therapies, now has $98 million in the first tests of its drugs in humans.Biotechnology supply 7 million shares at $14 a share, below the company’s planned price range of $15 to $17. The shares began trading on the Nasdaq on Friday under the ticker “VIGL.”
The disease target in Cambridge, Massachusetts is Adult-onset leukoencephalopathy with axonal spheroids and pigmented glial cells (Alsp). The rare disorder is a leukodystrophy, a condition that causes the brain’s white matter to be depleted. Neurodegeneration of ALSP leads to changes in personality, cognition, and muscle function. This decline can progress to dementia and eventually to a vegetative state. According to Vigil, the estimated US population affected is 10,000.
ALSP arises from mutations in the gene that produces the colony-stimulating factor 1 receptor, a protein expressed on the surface of microglia and macrophages, both types of immune cells found in the central nervous system.inside IPO application, Vigil noted that the colony-stimulating factor 1 receptor shares a downstream signaling pathway with another protein, TREM2. Vigil aims to compensate for the loss of function in microglia by targeting TREM2.
Vigil’s drug VGL101 is an antibody designed to activate TREM2. The first phase of testing on healthy volunteers began last month. In its IPO filing, Vigil said it expects preliminary data to be released in the second half of the year.
Founded in 2020, Vigil was co-founded by venture capital firm Atlas Venture. The company acquired the intellectual property rights to the TREM2-targeting molecule from Amgen. In addition to the antibody approach taken by VGL101, the biotech is developing a small-molecule drug targeting TREM2.According to filings, Vigil raised $120 million ahead of its IPO, most recently August $90 million Series B financing. According to the prospectus, Atlas is Vigil’s largest shareholder with an 18.7% stake. Northpond Ventures and Vida Ventures own 13.6% and 11.8%, respectively. Amgen’s stake is about 11.3%.
At the end of the third quarter of 2021, Vigil reported having $110.6 million in cash. Combined with the IPO proceeds, Vigil plans to spend approximately $105 million to advance the development of VGL101 in ALSP and other rare leukoencephalopathy and leukodystrophies. Another $30 million is planned for research into small molecules that could treat neurodegenerative diseases associated with microglial dysfunction, while $20 million is earmarked for additional research and development.
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