Justin Klee (L) and Joshua Cohen (Amylyx)
The biotech that has gone all-in on ALS on its way to earning one of the highest neuroscience financing rounds in the last five years is now ready to take the Nasdaq leap.
Amylyx Pharmaceuticals filed its S-1 with the SEC late Thursday, penciling in a $100 million raise estimate to help bring its only product candidate to market. The move comes about five months after Amylyx pulled in $135 million in a Series C.
Founded by two Brown University roommates with little drug development experience, Amylyx has seen a sharp rise since September 2020 when a Phase II study for its ALS program AMX0035 came back positive on function, a rare feat in the disease. The candidate beat placebo on a functional rating scale — a scale some experts argue has serious issues — with the biotech focusing all efforts on getting it approved.
As laid out in the SEC paperwork, that strategy hasn’t changed. The company plans to funnel much of its IPO cash toward the regulatory process and pre-commercial launch of AMX0035, as well as toward the drug’s Phase III study currently being conducted in ALS.
And the S-1 appears to further raise the stakes on just how much Amylyx is relying on AMX0035 to be successful. The filing’s ‘Risk Factors’ section is littered with warnings over the singular focus on the ALS program, at one point noting ‘the denial of regulatory approval for AMX0035 in any jurisdiction could mean that we need to delay or even cease operations.’
Since that Phase II readout, Amylyx has hustled to ensure that doesn’t happen, as the biotech is currently seeking approval in Canada, the US and the EU. At first, it appeared the FDA wasn’t receptive to that dataset, initially requesting Amylyx run an additional study before submitting its application.
But the neuro landscape changed in June after the FDA controversially approved Biogen’s aducanumab for Alzheimer’s disease despite concerns over its efficacy and safety, as well as a unanimous adcomm vote recommending against approval. Regulators reversed course on AMX0035 in September, allowing Amylyx to submit its NDA after viewing the company’s Phase III protocols, co-CEO Joshua Cohen said at the time.
Amylyx ultimately approached the agency last month with its application. The Phase III study also launched this quarter, with Amylyx planning to enroll 600 patients — more than four times its Phase II trial population. Though the biotech says these data won’t be required for an FDA approval, it notes in the S-1 the FDA can always change its mind.
That reversal comes as ALS drug development also remains top of mind on Capital Hill. About a week after the House passed a bill to pump $500 million into ALS drug research, the Senate voted to pass it late Thursday evening, and President Biden is expected to sign it into law.
The S-1 also gave a peek behind the curtain at some of Amylyx’s shareholders. Both Cohen and his co-CEO, Justin Klee, each own a 6.1% stake in their biotech. Morningside Ventures would be the biggest winner here with about 22%, while ALS Invest gets 12.8% and Viking Global owns 10.5%.
Some IPO money will also be set aside for pursuing other indications, such as Alzheimer’s and Wolfram syndrome. When Amylyx begins trading, it will do so under the ticker $AMLX.
Biotech IPOs have substantially slowed down since the pandemic boom of 2020 and early 2021, with analysts noting investors have shifted toward early-stage private financings as their preferred choice. Though the industry has raised more than $31 billion over the last two years, it appears increasingly likely 2022 won’t keep up that pace.
Sensor-based technology for clinical trial data collection represents the latest medical paradigm shift. There are more than 700 clinical studies involving wearable devices currently underway in the United States. A study from Intel IT projects their inclusion in clinical trials will surge to 70% by 2025.
Apps, biosensors and patient-centered technologies increase visibility of comprehensive patient data. Pharma leaders anticipate the benefits of wearables to include better data (58%), faster results (33%) and lower trial costs (10%).
Richard Pazdur (via AACR)
There’s no denying that Merck’s Keytruda set a high bar for checkpoint inhibitors in development everywhere. But when it comes to the often redundant development of PD(L)-1 antibodies worldwide, FDA’s top cancer doctors Rick Pazdur and Julia Beaver are calling for more industry coordination.
‘Efforts to corral this enthusiasm should focus on increased international partnerships between sponsors of approved checkpoint inhibitors and those developing novel agents to be used with anti–PD-1 and anti–PD-L1 antibodies rather than developing ‘me too’ drugs,’ Beaver and Pazdur wrote Wednesday in the New England Journal of Medicine.
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Otello Stampacchia, Omega
Amid a very difficult back half of the year for biotech investing, some bullish investors are making the case for a major rally in the coming year as pharma looks to utilize its growing mountain of cash. Now, biotech blue-chipper Omega Funds is getting itself set at the starting line.
Omega has closed a $650 million investment fund it’s calling Omega Fund VII, the VC firm’s latest and largest round in its 17 years in existence, it said Friday.
The European Medicines Agency announced Friday it recommended the European Commission reject Aduhelm, dealing a new — albeit expected — blow to Biogen’s hopes of finding a widespread market for its struggling Alzheimer’s drug.
The EMA recommendation had been expected for a month, since the EMA’s human medicines committee gave Biogen ‘a negative trend vote’ after an oral presentation from the company. As such Biogen’s stock, which has lost all the stratospheric value it gained after Aduhelm’s approval in June, only ticked down 3% pre-market Friday, from $235.52 to $235.
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Janet Woodcock, acting FDA commissioner (Al Drago/Bloomberg via Getty Images)
Since the pandemic began, the FDA has allowed women to access the abortion drug mifepristone via the mail, making it easier for them and ensuring that they don’t have to go to a clinic to receive the pills.
The FDA’s temporary change became permanent on Thursday afternoon, with the FDA announcing that the REMS on mifepristone (brand name Mifeprex) and its generic versions must be modified by removing this in-person dispensing requirement.
Pascal Prigent, Genfit CEO
Somebody believes in elafibranor after all.
The drug, which had been considered a bright prospect in the failure-prone but highly lucrative race to treat non-alcoholic steatohepatitis, imploded after it failed a closely watched trial, forcing its developer, Genfit, to throw in the towel. But after a lonely year steering past the wreckage — chopping nearly half of its workforce in a painful restructuring — Genfit has found a trusting partner in fellow French drugmaker Ipsen.
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The FDA on Wednesday not only approved the first generic versions of the decades-old diabetes insipidus treatment vasopressin, but also simultaneously offered a particularly damning rebuke of a citizen petition attempting to block the generic, while promising to pass along the matter to the Federal Trade Commission.
The response could prove troublesome for the sponsor of the brand name version of the drug, Endo’s Par Sterile Products, which brought in more than $780 million in 2020 for its brand name version of the drug Vasostrict.
Robert Califf (Graeme Sloan/Sipa via AP Images)
As Rob Califf likely makes his return as FDA commissioner next month, his confirmation hearing yesterday offered a peek into some of the larger obstacles he’s going to face in the coming months and years.
The pandemic isn’t going away anytime soon with Omicron, and some vaccines and therapeutics may need to be tweaked or pulled from the market entirely as they prove to be ineffective against the new variant. The FDA, meanwhile, needs to get back on even footing with some longer-term direction.
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Crowd gathering at the Westin St. Francis for JPM in 2019 (Endpoints News)
Well, see you in January 2023.
In a surprise about-face, #JPM22 will now be fully virtual after organizers of the popular biotech conference decided to pull the plug on a live event in San Francisco given fears over the Omicron variant and a growing chorus of drugmakers opting out.
The move is no big surprise after reports swirled about some of the industry’s biggest players nixing plans to attend live and pressuring the bank to reconsider the annual meet at the Westin St. Francis. STAT reported Tuesday that Moderna and Amgen, among other large drugmakers, had already pulled out.
https://endpts.com/amylyx-takes-its-als-program-to-nasdaq-raising-the-stakes-for-the-biotechs-only-drug/
