Amylyx Pharmaceuticals, Inc.

Plus, news about CAMP4 Therapeutics, Serina Therapeutics, Kriya Therapeutics, Cutiss, Dianthus Therapeutics, Alnylam and Eyexora: 🔬 PMV touts Phase 2 data in p53 mutation cancers: In a 97-patient trial for rezatapopt, PMV observed 32 responses across a variety of tumor types. That’s good for a 33% overall response rate. The highest response rates came in ovarian cancer, in which 43% of patients responded (19 of 44), and endometrial cancer, in which 60% responded (3 of 5). The company plans to enroll another 20 to 25 patients with ovarian cancer after meeting with the FDA. If everything goes well, PMV plans to submit for approval in the first quarter of 2027. PMV’s stock $PMVP rose 24% in premarket trading on Wednesday. — Max Gelman 💰 Amylyx’s $175M offering: The company is selling 17.5 million shares at $10.00 a piece, in part to support avexitide , a GLP-1 antagonist that it licensed from Eiger BioPharmaceuticals last year. Amylyx is currently running a Phase 3 trial assessing avexitide in post-bariatric hypoglycemia. — Jaimy Lee 🧠 CAMP4 Therapeutics gets up to $100M: It plans to use the money from the private placement to fund its SYNGAP1 program, among other uses. CAMP4 will get $50 million upfront; the rest of the financing is tied to milestones for CMP-SYNGAP-01. Coastlands Capital led the financing. — Jaimy Lee 💸 Serina Therapeutics gets up to $20M: The biotech wants to use the funding to support a Phase 1b study for SER-252, its experimental Parkinson’s disease therapy. Greg Bailey, a member of Serina’s board, led the financing. — Jaimy Lee 🧾 Kriya Therapeutics closes $320M Series D: The North Carolina-based gene therapy startup ended up raising $7 million more than what was disclosed in an SEC filing last month . Patient Square Capital and Premji Invest, which steered Enveda’s recent $150 million Series D, co-led Kriya’s late-stage financing. Kriya said the money will bankroll clinical trials for five programs and their manufacturing activities. — Kyle LaHucik 🩺 Cutiss closes $70M Series C: The company plans to use the money to fund a Phase 3 trial for its skin graft product, denovoSkin, and prepare for a commercial launch. — Max Gelman 💵 Dianthus Therapeutics ended up raising $251 million in its public offering. That’s much more than the $172.5 million it intended to raise. — Jaimy Lee 💲 Alnylam upsized its offering of convertible senior notes to $575 million from $500 million . — Jaimy Lee 👁 New startup to focus on ophthalmology: Eyexora will use a “hub-and-spoke model” to develop drugs and devices for eye diseases. The company launched with an undisclosed seed round and in-licensed its initial programs from the Singapore Eye Research Institute. — Max Gelman

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the pricing of an underwritten public offering of 17,500,000 shares of its common stock at a public offering price of $10.00 per share. All of the shares of common stock are being offered by Amylyx. In addition, Amylyx has granted the underwriters a 30-day option to purchase up to an additional 2,625,000 shares of its common stock at the public offering price per share, less underwriting discounts and commissions. The gross proceeds to Amylyx from this offering are expected to be $175.0 million, before deducting underwriting discounts and commissions and offering expenses and excluding any exercise of the underwriters’ option to purchase additional shares. Amylyx intends to use the net proceeds from this offering, together with existing cash, cash equivalents and marketable securities, for: Avexitide commercial readiness; Research and development; and Working capital and other general corporate purposes. The offering is expected to close on or about September 11, 2025, subject to the satisfaction of customary closing conditions. Leerink Partners and Guggenheim Securities are acting as lead bookrunning managers for the offering. H.C. Wainwright & Co. is acting as lead manager for the offering. The shares described above are being offered pursuant to a shelf registration statement on Form S-3 (File No. 333-285525) that was filed with the Securities and Exchange Commission (the “SEC”) on March 4, 2025 and declared effective by the SEC on March 26, 2025. This offering is being made only by means of a prospectus supplement and an accompanying prospectus that form a part of the registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the offering were filed with the SEC on September 9, 2025, and are available on the SEC’s website located at www.sec.gov. A final prospectus supplement containing additional information relating to the offering and accompanying prospectus will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. When available, copies of the final prospectus supplement and the accompanying prospectus relating to this offering may also be obtained by contacting: Leerink Partners LLC, Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, or by telephone at (800) 808-7525 ext. 6105, or by email at syndicate@leerink.com; and Guggenheim Securities, LLC, Attention: Equity Syndicate Department, 330 Madison Avenue, 8th Floor, New York, NY 10017, telephone: (212) 518-9544, email: GSEquityProspectusDelivery@guggenheimpartners.com. This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of that state or jurisdiction. About Amylyx Pharmaceuticals Amylyx’ mission is to usher in a new era of treating diseases with high unmet needs. Where others see challenges, Amylyx sees opportunities and pursues them with urgency, rigorous science, and unwavering commitment to the communities it serves. Amylyx is currently focused on three investigational therapies across several endocrine and neurodegenerative diseases in which it believes it can make the greatest impact. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including with respect to the completion of the offering, the potential exercise by the underwriters of the option to purchase additional shares, the anticipated closing date of the offering, the expected gross proceeds from the offering and the expected use of proceeds from the offering. No assurance can be given that the offering will be completed. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Any forward-looking statements in this press release 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. Risks that contribute to the uncertain nature of the forward-looking statements include those risks and uncertainties set forth in Amylyx’ SEC filings, including Amylyx’ Annual Report on Form 10-K for the year ended December 31, 2024, Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2025 and its subsequent filings with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. In light of the foregoing, investors are urged not to rely on any forward-looking statement in reaching any conclusion or making any investment decision about any securities of Amylyx. Media Amylyx Media Team (857) 320-6191 amylyxmediateam@amylyx.com Investors Lindsey Allen (857) 320-6244 Investors@amylyx.com

iStock/ Ekaterina Chizhevskaya After a demoralizing period punctuated by the withdrawal of one of the few marketed therapies for ALS, investment in new biotechs, state-backed collaborative initiatives and buzz at BIO2025 suggest a new day in drug development for one of medicine’s most intractable diseases. The past couple of years have been disheartening for the amyotrophic lateral sclerosis community—to say the least. With the approval of Biogen’s Qalsody shining as the one bright spot, ALS patients have otherwise been dealt one blow after another, from the market withdrawal of Amylyx’s Relyvrio to the failure of candidates from Sanofi and Denali Therapeutics . So it was especially encouraging at BIO2025 earlier this summer to learn of a budding resurgence in ALS drug development. Approximately one-third of the companies that BioSpace sat down with at the convention highlighted the intractable neurodegenerative disease as a key focus. Boehringer Ingelheim’s BI Venture Fund is shepherding two young biotechs, Rgenta Therapeutics and Libra Therapeutics, focused on novel targets for ALS; Korro Bio has an RNA-editing candidate in the discovery stages; and GATC Health is the AI engine behind a Louisiana-backed ALS initiative aiming to mine the world’s largest ALS multiomics dataset to accelerate the discovery of new drug targets. That the failures of the past two years have not dampened the appetite of investors or biopharma companies for ALS was not a surprise to the experts BioSpace spoke with. “[Relyvrio is] a medicine that was approved on the basis of a single Phase II trial, and I think there was a lot of still residual uncertainty about how effective it was,” said Eric Green, CEO of Trace Neuroscience, which launched last November with $101 million in series A funds and is initially targeting ALS. “But even so, you saw a really rapid uptake, and I think it really illustrates just how strong the desire is for anything new in these patients who really have very minimal options.” The Next Wave Unlike Relyvrio, Biogen’s Qalsody, an antisense oligonucleotide (ASO), has been on the market for over two years—and it’s helping patients. “We’re seeing not only slowing of disease, but in a number of patients we’re actually seeing frank reversal of disease,” said Green, whose company is also developing an ASO. “I think this has been a huge bright spot in ALS, one that I think offers a lot of lessons for how we think about discovering and developing medicines for the broader population.” Greenlit by the FDA in April 2023, Qalsody became just the fourth treatment for ALS. While a significant accomplishment, Qalsody is approved to treat just 2% of ALS patients—those with a mutation in the superoxide dismutase 1 ( SOD1 ) gene. ALS, like cancer or Alzheimer’s disease, is a heterogeneous malady—one comprising myriad patient subtypes. Green believes that Trace’s eventual therapy should be broadly applicable. “I think that’s kind of our call to action here. . . . We think that this is an opportunity to bring that genomic medicine to the other 98% of people with ALS who don’t have that option today,” he said. Trace is seeking to restore the function of UNC13A, a protein that plays an essential role in coordinating neuronal communication that studies have found to be insufficient in patients with ALS, according to the biotech’s website . “UNC” refers to uncoordinated, Green explained, because it was discovered that when it is missing in worms, mice or flies, they become paralyzed because nerves and muscle cells cannot communicate with each other. Then, nearly five years ago, Trace’s scientific founders found that “nearly all people with ALS lose the UNC13A protein” due to the improper processing of RNA, he said. As a result, RNA is degraded, and the protein doesn’t get made. Likewise, GATC president Rahul Gupta is hopeful that the company’s platform, which integrates genomics, transcriptomics and proteomics, can uncover novel treatments for a broad range of patients with ALS. Because of this multi-omic approach, “we are able to find those patterns across diverse patient subtypes, basically, then map those disease drivers and stratified mechanism types,” he told BioSpace . With the Answer ALS–backed Louisiana AI Drug Development Infrastructure for ALS (LADDIA) initiative, GATC and its partners are looking to marry the power of artificial intelligence with “one of the largest ALS databases in the world” to discover novel treatments for the disease. Gupta said the AI-powered ability to generate targets and discover treatments in a complex, combined, multiphasic way is like “going from the cart and buggy to a rocket.” “There’s a lot more that could be found without being traditional,” he added. Another common target across many neurodegenerative diseases is neuroinflammation. This is the focus of Houston-based Coya Therapeutics. “I think the big focus in ALS, and [the] realization a lot of people are coming to is neuroinflammation is the way to address ALS,” CEO Arun Swaminathan told BioSpace . “I think you’re increasingly seeing investor understanding of that, the science behind why neurodegenerative diseases are driven by neuroinflammation.” Specifically, Coya is developing therapies—including COYA 302 for ALS—to enhance the function of regulatory T cells (Tregs). “There’s a lot of publications that show that . . . the more dysfunctional [Tregs] are in ALS patients, these ALS patients tend to progress faster, meaning their disease progression is faster and also eventual death, the timelines are faster as well,” Swaminathan said. Tregs are the “internal brakes of the immune system,” he explained. They ensure that the T cells do not spin out of control and damage healthy neurons and nerve cells, “which is kind of what happens in diseases like ALS.” Ultimately, if you can make these brakes work more efficiently, and keep them working, “then in theory, you should be able to stop the progression of neuroinflammation, therefore translating to stopping the progression of the disease.” On Aug. 25, the FDA accepted Coya’s investigational new drug application for a Phase II trial of COYA 302 in patients with ALS, marking “a pivotal moment in [the company’s] journey,” Swaminathan said in a prepared statement. ALS Buzz at BIO2025 Meanwhile, back at BIO2025, Korro Bio CEO Ram Aiyar revealed that the biotech’s third development program will target the DNA/RNA-binding protein TDP-43 . The mislocalization of this protein from the nucleus to the cytoplasm leads to a cascade of cellular dysfunctions, including impaired RNA processing, defective axonal transport and mitochondrial issues. Korro’s strategy is to make a single protein change that keeps TDP-43 in the nucleus. It’s a “very elegant way of solving this problem,” Aiyar said. And ALS may well be Boehringer Ingelheim’s first foray into neurodegeneration—that is if it makes a play for two companies currently under the wing of the Boehringer Ingelheim Venture Fund . Detlev Mennerich, global head of the fund, noted that ALS is an attractive target for a small biotech because the trials are affordable. “ALS is such a progressive disease, where the motor neuron function drops so fast that you . . . can fund an affordable volume Phase II where you see a meaningful clinical endpoint with a limited amount of patients,” he told BioSpace on the sidelines of the conference. Gupta, for one, has seen his own patients die from ALS. “I’ve seen the whole cycle,” he said, adding that he believes we’re now at a turning point. “In the area of rare and ultra rare diseases, there hasn’t been enough investment focus or adaptation of science. There just hasn’t been. And somehow, now both the administration is interested [and] there’s datasets there. There are people that are suffering that need the treatment today, not five, ten years from now. It’s a race against time.”