Biohaven Ltd.

Scholar Rock contends its experimental drug for spinal muscular atrophy can achieve what none of the currently available therapies can do to treat this rare disease. After positive data from a pivotal study sent the biotech’s shares skyward, Scholar Rock has raised $300 million from a stock offering that will be applied toward regulatory submissions and launch preparation of what could become its first commercialized product. The stock sale follows the company’s Phase 3 data readout earlier this week showing its drug, apitegromab, met the goal of showing muscle function improvement in spinal muscular atrophy (SMA). The Cambridge, Massachusetts-based biotech is planning regulatory submissions to the FDA and the European Medicines Agency early next year. SMA stems from mutations to the gene that codes for survival motor neuron (SMN), a protein key to keeping motor neurons healthy. Patients who have the rare inherited disease develop progressively worsening muscle weakness. Few therapies are available for SMA. Biogen was first with Spinraza, an antisense oligonucleotide intended to increase production of functional SMN protein. Novartis offers a one-time treatment with Zolgensma, a gene therapy that replaces the defective gene with a functioning one. Evrysdi, a drug developed by PTC Therapeutics and commercialized by Roche, offers another approach. This oral small molecule is designed to get the SMN2 gene to produce more SMN protein. sponsored content Upcoming Webinar: Battle of the Budget: Employers vs. Rising Healthcare Costs Gain valuable knowledge and practical insights on how employers are addressing an increasingly complex healthcare budget landscape. By Personify Health Scholar Rock takes a different and perhaps complementary approach to available SMA therapies. Treatments that target SMN prevent further degeneration of motor neurons, but they do not directly affect muscle atrophy, the company said in an investor presentation. Apitegromab is a monoclonal antibody designed to block activation of myostatin, a protein in skeletal muscle that inhibits muscle growth. It’s administered as an intravenous infusion every four weeks. The placebo-controlled Phase 3 test evaluated the study drug in patients who were already taking the standard chronic SMA therapies Spinraza or Evrysdi. This trial enrolled 188 SMA patients ages 2 to 21. In preliminary results reported Monday, Scholar Rock said the drug achieved statistically significant and clinically meaningful improvement at 12 months, measured according to a scale used to assess muscle function in SMA patients (a higher score indicates better muscle function). The company added that 30% of patients who received the study drug showed a 3 point or greater improvement in score compared to 12.5% of patients in the placebo group. Early motor function improvement was observed at eight weeks and that benefit expanded at week 52, as measured according to the rating scale. The drug was well tolerated and no serious adverse events were reported. “The results clearly demonstrate robust and clinically meaningful improvement in motor function in patients with SMA,” Scholar Rock CEO Jay Backstrom said in a prepared statement. “At Scholar Rock, we are working with urgency to deliver the potentially transformative benefits of apitegromab to children and adults with SMA in the U.S., Europe, and around the world.” The apitegromab results will be presented on Friday during the Annual Congress of the World Muscle Society meeting in Prague. In the investor presentation, Scholar Rock claimed its drug is the first and only muscle-targeted therapy to show both clinically meaningful and statistically significant functional improvement in SMA. The company added that apitegromab is the first and only anti-myostatin therapy to show muscle improvement in a Phase 3 study. Biohaven might have something to say about that soon. That company’s experimental SMA therapy taldefgrobep alfa, an inhibitor of both myostatin and the activin A pathway, is expected to post Phase 3 data by the end of this year. Sponsored Post Get Ready for HLTH 2024: A Preview Guide HLTH 2024, scheduled for October 20-23 at the Venetian Expo Center in Las Vegas, will highlight new programming, a physicians roundtable, generative AI, the shifting landscapes of telemedicine, retail health, women's health, and lots more. By Stephanie Baum Shares of Scholar Rock soared more than 300% after the biotech’s data release, and the company seized on the opportunity to raise money. Late Tuesday, Scholar Rock priced an offering of more than 10.2 million shares at $28.25 each. The offering includes pre-funded warrants to purchase 353,983 shares at the public offering price. Scholar Rock has also granted underwriters a 30-day option to purchase up to 1.5 million shares of stock at the public offering price, which could raise $42 million more. Scholar Rock said regulatory submissions for apitegromab in the U.S. and Europe are planned for the first quarter of next year, setting the stage for a potential fourth quarter 2025 commercial launch. Scholar Rock sees additional potential applications for apitegromab. The company is evaluating the drug as a way to help preserve muscle in patients taking a GLP-1 drug for weight loss. Muscle loss is a known side effect of the GLP-1 drug class, leading a growing number of companies to try and address the complication. Late last month, BioAge Labs’ IPO raised more than $200 million to continue clinical development of an oral small molecule intended to preserve muscle mass. A Phase 2 study is underway evaluating the BioAge drug in combination with Eli Lilly obesity drug Zepbound; preliminary results are expected in the third quarter of 2025. By then, Scholar Rock should already have topline results for the obesity study, which on track to post data in the second quarter of 2025. In the prospectus, Scholar Rock said proceeds from the offering combined with its existing capital will be used to support commercialization of apitegromab and to advance other programs. The company estimates that the stock offering provides enough capital to fund operations into the fourth quarter of 2026. Image: koto_feja, Getty Images

iStock, Anton Vierietin Big Pharma can’t seem to get enough radiopharmaceutical biotechs. With Lilly, Sanofi and BMS chasing Novartis into the complex space, all eyes are on these specialty biotechs. Big Pharmas are snapping up radiopharmaceutical biotechs left and right, inspired by the market-leading position of Novartis, which has two therapies already approved. Behind these big ticket deals are a clutch of biotechs continuing to go at it alone. The use of targeted radioisotopes to treat cancer is not for the faint of heart. The specialized nature of this class of medicines means a Big Pharma can’t just buy the drug and cast off the rest of the company, as is often done when buying out a small company. Novartis has been the clear leader, with Pluvicto approved for prostate cancer and Lutathera for neuroendocrine tumors. The company developed Lutathera in-house and acquired Pluvicto through the acquisition of Endocyte in October 2018 in a deal valued at over $2 billion. The Swiss pharma has been hungry ever since to expand its pipeline in the space, with the acquisition of Mariana Oncology for $1 billion and a licensing deal expansion with PeptiDream, both this past spring. In 2023, Novartis’ peers started to take notice. Eli Lilly kicked off a string of deals in October 2023 with the $1.4 billion acquisition of Point BioPharma and followed that up with a partnership with Aktis Oncology that could also top $1 billion. Meanwhile, Bristol Myers Squibb dropped more than $4 billion in December 2023 for radiopharma-focused RayzeBio. And most recently, Sanofi is dipping its toe into the space, with a licensing deal worth $356 million upfront with RadioMedix. With so much cash being thrown around to buy into radiopharma, which biotechs are making waves on their own and may soon be wrapped up in the next Big Pharma deal? Here are five radiopharma companies you may be hearing about soon. Aktis Oncology Radiopharma-hungry Lilly inked a partnership with Aktis Oncology in May for $60 million, gaining access to the biotech’s targeted anticancer medicines. The deal could reach a whopping $1.1 billion in potential milestones and royalties later on. Aktis is using alpha-emitting particles for precision cancer targeting, while avoiding side effects. Lead programs target urothelial and other cancers. Late last month, Aktis also raked in a sizable $175 million oversubscribed Series B , underscoring the interest in the space. The round was led by RA Capital Management and co-led by RTW Investments and Janus Henderson Investors. Other participants included Bristol Myers Squibb, Lilly and MRL Ventures Fund, Merck’s venture fund, putting an exclamation point on the radiopharma frenzy. Aktis’ lead candidate is AKY-1189, a miniprotein alpha radioconjugate targeting Nectin-4 in development for solid tumors. The biotech has plenty of cash to go it alone for now, with CEO Matthew Roden reporting $300 million on hand. But with a trio of Big Pharmas making chase, Aktis is sure to be in the news again very soon. ARTBIO Launched in June last year, ARTBIO is creating a new class of radioligand therapies using the alpha-precursor isotope (Pb212) and tumor-specific targets. ARTBIO’s pipeline is still preclinical, but the most advanced is AB001, which targets prostate cancer. A December 2023 Series A , co-led by Third Rock Ventures and an undisclosed healthcare fund, brought in $90 million. Since then, the biotech has been signing deal after deal to shore up supply and manufacturing . Another deal from May will see ARTBIO work with fellow clinical-stage biotech FogPharma to develop so-called HEARTs , or Helicon-enabled 212Pb alpha radioligand therapies—a combination of both companies’ platforms. Nucleus RadioPharma This biotech is not developing therapies but instead offering integrated manufacturing and supply chain organization for radiopharmaceuticals. Nucleus’ platform has already attracted an investment from AstraZeneca, which pitched in to a Series A extension in June. Financial details were not disclosed, but the biotech raised $56 million in the original oversubscribed Series A one year ago, led by GE Health and Eclipse. The cash was expected to go towards multiple new manufacturing facilities around the U.S., including one near the Mayo Clinic, a contributor to the Series A round. Additional funds would help build technology for the development, manufacturing and distribution of radiopharmaceuticals. PeptiDream Novartis has returned to Japanese biotech PeptiDream time and time again to refill its stock of radiopharmaceuticals. In April, the two companies re-upped a partnership to total more than $2.7 billion in milestones. But the pair have been working together since 2010. And Novartis is not PeptiDream’s only partner. The company also has deals with Roche’s Genentech , Lilly and Merck , totaling billions in potential milestones. So what is PeptiDream’s secret sauce? Its Peptide Discovery Platform System can find new drugs based on macrocyclic peptides, which can be screened against any biological target of interest very quickly, according to the company. PeptiDream boasts a 95% success rate. The technology was licensed from the University of Tokyo and optimized in-house. The company’s extensive pipeline is populated by other partnerships, including with Biohaven and RayzeBio, which was acquired by BMS for $4.1 billion in December 2023. Most but not all are in cancer, with PeptiDream’s single internal program, a myostatin inhibitor, focused on developing radioligands for obesity and other muscle disorders. RadioMedix Sanofi found the ideal partner to join the radiopharma race in RadioMedix last month, signing a deal that also included French radioligand developer Orano Med. Sanofi handed over €100 million ($110 million) upfront, with €220 million ($242.5 million) in sales milestones, plus royalties, to access a neuroendocrine tumor candidate. The deal focused on AlphaMedix, a targeted radioligand therapy that uses lead-212Pb to target cancer. RadioMedix and Orano Med are running a Phase II clinical trial for the candidate in patients with neuroendocrine tumors, with a readout expected this year. The company also has two other clinical programs and some earlier-stage assets in preclinical testing. In addition to therapeutics, RadioMedix develops radiopharmaceuticals for diagnosis and monitoring of cancer and has capabilities from development up to manufacturing.

Pfizer has terminated development of its RSV treatment candidate sisunatovir, a viral inhibitor picked up in its up to $525 million acquisition of ReViral in 2022. The New York drug company — which is facing an activist investor that’s expected to call for major changes at the company — ended a Phase 2/3 trial and a Phase 1 study of the oral inhibitor, according to updates to a federal clinical trials database. “There have been ongoing challenges in the sisunatovir clinical development program, including a drug-drug interaction with antacids,” a Pfizer spokesperson said in an emailed statement to Endpoints News . “While Pfizer has been working diligently to resolve the challenges, we decided to stop the development of sisunatovir.” The company will now focus on “identifying and advancing the development of other investigational therapeutics that have the greatest potential to prevent and treat RSV disease and other viral respiratory pathogens,” the spokesperson said. The cull wasn’t because of safety issues, Pfizer said. The drug, which has FDA fast track designation for both pediatric and adult indications, was still listed on Pfizer’s latest quarterly update in July and still appears on its pipeline page. With the ReViral deal, Pfizer was hoping to be able to both prevent RSV with its vaccine, and treat it with sisunatovir — much as it did for Covid-19 with a vaccine and antiviral pill. And when the companies announced the deal in 2022, then-ReViral CEO Alex Sapir called Pfizer “an optimal partner given their commitment to RSV.” At the time, Pfizer said it expected ReViral’s programs to bring in annual revenue of $1.5 billion or more. Pfizer is one of three pharma companies to market a vaccine for respiratory syncytial virus. The company makes Abrysvo , which was approved in 2023, while Moderna sells mResvia and GSK markets Arexvy. This season’s vaccine uptake has been slower than projected , Wall Street analysts have said. ReViral was one of many targets on which Pfizer has spent its Covid-19 cash. It acquired cancer drug developer Seagen for $43 billion, $11.6 billion to get migraine drugs from Biohaven, and $6.7 billion for Arena Pharmaceuticals and its immuno-inflammatory programs. It also spent $5.4 billion for sickle cell biotech Global Blood Therapeutics — only to pull the main product behind the deal for safety reasons.