STRATA's Patents Control Future of Laser-Drug Combination Therapies

STRATA Skin Sciences (NASDAQ: SSKN) has affirmed its strong patent portfolio position for combination therapies using its XTRAC excimer laser with JAK inhibitors, systemic and biologic drugs. The company holds three key patents that protect the combined use of their technology with various therapeutic agents for treating dermatologic conditions.

The company is working with CMS to implement expanded CPT codes that would cover all inflammatory and autoimmune skin conditions, potentially tripling their addressable market by January 2026. This expansion includes conditions like vitiligo, atopic dermatitis, and alopecia areata, beyond their current psoriasis treatment.

STRATA's patents specifically cover the combination of UVB light therapy with systemic drugs, JAK inhibitors, and biological drugs, positioning the company at the forefront of integrative dermatologic treatment. The company is also engaged in defensive litigation with LaserOptek, expecting significant damages and injunctive relief.

https://www.stocktitan.net/news/SSKN/strata-skin-sciences-ip-portfolio-holds-exclusivity-in-combination-47evzoz3s283.html

Mineralys Therapeutics Jumps On Baxdrostat Hypertension Trial Results

 Shares of Mineralys Therapeutics Inc. (MLYS) are up nearly 40% at $21 in premarket trading, following AstraZeneca's (AZN) positive trial results for Baxdrostat, a highly selective aldosterone synthase inhibitor, or ASI, for hypertension. As Mineralys is also developing ASIs targeting hypertension and related cardiorenal conditions, the news has triggered a domino effect, lifting sentiment across the ASI space.

Mineralys' lead drug candidate is Lorundrostat, a proprietary, orally administered, highly selective aldosterone synthase inhibitor being developed for the treatment of uncontrolled hypertension (uHTN) & resistant hypertension (rHTN), chronic kidney disease, and obstructive sleep apnea.

In March of this year, Mineralys announced positive results from its Launch-HTN and Advance-HTN trials.

Launch-HTN is a pivotal phase III trial evaluating the blood pressure-lowering effects of Lorundrostat in adults with uncontrolled hypertension, including treatment-resistant hypertension, who are taking 2-5 antihypertensive medications.

Advance-HTN is a pivotal phase II trial evaluating the blood pressure-lowering effect of Lorundrostat administered on a background of a standardized antihypertensive medication regimen in adults with uncontrolled hypertension, including treatment-resistant hypertension.

According to the trial results, in the Launch-HTN study, a 50 mg dose of Lorundrostat significantly lowered systolic blood pressure by 16.9 mmHg at Week 6. Compared to those who got a placebo, the drop was 9.1 points greater, which is considered highly significant. The study also met its predefined goal, with the 50 mg dose of Lorundrostat lowering systolic blood pressure by 19.0 mmHg at week 12. After adjusting for the placebo effect, the drug showed an 11.7 mmHg greater reduction, a result that was highly statistically significant.

https://www.rttnews.com/3570930/mineralys-therapeutics-jumps-on-baxdrostat-hypertension-trial-results.aspx

FDA’s New Radiopharma Guidance a Greenlight for an Accelerating Space

 

With a flurry of recent Big Pharma investment in radiopharmaceutical therapeutics, the FDA issued draft guidance last month in a move former FDA Commissioner Stephen Hahn sees as the regulator “trying to get ahead on a new set of therapy that they see becoming very important for cancer.”

After its March 2022 approval for certain types of castration-resistant prostate cancer, Novartis’ Pluvicto hit blockbuster status last year, clearing $1 billion in sales.

Just over three years later, the radiopharmaceutical sector is pushing at the edges, with some projections putting the market at more than $16 billion by 2033, and powerhouses like AstraZeneca, Bayer and, of course, Novartis are seeking a piece of the pie. Last month, the FDA provided these drugmakers with new draft guidance, a move former FDA Commissioner Stephen Hahn welcomed.

“I think what we’re seeing here is the agency trying to get ahead on a new set of therapies that they see becoming very important for cancer,” Hahn, who recently accepted the CEO position at North Carolina–based CDMO Nucleus RadioPharma, told BioSpace in an interview. “It’s nice to see the agency provide some clarity on what expectations should be.”

Hahn observed the accelerated trend in new drug applications (NDAs) and investigational new drug (IND) applications for radiopharmaceuticals. When this happens, he said, “it becomes pretty clear to review teams and leadership that there needs to be some consistency in the development process and what’s needed for approval.”

In April 2024, Novartis picked up another radiopharmaceutical approval, this time for Lutathera for pediatric patients with gastroenteropancreatic neuroendocrine tumors. Together with Pluvicto, sales totaled more than $2 billion that year. But Novartis could soon face competition, as a spate of large and small companies including specialty outfits like Perspective Therapeutics and giants like Eli Lilly, AstraZeneca and Bristol Myers Squibb also working in the space.

Hahn sees the FDA’s actions as providing a greenlight for the industry, with the regulator signaling that it expects to see the flow of radiopharma applications increasing further.With all the considerations that new modalities engender, he said the regulator is trying to keep tabs on issues that might occur down-the-line—an arduous process with which Hahn has first-hand experience.

Andrew Tsai, biopharma analyst and senior vice president at Jefferies, agreed that the FDA was driven to issue the guidance based on the increasing number of players in the space. “It’s good to know that the FDA is paying attention, and it suggests that the FDA wants to collaborate by issuing the guidance,” he told BioSpace. “They didn’t have to do this.”

A Whole New Field

The draft guidance, which is open for comments until Oct. 20, focuses on a range of subjects, particularly dose optimization, clinical trial design and patient selection, as well as safety concerns.

Radioligand therapies (RLT) comprise a radioactive isotope conjugated to a targeting molecule that guides the isotope to a specific tissue. In Pluvicto’s case, a lutetium isotope is joined with a prostate-targeting molecule. But the dosing for these types of therapies has often been based on traditional radiation treatments that has been used to treat cancer for more than a century.  

Mike Ritchie, chief commercial officer at cancer-focused CRO Champions Oncology, described radiation as “quite literally an external machine that shoots a beam into sites of tumors.”

“All of the clinical safeguards were around that type of radiation therapy,” he told BioSpace. “Fast forward to now, there’s a new field where we’re injecting radiation into patients, with a targeting moiety that treats the tumor in a targeted way.” Since targeted radiotherapy is still a new field, the FDA is seeking more details in applications—such as number of doses tested and toxicities that occur—because there is simply less familiarity with the modality at both the agency and across the sector.

“They’re saying that ‘we don’t know what we don’t know,’” Ritchie said.

Depending on the type of radiotherapy, the FDA may even ask sponsors to show a reading of the level of radiation within each organ, he continued. According to the new guidance, sponsors applying for INDs and NDAs should consider new safety precautions and exposure standards, given that RLTs get isotopes to tissues in a far more precise way than an external beam. For instance, becauseif the antigen is specific enough, RLTs should in theory only accumulate in the target organ. However, because many cancers express genes in off-target tissues, sponsors will need to keep tabs on radiation levels in multiple tissues during clinical trials.

Ritchie compared RLTs to a therapy with a similar structure: antibody-drug conjugates. The first ever FDA-approved ADC, Pfizer’s Mylotarg, got the greenlight in 2000 but was later withdrawn from the market later due to safety issues. That’s something the FDA would want to avoid with another new modality, he said. 

In the past, “ADCs have been clean in monkeys and in humans there was toxicity,” Ritchie continued. “The FDA is just doing what it should be: keeping patients first.”

A Healthy Sign

The FDA’s move to issue guidance on radiopharmaceuticals is a healthy sign for a sector that has seen a flurry of activity over the last few years. In 2024, four different Big Pharmas—Novartis, Eli Lilly, AstraZeneca and Bristol Myers Squibb—all made low single-digit billion dollar acquisitions of small radiopharmaceutical companies.

“There’s a good amount of money flowing in a space that people didn’t really think about 5, 10 years ago,” Tsai said. “At least in the investor’s lens, it’s caught people’s attention.”

To Hahn, the field is already on track to improve patients’ lives. He noted that Pluvicto’s initial approval was limited to subsets of prostate cancer patients, but the therapy has picked up multiple label expansions just this year.

“We really didn’t have an alternative to hormones and then chemotherapy in [castration-resistant prostate cancer] and chemotherapy has relatively modest effectiveness in that disease.”

There was significant unmet medical need in castration or hormone-resistant prostate cancer, Hahn said, “and being able to apply [radiopharmaceuticals], extend survival, improve quality of life and not make people sick with it is a really important advance.”

Hahn said he envisions a world where radiopharmaceuticals can be combined with currently available immuno-oncology agents to double up on both modalities’ effectiveness. “One could imagine that being the pathway of development in diseases that are very difficult to treat,” he said, like breast cancer, lung cancer and malignant melanomas.

“There’s a lot of situations where we really don’t have good tools or the tools are pretty toxic,” Hahn told BioSpace. “As you know in oncology . . . there are a lot of unmet medical needs.”

https://www.biospace.com/fda/fdas-new-radiopharma-guidance-a-greenlight-for-an-accelerating-space

'Trump’s Push To Shore Up Pharma Supply Chain With US API Welcomed but Questioned'

 

While trade groups hail the executive order as a national health security opportunity, analysts warn that production costs could go up in the near term.

Pharma industry trade groups have responded positively to President Donald Trump’s plans to make the supply chain more resilient. However, the upbeat response was offset by concerns about the near-term impact on the cost of drug production and a call for further actions to fully secure Americans’ access to medicines.

Trump’s executive order instructs the Administration for Strategic Preparedness and Response (ASPR) to create a list of around 26 drugs that are vital to national health and security within 30 days. Once the list is in place, U.S. authorities will prepare the existing Strategic API Reserve (SAPIR) to receive enough active pharmaceutical ingredient (API) to support a six-month supply of the vital drugs. Domestic API sources will be preferred for filling SAPIR.

In the second phase of the initiative, ASPR will update an existing list of 86 essential medicines and propose a plan to obtain and store a six-month API supply for the products. Trump has also tasked ASPR with developing a proposal for opening a second SAPIR.

The practicalities and implications of this plan remain murky, Truist Securities analyst Les Sulewski and associate Jeevan Larson wrote in an Aug. 14 note to investors.

“The intent of the administration is to incentivize onshoring of API manufacturing and integrate the drug manufacturing process in the U.S. for critical drugs. We think API suppliers and marketers of these ‘to be selected’ medicines will be mandated to act accordingly,” the Truist team wrote. “However, it is unclear at this time which drugs will be selected and what steps will be needed, if at all, to on-shore production.”

Industry Backs Trump’s Plan

The lack of information on what drugs are covered by the order is a key uncertainty at this stage, John Murphy, president and CEO of the Association for Accessible Medicines (AAM), told BioSpace. Yet, while Murphy has some unanswered questions, the generic drug trade group leader sees the potential benefits of the executive order for this class of medicines.

“We view this as a promising step in the direction of creating a more resilient supply chain for generic drugs in the United States,” Murphy said. “We’ve long said that the government could play a role in stabilizing the market for generic drugs by serving as a direct purchaser and providing certain long-term commitments to help facilitate the investment environment.”

Representatives of the API industry also responded positively to the president’s moves on this front. A spokesperson for the Bulk Pharmaceutical Task Force (BPTF) told BioSpace via email that the API trade group “supports the initiative defined” in the executive order, adding that it “fully supports this Administration’s efforts to leverage domestic API capacity, where possible.”

A spokesperson for API Innovation Center (APIIC), a nonprofit focused on U.S.-based supply of APIs, told BioSpace via email that the order “is an important opportunity for U.S. national health security.”

Historically, Murphy argued, purchasers in the U.S. have focused on price at the expense of supply chain security, ultimately resulting in generics that are cheaper than in other countries. But the prices are only profitable when APIs and drugs are made in low-cost locations, the argument goes, driving production away from the U.S. If production is to return to the U.S., prices may need to increase to the point that U.S.-based production is profitable.

“Many of the insurers and many of the group purchasing organizations literally look at nothing but cost and they go for the lowest cost possible. That’s led to that shift away from U.S. domestic manufacturing,” he said. “The only way to get a piece of that back is to prioritize government and insurance company purchases of those products that are available in and made in the United States.”

The BPTF spokesperson agreed that a shift away from price is warranted and specifically recommended that purchasing decisions consider the domestic API industry’s sustainability, quality, patient health and safety.

While welcoming the executive order, Murphy also called for reform of pay systems to help support patient access to drugs. “I don’t think it is going to be the only thing that is necessary to fully change our supply chain behavior.”

Analysts Flag ‘Prohibitive Costs’

Sulewski and Larson outlined potential downsides to Trump’s executive order. While agreeing that the move “could potentially mitigate U.S. supply chain vulnerabilities long-term,” the Truist analysts warned that generic and API companies could face implementation challenges and higher costs in the near term.

“Vertical integration and on-shoring of precursor chemicals, API and finished dosing manufacturing is cost prohibitive to the majority of suppliers and disruptive to the global supply chain, specifically U.S. manufacturing of API and input material sourcing,” Truist wrote.

The analysts predicted that if forced to act, “API suppliers will selectively on-shore products under mandate but demand economically feasible contracts and longevity.” Limiting their analysis to companies they cover, Sulewski and Larson said ANI Pharma, Amneal Pharmaceuticals, Bausch Health and Teva Pharmaceuticals could face “some potential disruption.” The analysts wrote that the impact “should be manageable.”

Teva has “by far the largest API and generics exposure” among the companies Truist covers, they continued.. However, the company could reduce its exposure if the proposed divestiture of its API business goes ahead.

https://www.biospace.com/policy/trumps-push-to-shore-up-pharma-supply-chain-with-us-api-welcomed-but-questioned

New Wave of ALS Therapies Signals Renaissance After Incremental Regression

 

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.”

https://www.biospace.com/drug-development/new-wave-of-als-therapies-signals-renaissance-after-incremental-regression

Rare Diseases Secure Four FDA Firsts in August

 

Aside from the rare disease market, Novo Nordisk also scored a key regulatory win last month for its blockbuster GLP-1 drug Wegovy, which can now be used to treat patients with metabolic dysfunction-associated steatohepatitis.

August was a big month for the rare disease space, which saw four history-making approvals—though the streak was marred by one rejection. Also walking away with a win last month was Novo Nordisk, which secured a critical label expansion that, according to analysts, could help its blockbuster GLP-1 medication gain access to nearly $2 billion in added peak sales.

Read below for more.

Four Firsts for Rare Diseases

Company: Jazz Pharmaceuticals

Drug: Modeyso

Indication: Glioma

The first rare disease nod came on Aug. 6 for Jazz Pharmaceuticals’ dordaviprone, which was approved for patients aged 1 year and above with diffuse midline glioma with an H3 K27M mutation who have progressed after prior treatments. The drug, which will carry the brand name Modeyso, is the first systemic therapy for this specific type of glioma, according to the FDA’s announcement of the approval.

Modeyso was approved under the FDA’s accelerated pathway, supported by overall response (OR) data from a pair of Phase I and Phase II studies. According to Jazz’s news release announcing the approval, Modeyso achieved an OR of 22% in 50 adult and pediatric patients. Among responders, median duration was 10.3 months. To maintain Modeyso’s approval, Jazz is running a confirmatory Phase III trial that is expected to be complete in August 2026.

Company: Insmed

Drug: Brinsupri

Indication: Non-cystic fibrosis bronchiectasis

Not a week after Jazz, Insmed secured an FDA nod for brensocatib—now sold as Brinsupri—for the rare respiratory disorser non-cystic fibrosis bronchiectasis. Brinsupri, taken orally once daily, is indicated for patients aged 12 years and up. Insmed has set its price at $88,000 per year—“a little higher” than what analysts were expecting, Guggenheim Partners wrote at the time.

Brinsupri marks two firsts for the biopharma industry: Not only is it the first drug for bronchiectasis to reach the market, but it is also the first approved treatment that works by blocking DPP1, an enzyme that plays a role in activating the inflammatory response in airways.

Brinsupri is backed by data from the Phase III ASPEN trial. Data published in The New England Journal of Medicine in April showed that Brinsupri lowered the rate of pulmonary exacerbations by around 20% versus placebo. The Phase II WILLOW trial also supported Brinsupri’s approval, demonstrating a roughly 40% reduction in the risk of exacerbations relative to placebo.

Company: Precigen

Drug: Papzimeos

Indication: Recurrent respiratory papillomatosis

Then, on Aug. 14, Precigen won the FDA’s go-ahead for zopapogene imadenovec-drba, now named Papzimeos, for the treatment of recurrent respiratory papillomatosis (RRP). In its announcement of the approval, the FDA said Papzimeos is a “first-of-its-kind” non-replicating immunotherapy for this disease.

With around 1,000 new cases in the U.S. annually, RRP is a rare disease that manifests as benign tumors in the airways, leading to difficulties in swallowing and breathing. If left unchecked, RRP can lead to death. Papzimeos works by helping the body mount an immune response against cells infected by HPV 6 and HPV 11, both associated with RRP. Pivotal Phase I/II data supported Papzimeos’ approval, showing that the biologic elicited a complete response rate of more than 50% in treated patients, while over 85% needed fewer surgical interventions in the year after treatment.

Company: Ionis Pharmaceuticals

Drug: Dawnzera

Indication: Hereditary angioedema

Capping off the rare disease rally this month is Ionis Pharmaceuticals. On Aug. 22, the California biotech’s antisense oligonucleotide donidalorsen, now branded Dawnzera, became the industry’s first RNA-targeting prophylactic for hereditary angioedema (HAE). Patients 12 years and older can receive the therapy, which is given via a subcutaneous injection every four weeks.

Dawnzera targets the prekallikrein mRNA, causing its destruction. This, in turn, reduces overall levels of PKK, a protein that plays a role in swelling and pain attacks in HAE. Data from the OASIS-HAE study backed Thursday’s approval, demonstrating an 81% reduction in HAE attack rate versus placebo over 24 weeks of observation. Results were published May 2024 in the New England Journal of Medicine and additionally showed a significant improvement in patients’ quality of life.

Wegovy Win For Novo

On Aug. 15, the FDA gave the go-ahead for Novo Nordisk’s blockbuster injection Wegovy to be used in adults with metabolic dysfunction-associated steatohepatitis. The GLP-1 drug, indicated for patients with moderate to advanced liver scarring but without cirrhosis, should be used in conjunction with a reduced-calorie diet and higher physical activity.

Wegovy’s approval in MASH is a “step in the right direction” for Novo, analysts at BMO Capital Markets wrote in an Aug. 17 note to investors, adding that breaking into the MASH market “could start to help shift the momentum” for Wegovy, which in the first half of 2025 has been hit hard by the rise of compounders. BMO anticipates peak MASH sales of $1.9 billion for Wegovy.

Data from the Phase III ESSENCE trial, which supported the label expansion, showed that Wegovy improved liver fibrosis without worsening steatohepatitis in 37% of treated patients at 72 weeks, versus 22.5% in placebo comparitors. At the same time, Wegovy resolved steatohepatitis without worsening fibrosis in 62.9% of patients, as compared with 34.1% of placebo participants.

Wegovy has “clear efficacy in MASH,” the BMO analysts wrote, adding that its “clean safety profile and broad benefits across metabolic disease” could help establish the drug “as a backbone treatment for MASH.”

Wegovy joins Madrigal Pharmaceuticals’ Rezdiffra in the MASH space. Approved in March 2024, Rezdiffra made $180.1 million last year.

Three COVID-19 Vaccines Cleared, With Restrictions

The FDA on Aug. 27 approved updated COVID-19 vaccines from Pfizer, Moderna and Novavax, but with key limitations: The shots can only be used in adults 65 years and older and younger people who are at elevated risk of severe outcomes.

There are some minor differences across the three, particularly as it pertains to use in the at-risk younger population.

• Novavax’s Nuvaxovid, a protein-based shot, is authorized for individuals 12 through 64 years.
• Pfizer and BioNTech’s mRNA-based Comirnaty can be given to children as young as 5 years.
• Moderna’s mNEXSPIKE is indicated for people 12 through 64 years, while Spikevax is indicated for individuals 6 months through 64 years. Both vaccines are mRNA-based.

These approvals come after Health Secretary Robert F. Kennedy Jr. in May removed routine COVID-19 vaccination for healthy children and healthy pregnant women from CDC recommendations.

PTC Hit With Rejection in Friedreich’s Ataxia

The FDA on Aug. 19 turned down PTC Therapeutics’ vatiquinone, which the company has proposed for the treatment of Friedreich’s ataxia in children and adults.

In its complete response letter, the FDA stated that “substantial evidence of efficacy was not demonstrated” and that PTC would need an additional “adequate and well-controlled study” to support a resubmission. Matthew Klein, the company’s CEO, said in a statement at the time that PTC is planning to meet with the FDA to discuss potential next steps.

Vataquinone is a small molecule that blocks some of the cellular pathways that go awry in patients with frataxin mutations, the underlying cause of Friedreich’s ataxia. Patients with this rare neuromuscular disease suffer from loss of coordination and muscle strength, as well as difficulty speaking, swallowing and breathing. Around 25,000 patients have been diagnosed worldwide, according to PTC.

Vataquinone missed the mark in a Phase III, registration-directed trial back in 2023, failing to meet its primary endpoint of improving gait, stability and limb function after 72 weeks. PTC sought registration with the FDA based on secondary outcomes such as stability.

https://www.biospace.com/fda/rare-diseases-secure-four-fda-firsts-in-august

Lilly Ends Two Mid-Stage Trials for Second Obesity Asset

 

While Eli Lilly’s orforglipron is full speed ahead for a regulatory filing this year, the pharma is also pushing forward with one more Phase II study of naperiglipron, which uses the same scaffold as Pfizer’s failed obesity drugs danuglipron and lotiglipron.

Eli Lilly has terminated two Phase II studies of naperiglipron, an investigational oral obesity therapy, while it continues to forge ahead with a third mid-stage trial.

Both studies were discontinued for “strategic business reasons,” according to updates to their respective clinicaltrials.gov pages. A spokesperson for Lilly, who confirmed the trial terminations to Endpoints News on Friday, declined to provide additional information, noting only that results from the remaining ongoing study of naperiglipron “will inform the next steps for this program.”

One of the cut studies was originally designed to test the weight loss pill versus placebo in 150 patients with overweight or obesity and type 2 diabetes. Only one patient enrolled in the trial. Similarly, the second study was only able to onboard one patient out of its goal of 220 participants. It was designed to focus on a population of adults aged 55 to 80 years with a body mass index between 22 and 25 kg/m2.

The last remaining ongoing study for naperiglipron remains open to enrollment, with a recruitment target of 275 patients with overweight or obesity. Participants will be dosed once daily with naperiglipron or placebo. According to its clinicaltrials.gov page, the study began in November last year and has a primary completion date of April 2026.

Naperiglipron is an orally available small molecule agonist of the GLP-1 receptor. Like other drugs in this class, it works by promoting the release of insulin from the pancreas in response to blood sugar levels, and by suppressing appetite. In a June 23 note to investors, however, analysts at BMO Capital Markets noted that naperiglipron uses a similar design scaffold as Pfizer’s danuglipron and lotiglipron—both of which have been discontinued due to safety issues.

“While history for the scaffold has been challenging, we are interested to learn more why Lilly believes it can evade these past pitfalls,” BMO wrote at the time, adding that the asset could also help the pharma diversify its oral obesity portfolio.

Currently, Lilly’s oral weight-loss push is anchored by the late-stage orforglipron, which likewise works by activating the GLP-1 receptor. While many analysts consider orforglipron the de facto leader in the oral obesity space, recent readouts have cooled expectations. Last month, for instance, a readout from the Phase III ATTAIN-1 study disappointed investors, sending the pharma’s shares sliding 7% in its aftermath.

In the trial, a 36-mg dose of orforglipron cut weight by 12.4% in patients with obesity or overweight but not diabetes, falling shy of the market’s 13.4% expectation based on Novo Nordisk’s injectable semaglutide.

Last week, results from the late-stage ATTAIN-2 trial demonstrated a 10.5% weight reduction in patients with overweight or obesity and type 2 diabetes. These data, according to analysts at Truist Securities, were “in-line” with expectations, but leave “room for competition.”

https://www.biospace.com/drug-development/lilly-ends-two-mid-stage-trials-for-second-obesity-pill