Tag: Biopharma

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STAT+: Pharmalittle: We’re reading about a telehealth firm and patient safety, PBMs overcharging, and more

Good morning, everyone, and welcome to another working week. We hope the weekend respite was relaxing and invigorating because that oh-too-familiar routine of meetings, deadlines, and the like has returned with a vengeance. You knew this would happen, yes? To cope, we are relying, as always, on cups of stimulation. Our choice today is laced with traces of cocoa. Feel free to join us. Remember, no prescription is required. Meanwhile, here are some tidbits to help you along. Best of luck accomplishing your goals today and, of course, do keep in touch. …

Novo Nordisk, maker of Ozempic and Wegovy, lists the telehealth company LifeMD on its website as a provider that offers “legitimate medicine sourcing and patient support” for people seeking GLP-1 drugs. But some former employees describe LifeMD differently, as a company that has sought to maximize the volume of prescriptions it doles out at the expense of patient safety, STAT reports. Former workers told STAT that providers were pressed to expedite their work to a pace that was not clinically responsible, with two of them saying providers at times were expected to review the cases of 25 people per hour based only on electronic forms the patients filled out — the equivalent of spending about two minutes on each case.

A recent audit of Iowa Medicaid records revealed how pharmacy benefit managers are using complicated and sophisticated approaches for handling prescription drug claims that ultimately overcharge taxpayers, a finding that underscores controversy surrounding these crucial middlemen in the pharmaceutical supply chain, STAT explains. The audit found that one large pharmacy benefit manager appeared to have made more than $100 million by adjusting the amount of money paid to pharmacies without passing some of the savings back to managed care plans working on behalf of the state, according to Iowa officials. The review scrutinized records from 2019 through 2021.

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STAT+: Bristol Myers Squibb becomes latest company to claim it’s building pharma’s largest NVIDIA AI supercomputer

It’s officially a trend. For the third time in nine months, a pharma company has announced that it is assembling the largest AI supercomputer in the life sciences industry. This time, it is Bristol Myers Squibb. 

When the company began its partnership with NVIDIA three years ago with a smaller computing cluster, it was focusing on simpler problems with individual AI tools, like protein structure prediction. But “we actually consumed all the space we had,” said Greg Meyers, chief digital & technology officer at BMS.

Adding extra computing power is necessary for the company now that it’s “become more convinced” that computationally hungry foundation models can give the company valuable insight into how its drug candidates interact with both the body and with disease, Meyers said in an interview with STAT. He mentioned oncology and neurodegeneration as examples of areas where BMS has developed such models.

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STAT+: State audit of Medicaid records points to methods used by PBMs to obscure drug costs

A recent audit of state Medicaid records revealed how pharmacy benefit managers are using complicated and sophisticated approaches for handling prescription drug claims that ultimately overcharge taxpayers, a finding that underscores controversy surrounding these crucial middlemen in the pharmaceutical supply chain.

The audit of Iowa’s state Medicaid program found that one large pharmacy benefit manager appeared to have made more than $100 million by adjusting the amount of money paid to pharmacies without passing some of the savings back to managed care plans working on behalf of the state, according to Iowa officials. The review scrutinized records from 2019 through 2021.

The overall conclusion was similar to audit results conducted in a few other states, but in this instance, the auditors identified what amounted to creative accounting maneuvers, which not only made it possible to obscure the flow of money but evade prohibitions on a controversial pricing practice that is now outlawed in Iowa and some other states.

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StockWatch: Lilly’s Up-to-$3.8B Deal for AtaiBeckley a Good Trip for Psychedelic Drugs, Analysts and Investors Agree

It wasn’t too long ago that biopharma giants stayed away from developing psychedelic drugs—but positive clinical data plus a friendlier regulatory climate in Washington have prompted the largest drug developers to embrace the field.

The latest and most telling example of pharma embracing psych drugs came when Eli Lilly (NYSE: LLY) announced that it agreed to acquire AtaiBeckley (Nasdaq: ATAI) for up to $3.8 billion—of which Lilly will pay $2.8 billion upfront. The deal, set to close in the third quarter, expands Lilly’s neuroscience portfolio by adding the pipeline of AtaiBeckley led by BPL-003 (mebufotenin benzoate), a Phase III candidate for treatment-resistant depression (TRD) that is a synthetic form of 5-MeO-DMT administered intranasally. BPL-003 has been granted the FDA’s Breakthrough Therapy designation.

BPL-003 wowed analysts and others back in April after AtaiBeckley published positive data from a Phase IIa trial (NCT05660642) showing that a single intranasal dose of BPL-003 led to rapid and sustained reductions in Montgomery-Åsberg Depression Rating Scale (MADRS) scores from baseline in 12 TRD patients who remained on stable SSRI therapy throughout the study. Both the six patients dosed at 10 mg and six at 12 mg showed a 66.7% antidepressant response rate (defined as ≥50% reduction from baseline MADRS score) at Day 2, with five of six participants in the 10 mg cohort (83%) and four of six in the 12 mg cohort (66.7%) maintaining their response at Week 12.

“Especially with progress on BPL-003, we see the company as positioning itself well to becoming a significant player in the mental health therapeutics space,” Sumant Kulkarni, a senior analyst covering biotechnology with Canaccord Genuity, wrote on news of the positive data, adding: “We also still see this space as large enough to accommodate multiple approaches/competitors.”

$3.7B in projected peak sales

Kulkarni also raised Canaccord Genuity’s peak unadjusted U.S. sales forecast for BPL-003 to $3.7 billion by 2036 from $2 billion, after revising the firm’s model by raising the list price from $20,000 to $30,000 per annual treatment course (not accounting for insurance coverage), about the same price as Spravato® (esketamine), also a nasal spray marketed by Johnson & Johnson (NYSE: JNJ) for TRD plus some depressive symptoms in adults with major depressive disorder (MDD).

Spravato, a noncompetitive N-methyl D-aspartate (NMDA) receptor antagonist, crossed the $1 billion sales threshold during the second quarter, as it generated $584 million, up 25% quarter-over-quarter from $464 million in Q1—and up 43% from $734 million in the first half of 2025.

“Sales are tracking to reach annual sales guidance of $3-3.5B+ by 2027–28,” Jefferies equity analyst Andrew Tsai wrote in a research note focused on J&J’s second-quarter results. “Spravato’s trajectory supports the notion psychedelics can be commercially viable in hard-to-treat mental health disorders, by leveraging JNJ’s infrastructure.”

Given the data for BPL-003, Lilly got a bargain, Tsai wrote in a separate note on the Lilly-AtaiBeckley acquisition.

“We think the deal heavily favors LLY, as ATAI’s lead asset BPL-003 (intranasal 5-MeO-DMT) should have multibillion dollar peak sales potential,” Tsai wrote, rather than the $1 billion-plus that he thinks was implied by the deal price.

Tsai and Jefferies had previously forecast peak sales of between $1 billion and $2 billion—a range he said was “arguably conservative” since BPL-003 could, if it aces its Phase III trial, show superiority to Spravato, which is on track to reach up to $5 billion-plus in peak sales.

Positive implications

“At the same time, we appreciate LLY has significantly more resources to maximize the long-term value of ATAI’s psychedelic assets. In any case, the deal has (+) [positive] implications for the entire psychedelic space,” Tsai added.

Among pharma giants joining J&J in embracing psychedelic drug development in recent years:

  • AbbVie (NYSE: ABBV), which last year acquired the lead pipeline program of privately held Gilgamesh Pharmaceuticals, the moderate-to-severe MDD candidate bretisilocin (GM-2505), for up to $1.2 billion.
  • Otsuka Holdings (Tokyo Stock Exchange: 4578), which in 2023 acquired Mindset Pharma, a Canadian psych drug developer focused on psychiatric and neurological disorders, for C$80 million ($56 million).

With its deal for AtaiBeckley, Lilly becomes the latest pharma giant to perceive the positive implications Tsai cited.

“Treatment-resistant depression persists even after multiple treatments have failed. Millions of people are still searching for relief and desperately need a therapy that works,” Carole Ho, executive vice president and president, Lilly Neuroscience, said in a statement. “Advancing AtaiBeckley’s investigational therapies gives us a real chance to change that.”

Investors agreed with Lilly, giving the pharma a 1% increase Thursday, the day the acquisition was announced, from $1,156.63 to $1,169.17—no small feat since buyers typically stay flat or see their shares slide after announcing an acquisition. And not surprisingly, AtaiBeckley investors were enthusiastic about the deal, as its stock leaped 33% from $5.36 to $7.15. On Friday, Lilly inched up 0.8% to $1,178.58 while AtaiBeckley rose 1% to $7.22.

The AtaiBeckley buyout is Lilly’s eighth announced acquisition of a smaller biopharma this year.

Lilly is acquiring three infectious diseases vaccine developers—Vaccine Company for up to $1.55 billion, Curevo for up to $1.5 billion, and LimmaTech Biologics for up to $780 million—as well as in vivo chimeric antigen receptor T-cell (CAR T) developer Kelonia Therapeutics for up to $7 billion); JAK inhibitor developer Ajax Therapeutics for up to $2.3 billion; next-generation dual-payload antibody-drug conjugate (ADC) developer CrossBridge Bio for up to $300 million; and nonviral DNA delivery-focused drug developer Engage Biologics for up to $202 million cash.

The deal spree reflects Lilly’s desire to capitalize on the billions of dollars it is generating from sales of its obesity and diabetes drugs based on glucagon-like peptide 1 (GLP-1) receptor agonists alone or in tandem with a glucose-dependent insulinotropic polypeptide (GIP).

“If we see great ideas that we think we can use to help people that need them, of course we’ll do deals,” Daniel M. Skovronsky, MD, PhD, Lilly’s chief scientific and product officer and president of Lilly Research Laboratories, said on CNBC.

“Positive development”

David Risinger, a senior managing director and senior research analyst covering diversified biopharmaceuticals at Leerink Partners, said his firm viewed Lilly’s buyout of AtaiBeckley “as a positive development because it enhances LLY’s pipeline of potential neuroscience blockbuster candidates.”

That pipeline is led by five Phase III programs involving four drugs, none of them a psychedelic. Two of the programs belong to brenipatide, a dual agonist of both the GIP and GLP-1 receptors. Brenipatide is being developed for both MDD and alcohol use disorder.

Also in Lilly’s late-stage neuroscience pipeline are:

  • Donanemab, which binds to deposited amyloid plaque in the brain and is being studied for the treatment of cognitively unimpaired Alzheimer’s disease.
  • Ixoberogene Soroparvovec (Ixo-Vec), an intravitreal gene therapy being studied as a single one-time treatment for vision loss associated with neovascular (wet) age-related macular degeneration (AMD).
  • Remternetug (LY3372993), which also binds to deposited amyloid plaque in the brain and is under study as a treatment of cognitively unimpaired/mild cognitive impairment due to Alzheimer’s disease, with potential for subcutaneous delivery.

In addition, AtaiBeckley “would provide ​differentiated exposure in psychiatry and reinforce [Lilly’s] ​broader effort to diversify beyond ​its cornerstone cardiometabolic franchise,” observed Evan David Seigerman, a managing director and head of healthcare research at BMO Capital Markets, as reported by Reuters.

AtaiBeckley was formed last November by the merger of atai Life Sciences and Beckley Psytech. The company’s stock has nearly doubled, soaring 98% over the past six months from $3.64 on January 16.

“Going mainstream”

“Psychedelic Medicine is going mainstream,” declared Steve Jurvetson, co-founder of Future Ventures, in a post on X. Jurvetson and Future were among early investors, along with Peter Thiel in atai Life Sciences.

AtaiBeckley is one of numerous psychedelic drug developers to show significant six-month gains since January: As of Friday’s closing bell, Compass Pathways (Nasdaq: CMPS) shares jumped 68% to $12.35, GH Research ballooned 69% to $28.71, while Definium Therapeutics (Nasdaq: DFTX) nearly tripled, zooming 194% to $44.29.

Interestingly, those three companies did not get a solid bounce from AtaiBeckley’s acquisition by Lilly. Since the deal was announced Thursday, Compass fell 7% from $13.31 pre-announcement, Definium dipped 3% from $45.66. GH rose 8% Thursday from $26.92 to $29.13, before sliding 1.4% the following day.

Bucking the trend was Cybin, d/b/a Helus Pharma (Nasdaq: HELP), which has climbed 11% since the Lilly-AtaiBeckley announcement, from $6.51 to $7.25. Its shares have slumped 6% since January—but soared 58% over the past month on positive news, such as the 88%+ enrollment rate of patients in Helus’ Phase III APPROACH pivotal trial (NCT06564818) of HLP003 in MDD, on track for topline data readout in Q4 2026.

“We see the potential for 150–200% upside [jump in stock price] if Phase III data in 4Q26 are positive,” Kulkarni wrote, making it the largest potential jump among psychedelic drug developers.

In addition to favorable data, the stock surges also reflect actions by President Donald J. Trump’s administration to encourage psychedelic drug development. In April, President Trump signed Executive Order 14401, directing the FDA and other federal agencies to accelerate research and improve access to psychedelic drugs, citing their potential as promising treatments for serious mental illnesses.

And on July 13, the FDA published “Psychedelic Drugs: Considerations for Clinical Investigations,” a final guidance designed to provide general considerations for developers of psych drugs, with recommendations for how to conduct clinical trials for the treatments.

“Rather than providing specific recommendations on study design, this guidance will present foundational constructs that all sponsors studying the therapeutic potential of psychedelic drugs, including sponsors without commercial drug development as primary interest (e.g., academic researchers), should consider,” the FDA wrote in the final guidance. “Sponsors are encouraged to request meetings with FDA for advice on a specific drug development program.”

Leaders and laggards

  • Q32 Bio (Nasdaq: QTTB) shares nearly doubled, leaping 91% from $11.21 to $21.38 July 13 after the autoimmune and inflammatory disease drug developer announced positive 36-week topline results from Part B of the Phase IIa SIGNAL-AA trial (NCT06018428) assessing bempikibart in patients with severe or very severe alopecia areata. Q32 said it saw clinically meaningful efficacy data on the primary endpoint of mean percent change from baseline in SALT score, with a reduction from baseline of 35.3% in the prespecified modified intent to treat (mITT) analysis. The company also reported that 40.0% of patients (10/25) achieved SALT-20 response at Week 36 in the mITT analysis, while 30.3% of patients (10/33) achieved SALT-20 response at Week 36 in the ITT analysis of all enrolled patients.
  • Veradermics (NYSE: MANE) shares yo-yoed this past week, climbing 12% from $110.17 to $123.70 Wednesday after the pattern hair loss drug developer announced positive topline results from its open-label Phase II Study 207 trial (NCT06527365) assessing VDPHL01, an extended-release oral minoxidil formulation, in women with mild-to-moderate pattern hair loss. Veradermics said most study participants reported improved hair coverage at Month 2, with approximately 88.9% of patients dosed once daily and 90.0% dosed twice daily reporting “improved” or “much improved” outcomes at Month 6. Participants dosed once daily showed a mean increase in non-vellus target area hair count (TAHC) of 22.7 hairs/cm² at Month 6, an average that rose to 23.3 hairs/cm² in twice daily dosed patients. The mini surge was short-lived, however, as investors more than gave back the gain, selling off shares to send them tumbling 14% to $105.83 Thursday amid possible investor questions about whether the good clinical news was already reflected in the stock price.

The post StockWatch: Lilly’s Up-to-$3.8B Deal for AtaiBeckley a Good Trip for Psychedelic Drugs, Analysts and Investors Agree appeared first on GEN – Genetic Engineering and Biotechnology News.

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STAT+: Kalshi comes for biopharma, with prediction markets for clinical trials and FDA approvals

Prediction markets have permeated the worlds of sports, politics, and reality television shows. Now, the biopharma industry could be next.

On Thursday, Kalshi, one of the world’s largest prediction market exchanges, said it will start taking bets on clinical trials and regulatory approvals.

It’s starting with a small number of markets chosen in collaboration with its partner, AppliedXL, a tech company that monitors and predicts the outcomes of clinical trials.

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STAT+: Eli Lilly to acquire psychedelics-focused biotech AtaiBeckley

Eli Lilly is acquiring AtaiBeckley, the developer of psychedelic treatments for mental health conditions, to expand its portfolio of neuroscience medicines, the company said Thursday.

The AtaiBeckley deal is just the latest in a string of acquisitions by the pharma giant, flush with cash from its booming GLP-1 business. 

Lilly is paying $2.8 billion in cash upfront for AtaiBeckley, with the potential for another $1 billion payout contingent on certain development and regulatory milestones.

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AstraZeneca Licenses Global Rights to Dizal Lung Cancer Drug for Up-to-$1.5B

AstraZeneca has acquired exclusive global rights to develop and commercialize Dizal Pharmaceutical’s marketed lung cancer drug Zegfrovy® (sunvozertinib), through an agreement that could generate up to $1.5 billion for the spinout of the pharma giant’s onetime Chinese R&D operation.

Wuxi City, China-based Dizal has inked an exclusive license agreement with AstraZeneca to expand the development of Zegfrovy into new indications beyond the one for which it has approvals in the United States and China—namely the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations, whose disease has progressed on or after platinum-based chemotherapy.

Dizal has been pursuing approvals from the FDA and China’s Center for Drug Evaluation (CDE) for a new indication for Zegfrovy, as a first-line treatment for NSCLC with exon 20 insertion EGFR mutations. In May, Dizal presented at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting and simultaneously published in The New England Journal of Medicine (NEJM), positive results in the indication from its Phase III WU-KONG28 trial (NCT05668988).

In the study, Zegfrovy showed a median progression-free survival (PFS) of 10.3 months compared with 7.5 months PFS for platinum-doublet chemotherapy in untreated NSCLC patients with EGFR exon 20 insertion mutations (exon20ins).

“The efficacy of sunvozertinib was superior to that of chemotherapy as first-line treatment for advanced NSCLC with EGFR exon 20 insertions,” the researchers concluded in their study, “First-Line Sunvozertinib in NSCLC with EGFR Exon 20 Insertion Mutations,” which was published May 29 in NEJM.

Dizal also showed Zegfrovy delivering a BICR-assessed best objective response rate (BoR) of 68.1% vs. 35.4% with chemotherapy, and a median duration of response (DoR) of 11.2 months vs. 7.1 months for chemo.

Based on those results, Dizal has filed supplemental New Drug Applications (NDAs) for Zegfrovy in the first line to the FDA and China’s Center for Drug Evaluation (CDE). Both regulators have granted their Breakthrough Therapy designations to Zegfrovy in that setting.

“AstraZeneca is a leader in treating EGFR-mutated lung cancer, and we are eager to add Zegfrovy to our world-class portfolio of innovative medicines for patients whose tumors carry exon 20 insertion mutations,” Dave Fredrickson, executive vice president of AstraZeneca’s Oncology Hematology Business Unit, said in a statement. “With this agreement, we will bring a differentiated, oral targeted treatment to these patients with limited options across the globe.”

20% jump

Dizal shareholders reacted to the agreement with AstraZeneca warmly enough to send shares traded on the Shanghai Stock Exchange jumping 20%, from RMB 46.94 ($6.93) to RMB 56.33 ($8.31). But the news did not appear to wow AstraZeneca investors, as shares of the pharma giant traded on the London Stock Exchange dipped 1.95% today, from 12,610 pence to 12,364 pence. Shares traded on the New York Stock Exchange also fell 1.95% as of 2:17 pm ET, from $169.47 to $166.16.

Dizal was established in 2017 as a joint venture between AstraZeneca and China’s State Development & Investment Corp. (SDIC), with AstraZeneca spinning out the R&D operations of its China Commercial Innovation Center to Dizal as well as three preclinical candidates, one each in cardiometabolic disease, respiratory disease, and oncology, the drug that was eventually developed into Zegfrovy. Xiaolin Zhang, PhD, who headed the innovation center, was appointed Dizal’s CEO, a position he still holds.

AstraZeneca has agreed to pay Dizal $600 million upfront; up to $900 million tied to achieving development, regulatory, and sales-related milestones; plus tiered double-digit royalties on global sales of Zegfrovy. The milestone payments consist of up to $400 million in clinical development-related payments and up to $500 million in sales-related payments, Dizal disclosed in a regulatory filing to the Shanghai Stock Exchange.

In March, Dizal reported that Zegfrovy generated about RMB 576 million (about $85.057 million) in revenue last year, up 85% from 2024. Zegfrovy accounted for nearly three-fourths (72%) of Dizal’s total 2025 sales of RMB 801 million ($118.282 million).

Zegfrovy is a once-daily oral irreversible epidermal growth factor receptor (EGFR) inhibitor approved by the FDA in July 2025 based on evidence from the Phase I/II WU-KONG1B trial (NCT03974022) in patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations whose disease has progressed on platinum-based chemotherapy and received Zegfrovy 200 mg once daily with food.

WU-KONG1B enrolled 202 patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations who had received previous platinum-based chemotherapy. The trial was conducted at 89 sites in the United States, Argentina, Australia, Canada, China, Chile, France, Italy, Malaysia, South Korea, Spain, and Taiwan.

AstraZeneca’s licensing deal with Dizal is expected to close in the second half of this year, subject to customary closing conditions and regulatory clearances. AstraZeneca said the transaction does not impact its 2026 financial guidance to investors, which it reaffirmed on April 29 as calling for a mid-to-high single-digit percentage increase in total revenue, and a low double-digit increase in “core” earnings per share from primary ongoing business activities.

“As a leading global company with a strong lung cancer franchise, AstraZeneca will help ensure patients around the world can benefit from this innovation discovered by Dizal scientists in China,” stated Zhang.

The post AstraZeneca Licenses Global Rights to Dizal Lung Cancer Drug for Up-to-$1.5B appeared first on GEN – Genetic Engineering and Biotechnology News.

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Opinion: It looks like your doctor and talks like your doctor. But it’s not your doctor

Imagine a patient who arrives at her doctor’s clinic furious. She shows her doctor a video of him — white coat, plausible exam room, familiar cadence — endorsing an over-the-counter hormone supplement for menopausal symptoms, dismissing standard therapies as “pharma scams,” and offering a discount code.

But the physician never recorded that message. Someone built a deepfake from online recordings, including interviews, webinars, and patient-facing videos, and used the synthetic likeness to sell an unregulated product. This scenario is no longer hypothetical. Investigations have documented AI-generated videos impersonating specific clinicians whom they name to promote supplements and other dubious treatments on major platforms. 

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StockWatch: Insilico Projects Profit, Revenue Leaps as AI-Developed Lead Candidate Moves to Phase III

Insilico Medicine (Hong Kong Exchange: 3696), an AI-based drug developer whose profile within biopharma has risen with its recent collaborations with industry giants, has offered investors an upbeat revenue and profit forecast for the first half of 2026, driven by its series of partnerships and a wide-ranging pipeline whose first program has reached late-stage development this past week.

Insilico said it expects to finish the first half of 2026 in the black, with “net profit” or net income ranging from approximately $33.5 million to $39.5 million, compared with its $19.2 million net loss in January–June 2025. Insilico also released adjusted non-International Financial Reporting Standards (IFRS) net profit forecasts in the range of approximately $45.5 million to $51.5 million for H1 2026. Non-IFRS metrics exclude one-time costs, such as restructuring charges and asset sales.

The company is additionally forecasting record first-half revenue ranging from approximately $102.5 million to $106.5 million, up approximately 272.7% to 287.3% from a year ago.

By contrast, Insilico finished all of 2025 with $56.239 million in revenue, down 34% from $85.834 million a year earlier, as a 69% slide in pipeline development revenue (to $23.885 million) outpaced the company’s nearly eight-fold increase in drug discovery revenue, to $24.952 million.

Notably, Insilico last year incurred a $352.5 million net loss, more than 20 times the company’s $17.1 million net loss in 2024, a jump the company attributed to the revenue drop as well as a $296.7 million loss from changes in the fair value of financial liabilities at fair value through profit or loss. That loss stemmed from Insilico converting the preferred shares issued in previous financings into ordinary shares when the company went public in December, raising HKD 2.277 billion (about $292.3 million at the time; now worth $283.9 million) on the Hong Kong Exchange.

Yet just as notably, Insilico’s cash and cash equivalents more than tripled last year, to $393.338 million.

“We look forward to achieving sustained profitability,” Alex Zhavoronkov, PhD, Insilico’s founder and CEO, said in a statement.

The forecasts continued the small but noticeable rise in Insilico’s stock price since Wednesday when the company announced that its lead candidate rentosertib, a drug designed to treat idiopathic pulmonary fibrosis (IPF), has advanced to a Phase III trial, the first drug within the company’s expansive 40+ program pipeline to reach that clinical milestone.

“Full arc of our mission”

“Rentosertib is a very important program for Insilico because it represents the full arc of our mission: using AI not only to move faster, but to originate new biology, new chemistry, and new therapeutic opportunities in aging and disease,” Zhavoronkov stated.

That news sparked a mini surge that sent Insilico’s shares climbing 19% over three days. Shares rose 2.7% from HKD$36.02 ($4.49) Tuesday to HKD$37 ($4.61) Wednesday, followed by a 7.7% gain Thursday as the stock rose to HKD$39.84 ($4.97)—then a 7.5% jump Friday, with Insilico closing the week at HKD42.82 ($5.34).

At least one investment firm started coverage of Insilico’s Hong Kong-traded stock with positive commentary: Cui Cui, an equity analyst with Jefferies, initiated the firm’s coverage with a “Buy” rating and 12-month price target of HK$100 ($12.47).

Jefferies’ endorsement capped a year in which Insilico escalated its partnership activity with pharma giants and smaller biotechs, adding roughly up to $7 billion to a potential haul that could exceed $10 billion.

The largest of these collaborations is its up-to-$2.75 billion collaboration ($115 million upfront) with Eli Lilly (NYSE: LLY), under which Insilico granted Lilly an exclusive global license to develop, manufacture, and commercialize “potentially best-in-class, novel oral therapeutics in preclinical development for certain indications,” according to an announcement that didn’t specify the therapeutic areas where the companies plan to partner. The alliance expanded from an “over $100 million” R&D partnership inked last November, which in turn grew from a 2023 licensing agreement allowing Lilly to access Insilico’s Pharma.AI software suite.

“Combining first-mover advantage, wet-lab validation, deep medical science, and in-house clinical expertise to train and refine AI, plus LLY’s endorsement, Insilico looks well positioned for scalable BD [business development] and LT [long-term] monetization,” Cui wrote in a research note.

“As AI-driven productivity cont[inues] to scale PCC [preclinical candidate] output, Insilico is positioned to expand its pool of proprietary assets, enhancing the likelihood of future out-licensing opp[ortunity] and strengthening LT monetization potential,” Cui added.

Zhavoronkov highlighted the research note on his LinkedIn feed, adding: “I think that in many ways the analysts know the industry and the company even better than some of the insiders. Definitely worth a read.”

Phase III plans

Cui’s comments followed Insilico announcing its Phase III plans for rentosertib (formerly ISM001-055), which is designed to treat IPF by targeting Traf2- and NCK-interacting kinase (TNIK), a serine/threonine kinase whose activation plays a crucial role in cellular processes that include signal transduction pathways essential for fibrosis development.

Insilico said its planned Phase III trial (NCT07687459) will be a randomized, double-blind, placebo-controlled, parallel-group study that is expected to enroll 320 participants across 47 centers in China. The trial’s primary endpoint will be the annual rate of decline in forced vital capacity (FVC) over 52 weeks, with a key secondary endpoint of time to first occurrence of any disease progression event.

The Phase III trial aims to assess whether rentosertib can provide clinically meaningful benefit in a larger patient population and over a longer treatment period than its two 12-week Phase IIa studies.

Rentosertib has completed a Phase IIa trial (NCT05938920) in China, published in Nature Medicine last year, and is in a separate Phase II trial (NCT05975983) in the United States. In the Chinese trial, rentosertib met its primary endpoint of safety and tolerability across all dose levels, as well as positive secondary endpoint data, namely dose-dependent FVC improvement with a mean FVC change of +98.4 mL at 12 weeks in patients dosed at 60 mg once daily, vs. -20.3 mL for placebo.

During the BIO International Convention in San Diego, Zhavoronkov hinted at the Phase III, highlighting a planned “next step” for the program “in the second half, but maybe closer to the earlier second half,” he told GEN.

At the convention, Zhavoronkov led Insilico in celebrating its latest big-money collaboration, an up-to-$2.5 billion partnership with SK Biopharmaceuticals to discover new AI-based drug candidates for disorders affecting the neuroimmune area of the central nervous system (CNS). Insilico agreed to apply its Pharma.AI platform, which addresses target validation, generative chemistry, and molecule optimization, along with its preclinical drug discovery expertise, to discover, design, and optimize candidates for neuroimmune indications against targets that will originate with SK.

SK is part of a privately held, family-owned chaebol or conglomerate whose parent holding company is public, SK Inc. (Korea Exchange: 034730). Another SK-owned company—SK Hynix (Nasdaq: SKHY), a supplier of high-bandwidth memory chips that power the AI processors of Nvidia and AMD—went public Friday, raising a staggering $26.5 billion by pricing its U.S. American depositary shares (ADS) at $149 each.

Potentially lucrative partnerships

In addition to SK and Lilly, Insilico also has potentially lucrative partnerships with Sanofi (Euronext Paris: SAN), with which Insilico plans to advance up to six targets (up to $1.2 billion); privately held Menarini Group, to which it has outlicensed Phase I cancer treatments targeting KAT6 and KIF18A (up to $1.05 billion in collaborations launched 2024 and 2025); privately held, French-based Servier, also cancer focused (up to $888 million); and Takeda Pharmaceutical (Tokyo Stock Exchange: 4502), drug discovery across its therapeutic areas (up to $600 million).

Also among Insilico’s collaboration partners: Exelixis (Nasdaq: EXEL), to which Insilico outlicensed in 2023 a Phase I BRCA-mutated cancer drug targeting USP1 (“close to” $1 billion plus royalties), Fosun Pharma (Shanghai Stock Exchange: 600196; Hong Kong Exchange: 02196), which is joining Insilico on R&D for four biological targets plus co-development of Insilico’s QPCTL program (up to $82 million, including $13 million upfront and a $15 million equity investment); Fosun-backed but privately held Hygtia Therapeutics, which is co-developing with Insilico ISM8969, a Phase I oral brain penetrant NLRP3 inhibitor, in CNS disorders (up to $66 million, including $10 million upfront and milestones); and Taipei-based TaiGen Biotechnology (Taipei Exchange: TWD), which holds Greater China rights to an oral PHD1/2 inhibitor in anemia of chronic kidney disease (milestones and royalties totaling “two-digit million dollars”).

Rounding out the list of Insilico’s disclosed collaboration partners are Chinese-based Qilu Pharmaceutical Group, which is partnering to jointly develop small molecule inhibitors for specific targets in cardiometabolic disease management (up to “near” $120 million, including milestones and single-digit royalties); China Medical System Holdings (CMS; Hong Kong Exchange: 867 and Singapore Exchange: 8A8), which is teaming up with Insilico on discovering drugs for central nervous system and autoimmune diseases (up to “tens of millions in Hong Kong dollars per project in R&D support”); and Tenacia Biotechnology, a Bain Capital-backed, privately held Sanghai-based drug developer which in March joined Insilico to expand a year-old R&D collaboration aimed at developing therapies for “underserved” neurological disorders (up to $94.75 million in near-term and milestone payments).

Insilico has out-licensed to undisclosed partners rights to a GLP-1R-targeting program designed to treat obesity and metabolic diseases; and Greater China rights to a Nav1.8-targeting program designed to treat pain.

Vaxart takes a double dose of good news

Settlement ends threat of proxy war; COVID-19 pill aces Phase IIb trial

This week’s annual shareholder meeting had threatened to be anything but routine for Vaxart (Nasdaq: VXRT) after its current executive team and three of its six nominees for board seats had been challenged by an activist shareholder through a proxy campaign.

Since last fall, shareholder Daniel P. Houle and allies have offered persistent criticism of Vaxart’s management—led by CEO Steven Lo and Sean Tucker, PhD, senior vice president and CSO—and the company’s board, whose operations and independent oversight are led by a lead independent director, W. Mark Watson, rather than a traditional chair.

But earlier this month, the threat of a proxy war over Vaxart’s direction ended when Houle and five allies signed a cooperation agreement with the company. Vaxart agreed to begin a search for an additional independent director to be conducted within 90 days of the conclusion of the 2026 annual meeting. Vaxart also agreed to work with Houle and allies to identify a “mutually agreeable” candidate for appointment to the board.

In return, the stockholder group consisting of Houle and his allies—Mark Silverberg, MD; Matthew M. Wallace, MD; Patrice Raffy; Marc Eustace Pereira; and Q3 Nominees Pty Ltd.—agreed to withdraw their board nominations for Houle, Silverberg, and Wallace.

The cooperation agreement also calls for:

  • Creation of a Stockholder Engagement Committee and a Clinical and Regulatory Affairs Committee
  • A revamp or “refreshment” of board committee chairs, including the selection of new chairs for the Nominating and Governance and Compensation Committees
  • Adoption of director stock ownership and resignation policies
  • Customary standstill, voting, engagement, and other provisions

“Vaxart is approaching a series of important value-inflection milestones, and these actions enable the company to move forward with a unified focus on executing its strategy,” Watson said in a statement. “We appreciate the constructive dialogue with the stockholder group toward our shared goal of creating value and are pleased to resolve our proxy contest so we can dedicate our full resources and attention to advancing our pipeline with stockholder interests in mind.”

Houle and allies insisted they believe “deeply” in the promise of Vaxart’s oral vaccine platform and resulting commercial opportunities—but took issue with the company’s declining stock price, capital raises that they said diluted the value of existing shareholders’ stock, and with what they termed insufficient oversight by the board.

In February, Houle launched his campaign to persuade shareholders to elect himself, Silverberg, and Wallace to Vaxart’s board. Silverberg is founder and CIO of Heatjac, a manufacturer of heated medical garments. Wallace is a double board-certified dermatologist and Mohs micrographic surgeon, and managing partner of a medical specialty practice focused on dermatology, dermatologic surgery, and oncology.

“We believe Vaxart possesses a unique technology platform with the potential to reshape vaccine delivery and transform global public health. Yet despite this promise, stockholder value has remained significantly compromised,” Houle and allies, calling themselves the Concerned Vaxart Shareholders, wrote in a June 9 letter to shareholders. “Despite these strengths, stockholders have endured years of disappointing performance, declining market value, and insufficient engagement from those entrusted to represent our interests.”

They also took issue with Vaxart’s two workforce reductions last year. The first was a 10% cut after Advanced Technology International, a nonprofit R&D collaboration manager acting on behalf of the U.S. Biomedical Advanced Research and Development Authority (BARDA), issued the first of two stop-work orders on the company’s Phase IIb trial assessing its government-funded COVID-19 oral pill vaccine. The second was a 21% cut in May–June 2025 intended to lower operating costs and better align Vaxart’s resources with higher-priority clinical programs.

“This election is not about creating conflict. It is about restoring confidence,” the Concerned Vaxart Shareholders added. “It is about restoring accountability, increasing transparency, and ensuring that stockholder interests are once again placed at the center of the company’s decision-making process.

Concerned Shareholders owned 1,515,343 shares of Vaxart stock—including 15,622 owned by Houle himself—as of a May 6 regulatory filing.

In an interview at the recent Biotechnology Innovation Organization (BIO) International Convention in San Diego, Lo and Tucker defended the company-endorsed board nominees as possessing greater biotech-related experience.

Lo defended the workforce cuts: “You want to be at the right size. You want to extend your runway. And we’re very careful with shareholder money. We don’t want to exhaust our funds. The reduction in the workforce was not only to extend our cash runway, but also make sure that this company had the right people to fulfill its mission.”

“Our case is, we have a very experienced management team. We have to stay the course,” Lo added. “We are in a great situation where we have good relationships with the U.S. government, as evidenced by being one of the only companies that has survived stop-work orders. We also have good relationships with pharma, as evidenced by our deal with Dynavax.”

Following a second stop-work order issued in August 2025, Vaxart and BARDA agreed to reduce funding for the Phase IIb trial to about $345 million from up to $453 million, but maintain the study at the estimated 5,485 patients recruited by Vaxart. In November 2025, Vaxart signed an up-to-$700 million global exclusive license for the oral COVID-19 vaccine with Dynavax Technologies, with Vaxart allowed to run the trial. Dynavax was acquired by Sanofi (Euronext Paris: SAN) for $2.2 billion, in a deal completed in February.

The cooperation agreement was one of two positive announcements Vaxart shared on July 6. The other was good clinical news: positive topline data from the approximately 400-participant sentinel safety cohort of its Phase IIb trial (NCT06672055) assessing the company’s oral pill COVID-19 vaccine candidate against an undisclosed approved mRNA vaccine comparator. Among key findings:

  • No vaccine-related serious adverse events (SAEs) or sustained Grade 3 or higher AEs were reported in either the oral pill vaccine or mRNA arms of the trial.
  • The most common AEs for oral vaccine patients were malaise/fatigue (20.9%), headache (18.9%), and anorexia (10.0%). Fewer than 10% of participants experienced any other AE.
  • By contrast, the most common AEs in participants receiving the mRNA vaccine were injection site pain (60.3%), injection site tenderness (40.2%), malaise/fatigue (35.2%), myalgia/muscle pain (33.2%), and headache (28.6%). Arthralgia, chills, anorexia, nausea, diarrhea, and induration/swelling at the injection site were experienced by between 10–15% of participants. Fewer than 10% of participants experienced any other AE.
  • Thirty-three participants in Vaxart’s oral pill vaccine arm and 30 in the mRNA vaccine arm had symptomatic COVID-19. Asymptomatic COVID-19 cases were reported in 12 participants in each of the trial arms.

“These topline safety data are encouraging and are consistent with the safety profile observed to date in other studies of our oral pill vaccine constructs,” stated James Cummings, MD, Vaxart’s chief medical officer.

Vaxart shares, which trade under $1, rose 16% from 55 cents on June 25 to 64 cents on July 2, the day of the filing disclosing the cooperation agreement. Since then, shares have given back the entire gain, sliding back to 55 cents at Friday’s close.

Leaders and laggards

  • Chemomab Therapeutics (Nasdaq: CMMB) shares tumbled 29% from $2.77 to $1.97 Wednesday after the developer of therapeutics for immune-fibrotic diseases with high unmet need said it agreed to merge with precision medicine developer Scipher Medicine through an all-stock merger. The combined company plans to operate under the Scipher Medicine name and trade on Nasdaq under the ticker symbol SCIP. Upon completion of the merger, the combined company plans to focus initially on advancing nebokitug, a first-in-class clinical-stage anti-CCL24 antibody, into a Phase II trial for the treatment of rheumatoid arthritis, Chemomab said. The combined company is valued at $150 million before a concurrent $30 million private placement from a syndicate of current Scipher investors led by Northpond Ventures, with participation from Khosla Ventures, Blue Owl Healthcare Opportunities, funds managed by Neuberger, and other leading investors, and is expected to have cash runway into the second half of 2028.
  • Forte Biosciences (Nasdaq: FBRX) shares rocketed 78% from $20.38 to $36.70 Thursday after the developer of treatments for autoimmune and autoimmune-related diseases announced positive results from the FB102 double-blind placebo-controlled Phase Ib study in vitiligo. FB102 achieved a 29.6% mean Facial Vitiligo Area Scoring Index (FVASI) improvement from baseline at week 24 (p-value = 0.020). Response to FB102 was seen early, Forte said, with statistically significant improvements observed by the day 64 visit (p=0.023), continuing through week 24, after completion of the 12-week treatment period. FB102 achieved 43.2% mean FVASI improvement from baseline at week 24 (p-value = 0.006) in subjects with greater disease involvement having baseline FVASI ≥0.75 (approximately one-quarter of face depigmented), including FVASI50 (58.8%) and FVASI75 (23.5%). Forte shares continued climbing Friday, rising another 20% to $43.92.

The post StockWatch: Insilico Projects Profit, Revenue Leaps as AI-Developed Lead Candidate Moves to Phase III appeared first on GEN – Genetic Engineering and Biotechnology News.

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STAT+: AstraZeneca, Ionis report major trial failure with heart disease drug

LONDON — In a blow to its cardiovascular aspirations, AstraZeneca said Thursday that its drug for a heart disease — one that has become an increasingly competitive target for biopharma companies — failed in a pivotal trial.

The drug, called Wainua, on which AstraZeneca is partnered with Ionis Pharmaceuticals, did not outperform placebo in reducing cardiovascular death and clinical events for patients with a condition known as ATTR-CM, or transthyretin-mediated amyloid cardiomyopathy. 

AstraZeneca’s U.S. shares were down some 8% in premarket trading, while its London-listed shares were down 9% early Thursday. Ionis shares, meanwhile, were down 12% in premarket hours.

Continue to STAT+ to read the full story…