Tag: Biopharma

Posted on

Supply Chain Digital Twins: An Evolution, Not a Breakthrough

Digital twins help optimize drug production processes by modeling the thousands of interactions that cells, raw materials, and reagents undergo in culture. And new analysis suggests they could do the same thing for supply chains.

Researchers at the U.S. National Institute of Standards and Technology (NIST) and EMD Millipore put forward the idea, arguing that twins could make drug distribution, which is also characterized by thousands of interactions, more resilient and efficient.

Lead author Perawit Charoenwut, a logistics researcher at NIST’s systems integration division, tells GEN, “A digital twin could be extremely helpful in all phases of the biopharmaceutical supply chain. Starting from demand planning triggered by global events such as pandemics, regional disease outbreaks, aging demographics, etc., through to being able to provide visibility on capacity requirements and limitations.”

In silico models could also provide solutions to disruption by identifying alternative supply options, such as distribution centers or regional inventories, in less time, Charoenwut says.

“Digital twins could also be helpful in evaluating different suppliers by running simulations on their potential performance, based on different demand scenarios versus their individual capacities and capabilities,” he continues.

Standards

In theory, digital twins are a good option for supply chain modeling and management. In practice, however, firms interested in the approach will need to overcome some technical challenges.

For example, one major hurdle is the lack of data standardization, according to study co-author Boonserm Kulvatunyou, PhD, a computer engineer at NIST. “Supply chain digital twins require data from across organizations and third-party sources,” he tells GEN. “The lack of industry standards creates challenges in obtaining all the necessary data.”

With this in mind, the NIST’s Industrial Ontology Foundry (IOF) is working with the National Innovation Institute for Manufacturing Biopharmaceuticals (NIIMBL) to develop open-source ontology and schema standards for connecting data.

Kulvatunyou says, “The aim is to provide a semantic foundation for connecting data and knowledge across the manufacturing and supply chain operations.

“Further work is being conducted to cover broader materials, processes, and quality data,” he says. “We would like to invite industry and academia to join this effort and benefit from these new standards.”

Industry interest

Biopharma firms interested in digital supply chains will also need to establish a solid data infrastructure, according to Charoenwut, who says companies should start small and pace themselves.

“We think that biopharma companies do believe that digital twins could make a significant difference in their supply chain efficiency and resiliency. Many of them are probably building prototypes and proofs-of-concept to demonstrate the value and potential benefits, but then soon realize the digital data foundation gaps that need to be addressed in parallel in order to fully adopt this technology.

“As digital twins can vary in detail and complexity, companies should strategize digital twin adoption by starting with lower-complexity cases based on available digital data and progressively moving up the scale to gain greater precision and new capabilities. In other words, the implementation of digital twins should be viewed as an evolution rather than a breakthrough,” he says.

The post Supply Chain Digital Twins: An Evolution, Not a Breakthrough appeared first on GEN – Genetic Engineering and Biotechnology News.

Posted on

Milk Exosomes Transform Therapeutic Bioprocessing

Breast milk has long been understood as more than infant nutrition. It is a biologically active system packed with molecular signals that help shape immune development, metabolism, and even brain function. Among its most intriguing components are milk-derived extracellular vesicles—tiny lipid-bound particles often called milk exosomes—that are rapidly becoming one of bioprocessing’s most promising therapeutic tools.

These nanoscale carriers are naturally designed for transport. They can survive digestion, move into circulation, and distribute cargo throughout the body, with studies suggesting they may even reach the brain during early development. Researchers have shown that these vesicles can influence central nervous system communication, particularly through interactions with microglia, which are crucial to the brain’s immune cells. The ability of milk exosomes to carry microRNAs and regulate epigenetic pathways, including DNA methyltransferase 1 (DNMT1), points to a sophisticated biological delivery system that the industry is now learning to harness.

That potential is especially compelling in drug manufacturing, where delivery often determines whether a therapy succeeds or fails. Traditional nanoparticles can trigger toxicity, instability, or poor absorption. Milk exosomes offer a more elegant alternative: they are biocompatible, naturally abundant, and scalable for pharmaceutical development.

Huiming Tu, MD, a researcher and clinician in the department of gastroenterology at the Affiliated Hospital of Jiangnan University in Wuxi, China, and his colleagues recently demonstrated this with ulcerative colitis. Their team developed an oral delivery platform called mEXOs@TOF, which loads the pan-JAK inhibitor tofacitinib into milk-derived exosomes. The resulting formulation showed strong pharmaceutical performance, including consistent particle size, high drug-loading efficiency, and strong stability during delivery.

More importantly, the therapy improved anti-inflammatory outcomes through multiple mechanisms. It lowered inflammatory mediators such as IL-6, IFN-γ, and nitric oxide, while increasing anti-inflammatory IL-10. It also reduced oxidative stress and suppressed activation of the JAK-STAT3 signaling pathway. In both laboratory and animal studies, the system delivered strong therapeutic benefits without detectable toxicity—an ideal benchmark for translational bioprocessing.

Cancer therapy is seeing similar innovation. Min Suk Shim, PhD, professor of nano-bioengineering at Incheon National University in the Republic of Korea, and colleagues focused on sonodynamic therapy, in which ultrasound activates a sensitizing drug to destroy tumors. Their challenge was improving intracellular delivery of chlorin e6 (Ce6), a common sonosensitizer.

The team engineered glutathione-responsive milk exosomes by incorporating a diselenide bond-bearing fatty amine derivative. This allowed the vesicles to remain stable during circulation but release Ce6 inside breast cancer cells, where glutathione concentrations are higher. Once ultrasound was applied, reactive oxygen species production increased dramatically, leading to significant cancer cell death in MCF-7 breast cancer models. The work shows how responsive bioprocess design can turn natural vesicles into precision-triggered therapeutics.

Meanwhile, scientists from Hong Kong and China have reviewed the broader landscape of milk exosomes in breast cancer treatment. Beyond acting as delivery vehicles for drugs like doxorubicin, paclitaxel, and 5-fluorouracil, milk exosomes may also have direct anti-tumor effects. They can promote apoptosis, interrupt the cell cycle, and regulate pathways such as NF-κB and STAT3. Combined with plant-derived compounds like curcumin and resveratrol, they form hybrid nanoparticles with enhanced therapeutic power.

For bioprocessing, the message is clear: milk exosomes are no longer a niche curiosity. They represent a scalable, safe, and highly adaptable platform for next-generation therapeutics—one that begins with biology’s oldest delivery system and may define medicine’s next one.

The post Milk Exosomes Transform Therapeutic Bioprocessing appeared first on GEN – Genetic Engineering and Biotechnology News.

Posted on

STAT+: A patent win for Pfizer and BridgeBio

Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.

Good morning. Several biotech leaders were named in this list of the most influential Bostonians. Is there anyone you think should have made the list who didn’t? Let me know your thoughts.

The need-to-know this morning

  • The Italian pharma company Chiesi is buying KalVista Therapeutics for $1.9 billion, adding an approved treatment for a genetic swelling disorder to its rare-disease portfolio. The deal values KalVista at $27 per share, or a 40% premium to its Tuesday closing stock price. The KalVista medicine, called Ekterly, is a pill used to treat acute swelling attacks in people with hereditary angioedema.
  • Biogen, AstraZeneca, Regeneron Pharma, Abbvie and GSK reported first-quarter earnings.

FDA plans to review AZ, Amgen trials in real time

In an effort to get drugs on the market faster, the FDA announced yesterday it will start reviewing trial data in real time, starting with oncology studies conducted by AstraZeneca and Amgen.

Continue to STAT+ to read the full story…

Posted on

STAT+: Pharmalittle: We’re reading about the FDA speeding up trials, a Supreme Court hearing on ‘skinny labels,’ and more

Top of the morning to you. The middle of the week is upon us and, since you made it this far, why not forge ahead? After all, there is always light at the end of the proverbial tunnel. You never know what you may accomplish. So please join us as we celebrate this notion with a cup or three of delicious stimulation. Our choice today is chocolate raspberry. Meanwhile, we have assembled the latest menu of tidbits to help you along. So please dig in. Have a smashing day, and please feel free to forward any secrets you come across. Our “in basket” is always open. …

The U.S. Food and Drug Administration announced efforts to make clinical trials more efficient, starting by reviewing data in real time from trials conducted by AstraZeneca and Amgen, STAT writes. The agency also asked the public to weigh in on a potential pilot program to work with companies that use AI to enhance safety monitoring and medication dose selections, identify safety signals, and improve patient recruitment in clinical trials. The trials will rely on a real-time data platform built by Paradigm Health, and the goal is to cut down on the time regulators and companies spend sending data back and forth. FDA Commissioner Marty Makary said at a press conference that agency reviewers will be able to view safety signals and clinical endpoints via Paradigm’s platform.  

Pfizer settled ‌patent disputes with three generic drugmakers over its blockbuster heart drug Vyndamax, effectively extending its patent protection until 2031 and delaying cheaper ​copies from entering the market, Reuters says. The deals resolve ​patent infringement lawsuits against Dexcel Pharma, Hikma Pharmaceuticals, ⁠and Cipla in Delaware federal court over Pfizer’s ​oral drug Vyndamax. A trial over the patent had started ​this week. Pfizer sold nearly $6.4 billion of Vyndamax and related drugs, which treat a serious heart condition called transthyretin amyloid cardiomyopathy, in 2025. The settlements extend ​U.S. patent protection for Vyndamax until June 1, ​2031, subject to other pending litigation. The company had previously expected ‌a ⁠sharp drop in U.S. revenue for the drug in 2029 but now expects sales to hold relatively steady from 2028 through mid-2031.

Continue to STAT+ to read the full story…

Posted on

Pencil Beam Laser Could Help Researchers Design Brain-Targeted Therapies

Scientists at MIT say they made a finding in optical physics that could enable a new bioimaging method that’s faster and higher-resolution than existing technology. They discovered that, under the right conditions, laser light clutter can spontaneously self-organize into a highly focused “pencil beam.”

Using this self-organized pencil beam, the team captured 3D images of the human blood-brain barrier 25 times faster than the gold-standard method, while maintaining comparable resolution, according to the scientists.

By showing individual cells absorbing drugs in real-time, this technology could help scientists test whether new drugs for neurodegenerative disease like Alzheimer’s or ALS reach their targets in the brain, with greater speed and resolution, they add.

“The common belief in the field is that if you crank up the power in this type of laser, the light will inevitably become chaotic. But we proved that this is not the case. We followed the evidence, embraced the uncertainty, and found a way to let the light organize itself into a novel solution for bioimaging,” says Sixian You, PhD, assistant professor in the MIT department of electrical engineering and computer science (EECS), a member of the research laboratory for electronics.

You is senior author of a paper “Self-localized ultrafast pencil beam for volumetric multiphoton imaging” on this imaging technique in Nature Medicine.

A better beam

When the researchers performed characterization experiments of this pencil beam, it was more stable and high-resolution than many similar beams. Other beams often suffer from “sidelobes,”  blurry halos of light that can distort images.

Their beam was more pristine and tightly focused, according to You. Building on those experiments, the researchers demonstrated the use of this pencil-beam in biomedical imaging of the human blood-brain barrier.

Scientists and clinicians often want to see how drugs flow inside the vasculature of the blood-brain barrier and whether they reach their targets within the brain. But with standard optical settings, the best one can do is capture one 2D section of the vasculature at a time, and then repeat the process multiple times to generate a fuller image, You explains.

Using this new technique, the researchers created an ultrafast, high-precision pencil beam that enabled them to dynamically track how cells absorb proteins in real-time.

“The pharmaceutical industry is especially interested in using human-based models to screen for drugs that effectively cross the barrier, as animal models often fail to predict what happens in humans. That this new method doesn’t require the cells to have a fluorescent tag is a game-changer,” notes Roger Kamm, PhD, the Cecil and Ida Green Distinguished Professor of Biological Science and Mechanical Engineering.

“For the first time, we can now visualize the time-dependent entry of drugs into the brain and even identify the rate at which specific cell types internalize the drug.”

“Importantly, however, this approach is not limited to the blood-brain barrier but enables time-resolved tracking of diverse compounds and molecular targets across engineered tissue models, providing a powerful tool for biological engineering,” points out postdoctoral fellow Sarah Spitz, PhD.

The team reports that it captured cellular-level 3D images that were higher quality than with other methods, and generated these images about 25 times faster.

“Usually, you have a tradeoff between image resolution and depth of focus—you can only probe so far at a time. But with our method, we can overcome this tradeoff by creating a pencil-beam with both high resolution and a large depth of focus,” You says.

In the future, the researchers want to better understand the fundamental physics of the pencil-beam and the mechanisms behind its self-organization. They also plan to apply the technique to other scenarios, such as imaging neurons in the brain, and work toward commercializing the technology.

The post Pencil Beam Laser Could Help Researchers Design Brain-Targeted Therapies appeared first on GEN – Genetic Engineering and Biotechnology News.

Posted on

Sun Pharma Aims for Top 3 in Women’s Health with $11.75B Organon Purchase

Sun Pharmaceutical Industries has agreed to acquire Organon, the women’s health drug developer spun out of Merck & Co., for $11.75 billion in a deal intended to catapult the buyer into a top 25 global biopharma—top three in women’s health—by growing its innovative medicines business and expanding its product offerings into biosimilar drugs, the companies said today.

Headquartered in Jersey City, NJ, Organon was spun out of Merck in 2021 and has since then grown its portfolio to more than 70 women’s health and general medicines products, including biosimilars, that have been commercialized in the U.S. and some 140 countries worldwide. In addition to the U.S., Organon’s largest markets include Brazil, Canada, China, and the countries of the European Union. Organon said it has six manufacturing facilities across the EU and emerging markets.

Sun Pharma said the combined company created by the deal will have annual revenue of $12.4 billion, a figure the company said would propel it into a top 25 global pharma—though the company was ranked No. 14 in GEN’s most recent A-List of Top 25 Biotech Companies Heading Into 2026, compiled last December, based on its market capitalization (share price times the number of outstanding shares) of INR 4.31 trillion ($50.8 billion).

Sun Pharma said Organon’s portfolio was similar to its own, and that the acquisition of Organon was aligned with its strategies of growing its Innovative Medicines business (to a 27% revenue share) and expanding into biosimilars as a Top 10 global company.

The combined company, Sun Pharma and Organon said, would be top three in global women’s health, creating a commercial platform for future growth; the seventh largest global biosimilar player; and a presence in 150 countries worldwide, with 18 large markets that would each generate more than $100 million in revenues.

“This transaction represents a significant opportunity for Sun Pharma to build on its vision of Reaching People and Touching Lives,” Sun Pharma executive chairman Dilip Shanghvi said in a statement. “Organon’s portfolio, capabilities, and global reach are highly complementary to our own, and we believe that bringing the two organizations together can create a stronger and more diversified platform. We have deep respect for Organon’s mission and look forward to building on its legacy while driving sustainable long‑term growth.”

Deal speculation

The deal ends two weeks of speculation that began with an April 10 report in the Indian news outlet The Economic Times stating that Sun Pharma had submitted a $12 billion all-cash offer for Organon. On Friday, the news outlet followed up with a report stating that Sun Pharma had submitted a revised $13 billion offer.

Investors appeared to support the deal, as Sun Pharma shares on India’s National Stock Exchange rose about 7% to INR 1,733.50 ($18.41) at the close of trading today.

Sun Pharma has agreed to acquire 100% of Organon’s issued and outstanding shares for cash. Sun said it planned to fund the acquisition through a combination of available cash resources and committed financing from banks.

“Together, we will become a partner of choice for acquiring and launching new products,” stated Kirti Ganorkar, managing director of Sun Pharma. “Our immediate priorities will be business continuity, disciplined integration, and responsible value creation. We see strong potential in leveraging Organon’s talent pool. In addition, there is a scope for synergies including significant revenue upside opportunities to be realized over the coming years.”

Those synergies were later quantified by Sun Pharma as approximately $350 million within two to four years of the deal’s completion.

Sun Pharma did say, however, that the acquisition of Organon will strengthen its generation of cash, with its earnings before interest, taxes, depreciation, and amortization (EBITDA) and cash flow set to nearly double, supporting future efforts to reduce the net debt/EBITDA of 2.3x resulting from the deal.

Sun Pharma finished the first nine months of its fiscal year ending March 31, 2026, with a net profit of INR 87.654 billion ($931.5 million) and EBITDA of INR 137.772 billion ($1.464 billion; up 19.2% from the year-ago period), on sales of INR 436.604 billion ($4.64 billion), up 11.3% year over year.

During its fiscal year ending March 31, 2025, Sun Pharma reported adjusted net profit (excluding one-time items) of INR 119.844 billion ($1.274 billion), up 19% from a year earlier, on sales of INR 520.412 billion (about $5.53 billion). Reported net profit for FY 2025 was INR 109.290 billion ($1.161 billion), vs. Rs. 95.764 billion ($1.017 billion) during FY 2024.

Organon finished last year with adjusted EBITDA of $1.9 billion on revenue of $6.2 billion. The company reported debt of $8.64 billion—down from the $9.5 billion in debt it reported when it separated from Merck—and a cash balance of $574 million.

Planned sale

In November, Organon announced plans to sell its JADA® System, designed to control and treat abnormal postpartum uterine bleeding or hemorrhage, to Laborie Medical Technologies for up to $465 million—$440 million to be paid at closing, subject to adjustments, and up to $25 million tied to achieving 2026 revenue targets. Net proceeds from the divestiture will contribute to Organon’s cash balance as of March 31, 2026.

Organon will merge with a subsidiary of Sun Pharma, with Organon surviving the merger. The transaction is expected to close in early 2027 subject to customary conditions, including regulatory approvals and Organon stockholder approval.

The boards of both Sun Pharma and Organon have approved the deal.

“Following a comprehensive review of strategic alternatives, our Board determined that this all‑cash transaction offers compelling and immediate value to Organon stockholders,” stated Carrie Cox, executive chair of Organon. “We believe Sun Pharma is well positioned to support Organon’s businesses, employees, and patients globally, and to further advance our commitment to delivering impactful medicines and solutions.”

The post Sun Pharma Aims for Top 3 in Women’s Health with $11.75B Organon Purchase appeared first on GEN – Genetic Engineering and Biotechnology News.

Posted on
<![CDATA[DT120, a pharmaceutical-grade formulation of LSD, shows rapid, lasting anxiety relief—single dose, no therapy—with 48% remission at 12 weeks.]]>
Posted on

STAT+: Intellia says CRISPR-based treatment for rare disease reduced swelling attacks in pivotal trial

Intellia Therapeutics said Monday that a single dose of its gene editing treatment dramatically reduced swelling attacks in patients with a rare genetic disorder in a Phase 3 trial, setting up a potential approval.

The therapy, known as lonvo-z, would be the second approved CRISPR-based medicine, after Vertex Pharmaceutical’s sickle cell treatment Casgevy. Intellia has already initiated a rolling submission with the agency. It would be the first in vivo treatment, meaning it edits patients’ DNA directly in the body. 

In the 80-patient study, volunteers with hereditary angioedema (HAE) who received lonvo-z saw their attack rates drop 87%, relative to placebo. Just over 60% were entirely attack-free over the period, compared to 11% for the placebo patients.

Continue to STAT+ to read the full story…

Posted on

StockWatch: Trump Order Lifts Psychedelic Drug Shares

Stocks of most publicly traded psychedelic drug developers jumped when President Donald 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.

Among its provisions, the order directs the FDA to provide Commissioner’s National Priority Vouchers (CNPVs) to “appropriate” psychedelic drugs that were granted the agency’s Breakthrough Therapy designation and met the voucher program’s criteria. The FDA’s parent agency, the Department of Health and Human Services (HHS), is required to spend at least $50 million through the Advanced Research Projects Agency for Health (ARPA-H) “to support and partner with” state governments that have enacted or are developing programs to advance psychedelic drugs for serious mental illnesses.

“This is an unmet public health need and potentially promising treatments. That’s why there’s a sense of urgency around this, and why we’re doing it now,” FDA Commissioner Martin A. Makary, MD, said at the ceremony where Trump signed the order. “Applications are about to come in, and this is the perfect timing for this announcement.”

At least one analyst agreed that the timing was right for Washington to spur the development of psychedelic drugs.

“Investor mindshare should rise meaningfully ahead of pot’l approvals in 2027–30,” Andrew Tsai, equity analyst with Jefferies, observed in a research note. “As we approach the first pot’l FDA approval of a psychedelic in 2027, President Trump is providing an official stamp of validation to the class in the form of an executive order, reassuring us that the FDA/HHS/White House’s support of psychedelics is real/actionable (not rhetorical).”

Proving correct

Makary said the FDA planned to issue CNPVs to three serotonin 2a agonists, a class that includes LSD and other psychedelic drugs. While he did not reveal specific companies and drugs by name, market watchers immediately speculated that one of the drugs was COMP360 synthetic psilocybin, the lead clinical candidate of Compass Pathways (NASDAQ: CMPS)—speculation that proved correct when COMP360 won a CNPV on Friday.

COMP360 is expected, according to Tsai, to be the first psychedelic drug to win FDA approval in 2027. In February, Compass announced what it called statistically significant and clinically meaningful data from two Phase III trials assessing COMP360 in treatment-resistant depression (TRD), COMP005 (NCT05624268) and COMP006 (NCT05711940). The data showed positive effects for COMP360 within one day, lasting at least through six months after just one or two doses among those who have a clinically meaningful response.

COMP360 is also in Phase II trials for both PTSD and anorexia nervosa.

Compass fueled speculation about an FDA voucher approval by issuing a statement supporting the executive order: “Today’s announcement aligns regulatory urgency with patient need, and we applaud the Administration for taking this important step forward in accelerating access, without compromising rigorous science.”

Investors celebrated with Compass, whose shares soared 42% from $6.66 to $9.46 on April 20, the first trading day after the order signing. Shares yo-yoed the rest of the week, sliding 7.5% to $8.75 Wednesday before rebounding nearly 5% to $9.15 Thursday and rising another roughly 5% to $9.58 Friday on news of the voucher approval. Year-over-year, Compass shares have more than doubled, soaring about 140% from $5.22 on April 24, 2025.

FDA names additional voucher grantees

The FDA indeed issued three CNPVs on Friday—one to Compass as previously mentioned, one to Usona Institute, a nonprofit medical research organization, for psilocybin for major depressive disorder (MDD), and one to Otsuka Pharmaceutical (Tokyo Stock Exchange: 4578) for methylone (TSND-201) for post-traumatic stress disorder (PTSD). Otsuka is acquiring the methylone program as part of its up-to-$1.225 billion ($700 million upfront) purchase of privately held Transcend Therapeutics, announced last month.

Launched in October by Makary, CNPVs are awarded to drug developers whose work is deemed to address a health crisis in the United States, deliver more innovative cures, address unmet public health needs, and increase domestic drug manufacturing as a national security issue. The vouchers entitle companies to reviews of their final applications within a target timeframe of 1–2 months, rather than the current 10–12 months.

“Ultimately, we do not see the FDA’s issuance of the first set of CNPVs as precluding other psychedelic players from also obtaining CNPVs in the future—so we think the FDA’s action today bodes well for the space broadly,” Tsai wrote after the FDA announced the voucher recipients. “Net-net, the macro backdrop for psychedelics is improving.”

That improvement, Tsai added, reflects Trump’s endorsement of psychedelic drugs, a collaborative FDA, and growing interest in the space by big pharma giants such as Johnson & Johnson (NYSE: JNJ), which generated $1.696 billion in 2025 sales and $468 million in first quarter sales from Spravato (esketamine), an NMDA receptor antagonist indicated for treatment-resistent depression (TRD) and depressive symptoms in adults with MDD with acute suicidal ideation or behavior in conjunction with an oral antidepressant.

The voucher decision hardly budged Otsuka shares, which dipped nearly 1% Friday from ¥10,870 ($68.18) to ¥10,810 ($67.80).

However, Compass was one of several psychedelic drug companies to see their shares surge on news of the executive order.

AtaiBeckley (NASDAQ: ATAI), formed last November by the merger of atai Life Sciences and Beckley Psytech, jumped 22% from $4.03 to $4.90 on April 20, then plateaued the rest of the week, finishing Friday at $4.63 and a 15% one-week gain. AtaiBeckley shares year-over-year have more than tripled, rocketing 204% from $1.53 a year ago Friday.

Definium Therapeutics (NASDAQ: DFTX) shares rose 5% over two days, from $22.68 the Friday before Trump signed the order to $23.84 on Tuesday, but gave back all the week’s gain, finishing Friday at $22.48. Long-range investors have fared better, as Definium shares have more than tripled, zooming 249% from $6.43 on April 24, 2025.

GH Research (NASDAQ: GHRS) shares climbed 17% from $18.34 to $21.50 the first day after the executive order, only to drop 6% the rest of the week, closing Friday at $20.25 and settling for a 10% one-week gain. GH’s shares doubled year-over-year, growing 101% from $9.50 a year ago Friday.

Showing volatility

The executive order wasn’t enough to boost shares of Cybin, which operates under the name Helus Pharma (NASDAQ: HELP). Helus showed the most volatility in the days following the order signing, tumbling 12% from $5.61 to $4.93 on April 20. The drop followed Helus’ announcement that its CEO, Michael Cola, stepped down immediately at the request of its board, succeeded by interim CEO Eric So, while the board carries out a search for a permanent chief executive.

“It’s like they can’t give investors a break,” fumed “SamZaki320” on a Reddit chat board. “The only positive is that the executive order sentiment is pushing back against a total disaster. Not that it’s a good thing, other companies are up double digits.”

Helus shares bounced back 17% to $5.77 Wednesday and dipping 0.35% to $5.75 Thursday despite the company announcing two powerhouse additions to its scientific advisory board—Robert Langer, ScD, the David H. Koch Institute professor at MIT and a co-founder of Moderna (NASDAQ: MRNA); and Stephen Brannan, MD, a neuroscience drug development expert with over 20 years of experience designing and implementing clinical programs for psychiatric and neurological disorders. Shares fell 2% Friday, closing at $5.61.

In a statement, interim CEO So lauded Trump’s order: “The Executive Order reflects growing recognition of the urgent need for new treatment options in serious mental health conditions and the importance of advancing innovative therapies through rigorous, research-based development.”

Looking beyond Washington

Yet So acknowledged that Washington alone can’t advance psych drug development beyond what its science can accomplish: “Policy momentum is meaningful, but the future of this field will ultimately be determined by the strength of the clinical evidence and the ability to deliver safe, reliable treatments at scale.”

As did Helus and Compass, Definium also praised the executive order: “We applaud the Administration’s recognition that psychedelic medicines may represent meaningful new treatment options for patients,” Definium CEO Rob Barrow stated. He cited his company’s clinical development program for DT120 (lysergide tartrate) for conditions that include generalized anxiety disorder (GAD) and MDD.

At the ceremony where he signed the executive order, Trump acknowledged being asked to address psych drug development by podcaster Joe Rogan and others, which the president said led to talks with Makary as well as HHS Secretary Robert F. Kennedy Jr., NIH Director Jay Bhattacharya, MD, PhD, and Mehmet Oz, MD, administrator for the Centers for Medicare & Medicaid Services.

“Research has been going on for quite some time. But usually with things like this, nothing ever happens, no matter how the research ends up. We’re changing that,” Trump said. “Why would we wait three or four years to get it done? Or 10 years? Frankly, let’s get it done immediately—and that’s what happened.”

Leaders and laggards

  • Daiichi Sankyo (Tokyo Stock Exchange: 4568) shares slipped 10% from ¥2,790 ($17.50) to ¥2,499 ($15.67) on Friday after the drug developer announced it was delaying the release of its annual earnings results for the fiscal year that ended March 31, from April 27 to May 11,  “as additional time is required to finalize the financial figures.” May 11 is the day when Daiichi Sankyo plans to release its five-year business plan. “The company is currently reviewing the supply plans for its oncology products portfolio and development pipeline in light of rapidly changing business conditions. As a result, additional deliberation is required to reasonably estimate the amount of loss provisions to be recorded in connection with contracts with contract manufacturers,” Daiichi Sankyo added in a statement.
  • Inhibrx Biosciences (NASDAQ: INBX) shares leaped 37% from $84.08 to $115.09 Wednesday after Reuters reported, citing unnamed sources, that Merck & Co. (NYSE: MRK), Merck KGaA (XETRA: MRK), and Ono Pharmaceutical (Tokyo Stock Exchange: 4528) were in talks with Inhibrx for a joint spinoff of two precision-engineered cancer candidates, INBRX-106 and ozekibart (INBRX-109). The treatments could have a combined value of more than $9 billion if their clinical trials prove successful, the report stated. Inhibrx declined to comment, while the other companies cited did not respond to Reuters queries. INBRX-106 is a hexavalent sdAb-based, OX40-targeting candidate being studied as monotherapy and in combination with Merck & Co.’s cancer immunotherapy blockbuster Keytruda® (pembrolizumab). Ozekibart is a tetravalent death receptor 5 (DR5) agonist antibody designed to exploit the tumor-biased cell death induced by DR5 activation. On Tuesday, Inhibrx announced ozekibart showed positive data in a Phase I/II trial (NCT03715933) assessing the drug plus Folfiri in patients with locally advanced or metastatic, unresectable colorectal cancer.
  • Organon (NYSE: OGN) shares surged 31% from $8.60 to $11.26 Friday after the Indian news outlet The Economic Times reported that Sun Pharmaceutical Industries (NSE: SUNPHARMA and BSE: 524715) had submitted a $13 billion offer for the women’s health drug developer spun out of Merck & Co. (NYSE: MRK) in 2021. The deal would be Sun’s largest ever merger-and-acquisition (M&A) deal—if Sun can prevail over at least two other would-be suitors for Organon, German-based private family-owned drug developer Grünenthal, and EQT (Nasdaq Stockholm: EQT), a Swedish-based global investment organization. The latest surge comes two weeks after Organo shares zoomed 28% on an April 10 Economic Times report stating that Sun Pharma had submitted a $12 billion all-cash offer for Organon.
  • Spruce Biosciences (NASDAQ: SPRB) shares tumbled 26% from $69.89 to $51.69 Tuesday after the neurological disorder drug developer priced a $69 million offering of common stock and pre-funded warrants that generated $64.4 million in net proceeds. The offering consisted of 1.15 million shares of common stock priced at $50 per share and pre-funded warrants to purchase 50,000 shares at $49.99 per share. Al shares and pre-funded warrants were sold, and underwriters of the offering exercised in full their option to purchase up to an additional 180,000 shares at the public offering price on Tuesday. “We intend to use the net proceeds from this offering to advance the company’s pre-commercial and launch activities, for planned clinical trials, and for working capital, capital expenditures, and other general corporate purposes,” Spruce stated in its prospectus supplement filed Tuesday. Leerink Partners, Guggenheim Securities, and Oppenheimer & Co. acted as joint book-running managers, while Jones and Craig-Hallum acted as co-managers for the offering.

The post StockWatch: Trump Order Lifts Psychedelic Drug Shares appeared first on GEN – Genetic Engineering and Biotechnology News.

Posted on

STAT+: A biotech VC on what Eli Lilly saw in a struggling cancer startup for $3.2B

Kelonia Therapeutics became the newest biotech takeout target this week. The privately held company, which is developing cell therapies for cancer and autoimmune diseases, will be acquired by Eli Lilly. 

The acquisition is a boon for the small startup, which has subsisted on $60 million over the last five years and previously struggled to stay afloat. (Check out an earlier slide deck and memo on the company here.) Kelonia came within a week of running out of cash three times. Now it’s being bought for $3.2 billion with potential milestone payments that could double that payout.

On this week’s edition of its biotech podcast, “The Readout Loud,” STAT spoke with Bryan Roberts, a partner at VC firm Venrock, which incubated the biotech, to discuss how this small company managed to land a big deal. 

Continue to STAT+ to read the full story…