Medical Innovation Exchange


No ongoing sale process at Cytokinetics, CEO says following Novartis acquisition rumors

After being the center of speculation for several months, Cytokinetics’ CEO Robert Blum is putting the kibosh on the rumor that Novartis will be acquiring the biotech.
“Since sharing the positive results from SEQUOIA-HCM, Cytokinetics has been rumored to be an acquisition target,” Blum said on a Feb. 27 fourth quarter earnings call, referring to a late-December phase 3 win for the company’s cardiomyopathy treatment aficamten. “While we will not and cannot comment on specific speculations, let me please be clear about one thing—we did not initiate nor do we have a sale process ongoing.”

The cardiomyopathy readout was more than just a clinical win—it established Cytokinetics as a company with a near-approval asset in a blockbuster indication and sent the biotech’s stock skyrocketing.

After the SEQUOIA-HCM results were shared, whispers of a Novartis acquisition grew louder and louder. Then, in mid-January, Reuters and The Wall Street Journal took the air out of those claims, reporting that Novartis had dropped its pursuit of Cytokinetics. 
When asked about the acquisition allegations, a Novartis spokesperson told Fierce Biotech in January: “We don’t comment on market rumor or speculation.”

Now, Blum has laid the final nail in the coffin for the possibility of a Novartis buyout. However, the CEO reassured shareholders that the company will consider all options presented, hinting at other deal possibilities on the table.
In preparation for a potential approval and a global commercial launch for aficamten, Blum said the company intends to “monetize our R&D progress and preserve shareholder value via partnering.”
“Our priority remains focused on business development,” the Cytokinetics CEO said, adding that the company is looking to execute a “Japan deal for aficamten.”
“We’re in active discussions with multiple parties and I’m pleased with how that deal campaign is looking,” Blum said.

With cash runways ending this year, Nicox and Longeveron trim staff and narrow focus

Layoffs are in store for employees at both Longeveron and Nicox, although neither company is spelling out how many staff will be affected.
Regenerative medicine biotech Longeveron has made the “strategic decision” to discontinue a phase 2 trial of its lead candidate Lomecel-B in aging-related frailty in Japan. The move would result in “related staff reductions” that the company listed as part of “cost-saving measures.”

“Longeveron management continues to believe in the potential of Lomecel-B in this disease state and will evaluate options for continued development at a future date,” the company insisted in a full-year earnings report yesterday.

The biotech is also hunting for “potential partnerships or other sources of funding” to justify continuing to investigate the drug in Alzheimer’s disease. As recently as December, Longeveron was touting data from a successful phase 2 trial of Lomecel-B in Alzheimer’s, which it claimed “improved cognitive function in multiple measures in a dose-response fashion.”
Lomecel-B is made up of medicinal signaling cells from the bone marrow of adult donors that are culture expanded at a cell processing facility. Yesterday’s strategic shake-up means the biotech is now focusing resources on completing enrollment in a phase 2 trial of the living cell therapy in hypoplastic left heart syndrome (HLHS), which CEO Wa’el Hashad described as “our most important near-term value driver.”

A quick glance at the company’s finances makes clear why Longeveron is narrowing its focus so significantly. The biotech entered 2024 with $5.4 million in cash and equivalents, which was expected to run out by July.
“We are actively seeking financing opportunities to extend our cash runway while taking measures to reduce our cash expenditures as we focus our resources on our primary strategic program in HLHS,” the company said in yesterday’s release.
Tight finances are also behind Nicox’s plans to reduce operation costs. The ophthalmology biotech has agreed to an amendment to the repayment of a 16.9 million euros ($14.5 million) debt with Kreos Capital, which will extend the company’s cash runway to November.
But, to secure this deal, Nicox had to agree to “reduce its operations in France and Italy” as well as cut operating costs and restructure the company so it’s focused on completing the latest phase 3 Denali trial for glaucoma drug NCX 470.

“The additional cash runway and the overall reduction in cash needs gives us the flexibility to advance our core asset and continue partnering and strategic discussions,” Jean-François Labbé, chairman of Nicox’s board of directors, explained in the Feb. 28 release.
This means that an undisclosed number of staff tied to Nicox’s home country of France as well as working at its Italian subsidiary will be laid off. “The development team in the U.S., considered essential for the completion of the Denali trial, is not impacted by these changes,” the biotech added.
In connection with the downsizing, three members of Nicox’s board will also be departing: Adrienne Graves, Ph.D., Lauren Silvernail and Luzi von Bidder. In a separate announcement on the same day, the biotech revealed that Chief Business Officer Gavin Spencer had been promoted to CEO.
Fierce Biotech has contacted both Longeveron and Nicox for more details about how many staff will be affected by the respective workforce reductions but had not received a response at time of publication.

Palatin CEO claims ‘a positive study on its face,’ despite dry eye drug's primary endpoint miss

Palatin Technologies has some changes to make for the upcoming trials of the dry eye disease drug PL9643 after the phase 3 MELODY-1 study failed on the main goal of improving a clinical sign of the disorder.
CEO Carl Spana, Ph.D, spun the results as a positive on a Wednesday morning conference call, pointing to an analysis that considered age and sex that showed statistical significance on the primary endpoints measuring pain and a clinical sign of dry eye disease.

But analysts—and investors—weren’t convinced. The company’s shares fell nearly 40% to $2.40 as the markets opened Wednesday, compared to $3.97 at Tuesday’s close.

MELODY-1 had two co-primary endpoints measuring pain and a clinical symptom, plus other secondary symptom and sign endpoints of the condition. PL9643 was tested over 12 weeks with a four-week run-in period. The trial did not reach statistical significance on the sign endpoint or on secondary sign endpoints, although Palatin noted that the treatment “demonstrated positive treatment effects over vehicle.” Without any adjustments, the main goals of the test were unsuccessful.

On the pain endpoint, MELODY-1 did reach statistical significance.

“It is important to note that it is rare for one clinical study in DED to show efficacy for both a sign and a symptom. While additional analyses are ongoing, the initial results reinforce the potential of PL9643 as a treatment to address both symptoms and signs of DED,” Spana said in a statement.
When adjusted for age and sex, Spana said the therapy was a success.
“Although we did not reach the significance for pain, it actually had a p-value of 0.03—so very close,” Spana told investors on the call. “And in addition, we had multiple other symptom endpoints that actually did transcend 0.025, so we now have a positive study on its face.”
Spana explained that the FDA requires sponsors to examine covariances that could affect the outcome of the study, and in doing so, Palatin noticed the age and gender differences. Both are known to be factors in dry eye disease.

The study had a large population of people over the age of 60 and 70% were women.
“It’s standard, everyone does it. You’re required to do it. And in our case, we felt just to be transparent, we wanted to make sure everybody understands exactly what we’re doing,” Spana said. “So from our context, this is the analysis plan that they would be doing at the agency, this is what we will do in the next studies. So we feel very confident that they will be absolutely fine with what we’re doing.”
Palatin has much more work to do before a new drug application can be submitted. The company plans to conduct a detailed analysis of the MELODY-1 data and then meet with the FDA to discuss what would be required for an NDA. Two late-stage studies are being planned: MELODY-2 and an open label safety study called MELODY-3. Spana expects enrollment to get underway later this year.
The CEO hopes that the learnings from MELODY-1 can lead to stronger results.

“Just based on precedent, it is very rare for dry eye disease studies, particularly the first large one, to hit everything, to get everything you want,” Spana said.
For the next round of studies, Spana said the “covariances will be there” and they will make adjustments on the level of symptoms required to get into the study. That should make efficacy results clearer. But overall, Spana is pretty happy with the symptom results, so “the sign side is where we’ll focus.” Palatin will tweak the analytical methodology and enroll a larger population.
“We’ll make sure that we nail it,” Spana said.

Designing Compliant Wireless Medical Devices

Designing Compliant Wireless Medical Devices     Wireless medical devices make patient care safer and more efficient. They eliminate tripping hazards that may endanger both medical staff and patients. Secondly, the digital connection of various devices elevates monitoring and diagnostics to a higher level, significantly enhancing reliability in surgery and other medical areas. Wireless medical devices …

Designing Compliant Wireless Medical Devices Read More »

Closed-Loop Technologies Reduce the Burden of Chronic Pain

Closed-Loop Technologies Reduce the Burden of Chronic Pain     In August 2023, Medtronic received CE Mark approval for its Inceptiv closed-loop rechargeable spinal cord stimulator (SCS) — the first Medtronic SCS device to offer a closed-loop feature — for the treatment of chronic pain. The device senses the body’s neural response to stimulation 50 times …

Closed-Loop Technologies Reduce the Burden of Chronic Pain Read More »

Idorsia, weeks from cash-tastrophe, bags $350M upfront from Viatris for 2 late-phase assets

Idorsia has survived a high-wire financing act. Weeks from the end of its cash runway, the biotech has secured $350 million upfront from Viatris in exchange for the global rights to two phase 3 candidates. 
Switzerland-based Idorsia has burnt through cash in recent years, racking up losses of 375 million Swiss francs ($426 million) in the first half of last year alone, as it advanced a broad pipeline. Idorsia gained the assets in the spin out of Actelion as part of Johnson & Johnson’s $30 billion buyout in 2017. At that time, Idorsia had $1 billion and quickly added a further $230 million when J&J picked up an asset. 

Yet, the broad pipeline became as much of a burden as a boon as Idorsia’s share price fell and access to capital dried up, culminating in the biotech warning investors that it would run out of money in April. A deal to avert that apocalyptic scenario has arrived with weeks to spare. 

Viatris, the drugmaker created through the merger of Mylan and Pfizer’s Upjohn, took the other side of the deal. The company has a vast portfolio of generic and over-the-counter products but has been on the hunt for innovative molecules since Scott Smith, the former chief operating officer of Celgene, took over as CEO last year and began to make his mark on the business.

The search for licensing opportunities led Viatris to selatogrel and cenerimod. Idorsia is developing the P2Y12 inhibitor selatogrel to improve outcomes in patients who suffer a second heart attack. By giving people a selatogrel autoinjector to use if they feel the onset of a second heart attack, Idorsia believes it can improve outcomes by starting platelet inhibition sooner than under the current care pathway.

Cenerimod is a S1P1 receptor modulator that Idorsia is developing in systemic lupus erythematosus. The molecule is designed to prevent the migration of overactive T cells and B cells into target organs, while also reducing the transport of autoantigens to the lymph nodes and suppressing inflammation. 
Idorsia began a phase 3 trial of selatogrel in 2021 and started pivotal studies of cenerimod in 2022 and 2023. However, the primary completion dates of the clinical trials are all in 2025 and 2026, well beyond the end of Idorsia’s pre-deal cash runway. Idorsia expects the deal to close by the end of March and is still working on business development opportunities and equity pacts to further extend its cash runway. 
Handing the global rights to Viatris will give Idorsia a cash injection and help with the development costs, albeit while still leaving a sizable bill. The Swiss biotech will contribute up to $200 million to the cost of the programs over the next three years. Idorsia will move dedicated personnel to both programs to Viatris at closing, and has given its partner the right to first refusal and negotiation on other assets. 

At Viatris, the deal marks the start of the next phase of Smith’s plan for the business. Last year, Viatris struck deals to sell three businesses for $3.6 billion. Progress on plans to divest assets allowed Viatris to be “more aggressive in looking at opportunities,” Smith said at this year’s J.P. Morgan Healthcare Conference. Smith only needed to open his inbox to find potential ways to spend the money.
“The vast majority of the business development activity that’s coming in is inbound to me based on my past at Celgene, my past in biotech and other things. It’s been a little bit of a capital-starved world in the biotech world, so there’s been very significant outreach coming to me,” Smith said. 
Smith identified ophthalmology, dermatology and gastrointestinal disease as Viatris’ core areas but also expressed a willingness to be “opportunistic” about assets outside of those three spaces. With Idorsia weeks away from a cash catastrophe, Viatris spied and seized on an opportunity.  

Revolution via resolution: AbbVie pays $48M for OSE's preclinical chronic inflammation drug

AbbVie is talkin’ bout a resolution. The Big Pharma has bet $48 million on OSE Immunotherapeutics’ antibody platform, handing the biotech the upfront fee and dangling another $665 million in milestones for global rights to a novel approach to chronic inflammation. 
Inflammation drives tissue damage and scarring in patients with chronic inflammatory and autoimmune diseases. Conventionally, biopharma companies seek to prevent harm to tissue by developing drugs that block pro-inflammatory pathways. OSE’s anti-ChemR23 monoclonal antibody comes at the problem from a different angle.

The candidate, code-named OSE-230, is designed to resolve, rather than block, inflammation. In healthy individuals, the inflammation process concludes with the restoration of tissue integrity and the return to normal function. When that fails to happen, people develop chronic inflammation.

ChemR23, a G-protein coupled receptor, is expressed by various immune cells and plays a role in starting and ending inflammation. In preclinical tests, OSE has shown targeting ChemR23 affects the activity of macrophages and neutrophils in ways that may accelerate the resolution of acute inflammation. 

AbbVie sees promise in the approach and has secured the global rights to OSE-230. In a statement, Jonathon Sedgwick, Ph.D., AbbVie’s global head of discovery research, called ChemR23 agonism “a novel mechanism-of-action to treat chronic inflammation” and outlined plans to apply the company’s expertise in immunology to the development of OSE-230.

Shares in OSE jumped 60% to above 5 euros ($5.40) in early trading in Paris. The deal provides external validation of an idea that OSE has been working on for years. OSE-230 is the most advanced candidate from OSE’s “pro-resolutive antibody platform” but, with the biotech focused on assets such as its late-phase cancer vaccine Tedopi, it has remained stuck in the preclinical pipeline.
OSE published data from inflammatory preclinical and ex vivo human models in 2020. The biotech linked (PDF) the antibody to the resolution of inflammation in models of chronic colitis, Type 1 diabetes and multiple sclerosis. OSE published a Science Advances paper about its work in chronic colitis the next year and followed up with an abstract about the effect of the antibody on neutrophil recruitment in 2023. 
The early-stage nature of OSE-230 suits AbbVie just fine. On an earnings call early this month, Robert Michael, who will become AbbVie CEO in July, said the company is focused on finding assets to “drive growth in the next decade.” That means “early-stage opportunities, which are typically smaller-sized deals,” Michael said, and, in immunology, “new mechanisms of action that can elevate standard of care.”

ObsEva winds down and lays off all staff as money, options run out

With its money and options dwindling, ObsEva has finally decided to wind down operations and lay off all its employees.
The writing has been on the wall for the Swiss biotech since July 2022, when the company’s Nasdaq stock nosedived in the wake of the FDA’s claim that ObsEva’s highly touted uterine fibroid candidate linzagolix wasn’t fit for an on-time approval. In response, the company announced a 70% reduction in workforce two months later, with the remaining staff focused on preterm labor treatment ebopiprant and oral oxytocin receptor agonist nolasiban.

By November 2022, the company had sold off ebopiprant to Xoma in an attempt to resuscitate its share price. When that failed, the company let go of its Boston-based executives—including CEO Brian O’Callaghan—and consolidated its remaining operations in Switzerland in February 2023. The company’s narrower focus became nolasiban, which it was investigating as a way to improve in vitro fertilization success rates.

The company delisted from the Nasdaq the following month, but has now informed the SIX Swiss Exchange’s listing authority that it is “likely not going to be able to satisfy the requirements for maintaining its listing on SIX.”

The Geneva-based company has secured a four-month moratorium from being chased by creditors for its debts until the end of May while it winds downs operations.

Current CEO Fabien de Ladonchamps informed investors last month that ObsEva was “currently engaged in advanced negotiations for a transaction pertaining to nolasiban.”
“While we are pursuing our efforts to monetize nolasiban, our compound designed to increase live birth rate for women undergoing IVF, ObsEva’s cash position remains low, which compromises our ability to keep operating the company with existing workforce and to have financial statements prepared,” de Ladonchamps said in this morning’s release.
While de Ladonchamps’ employment will be terminated along with all of his colleagues, ObsEva said he will “remain in function during the notice period for the duration of the moratorium process and beyond, as needed.”
At its last earnings report back in the summer, ObsEva revealed that the $3.3 million it had available in cash and equivalents was likely to run out by the end of last year. Since being founded in 2012, the biotech had racked up losses of $482.7 million and “expects to continue to generate operating losses for the foreseeable future,” it said at the time.

NIST Releases Updated Cybersecurity Framework

NIST Releases Updated Cybersecurity Framework     The National Institute of Standards and Technology (NIST) has updated its Cybersecurity Framework (CSF) guidance document for reducing cybersecurity risk. The new 2.0 edition, which supports implementation of the National Cybersecurity Strategy, has an expanded scope that goes beyond protecting critical infrastructure, such as hospitals and power plants, to …

NIST Releases Updated Cybersecurity Framework Read More »

Janux's shares jump 130% as analysts see blockbuster potential in phase 1 T-cell engager data

Janux Therapeutics’ executives aren’t the only ones celebrating positive interim phase 1 data from two tumor-focused candidates, with analysts touting their blockbuster potential and investors doubling the biotech’s share price this morning.
The candidates in question are a tumor-activated T-cell engager called JANX007, which is being tested in patients with advanced or metastatic prostate cancer, and JANX008, which is being trialed in advanced or metastatic solid tumors expressing high levels of EGFR.

A total of 23 patients had been treated with JANX007 as of Feb. 12, as part of the dose-escalation portion of a phase 1a trial. Of the 18 patients who received a starting dose of 0.1 mg, 14 (78%) achieved a prostate specific antigen (PSA) decline of at least 30%, with 10 of those patients (56%) achieving a decline of 50%.

All six of the patients who received a 0.2-mg starting dose of JANX007 achieved a 30% reduction of PSA, with five of the six achieving a 50% reduction, Janux reported in the Feb. 26 postmarket release.
“Initial step doses of JANX007 ≥ 0.2 mg drove deeper and more durable PSA responses, including one subject that achieved a [90%] decline,” the biotech added.

Cytokine release syndrome (CRS)—a severe immune reaction that has haunted other biotechs’ T-cell therapy trials—was observed in the study, but Janux stressed that the cases were “temporary … presenting only as low-grade 1 or 2 events, and was quickly managed with treatment.”
“These incidents of CRS were mainly reported during the first treatment cycle, with no subsequent occurrences in later cycles,” the company added. Most non-CRS adverse events related to treatment were of low severity and also mainly occurred in the initial treatment cycle, with no grade 4 or 5 events reported.
By the same review point, 11 patients with either colorectal cancer, squamous cell carcinoma of the head and neck, non-small cell lung cancer (NSCLC) or renal cell carcinoma (RCC) were treated in the JANX008 phase 1a trial at doses of up to 1.25 mg. Janux said the drug has shown “encouraging signs of clinical activity,” including a NSCLC patient who saw a 100% reduction in their target lung lesion and no cancer growth beyond the liver, along with no CRS or treatment-related adverse events.
The other patient Janux picked out had a 12% reduction in their RCC, although they also experienced grade 1 CRS. Only one other patient experienced CRS and this was also grade 1, according to the biotech.

Based on the safety profiles of both drugs, Janux is continuing dose optimization for both trials. The company expects to provide an update on JANX007 doses for expansion in the second half of 2024.

The readouts “provide compelling proof-of-concept” for Janux’s TRACTr platform, which has been designed to “provide an entry point to multiple, large solid tumor indications that are intractable with conventional T-cell engager approaches,” CEO David Campbell, Ph.D., said in the release.
Analysts at William Blair also saw the potential in the tech, saying that yesterday’s readout had raised their estimation of JANX007’s success from 40% up to 60%.
“We estimate peak sales of $1.5 billion in chemotherapy refractory metastatic prostate cancer, but moving the treatment into earlier lines of the treatment paradigm creates multi-blockbuster potential,” they said in a Feb. 27 note.
Investors appeared similarly enthused, with Janux’s stock beginning trading on Tuesday at $36.27—a 137% increase on the $15.10 closing price on Monday.