Eli Lilly, AbbVie in M&A action; Gilead’s oncology deal spree; FDA puts its foot down on Makena; and more
22 Oct 2022
Gene TherapyAntibodyBiosimilarOligonucleotideCell Therapy
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I was somewhat detached from the news this week to work on a reporting project, so putting this weekly together was an especially helpful exercise in reviewing what happened. If it does the same for you, please do spread the word — or let us know what we can do better.
These days, M&A action is jumping out from unexpected corners. Korea’s LG Chem swooped in to buy Aveo Oncology, plucking it off Nasdaq in a $566 million all-cash deal that gives it a new commercial footprint in the US built on Aveo’s work commercializing Fotivda, for which it notched an FDA approval last year after a long, winding journey. Eli Lilly is similarly wading into a relatively new space in acquiring gene therapy developer Akouos — whose lead products are geared at hearing loss. That deal is good for $487 million cash plus $123 million in CVR. Then, AbbVie closed us out with a $255 million cash bid for the discovery outfit DJS Antibodies and its lead program for fibrotic diseases.
Gilead’s oncology deal spree
On a continual search for more baskets to put its oncology eggs in, Gilead landed on a hot target: CD123. Specifically, it grabbed a bispecific antibody from MacroGenics, a seasoned biotech player that’s seen its share of ups and downs, for $60 million upfront more than $1.6 billion in biobucks. Days later, it dipped its toes into early-stage discovery work in CAR-T, signing a deal with Refuge Biotechnologies to access a gene expression platform with the goal of developing next-gen cell therapies for blood cancer.
FDA puts its foot down on Makena
The FDA put all of its highest-ranking CDER officials on display on the first day of a two-and-a-half-day hearing on whether to pull Covis Pharma’s pre-term birth drug Makena and its generic versions from the market, and they all laid out a staunchly negative perspective. And its Obstetrics, Reproductive and Urologic Drugs Advisory Committee of outside experts adopted the same view, voting 14-1 to pull the controversial therapy from the market after its confirmatory trial from 2018 failed to confirm the drug’s benefit for babies or mothers.
Landmark Novartis licensing pact
The Medicines Patent Pool unveiled a voluntary licensing agreement with Novartis to increase access to its patented, second-line chronic myeloid leukemia drug Tasigna (nilotinib) in certain lower-income countries. The drug, which is on the WHO’s list of essential medicines, will now be made by select generic manufacturers and offered in seven middle-income countries — Egypt, Guatemala, Indonesia, Morocco, Pakistan, the Philippines and Tunisia — where patents on the product are pending or in force.
Trial coordinators jailed
Two Florida-based CRO study coordinators who pleaded guilty to falsifying clinical trial data have been sentenced to more than two years in prison each, along with $2.1 million in fines related to their conspiracy to commit fraud. They were accused of fabricating patient information, medical records and recruiting people who were not eligible for trials for profit.
SPACs tell different tales
If you’re wondering what the SPAC market is looking like, the answer might be it depends on who you’re asking. As Eduardo Bravo’s European Biotech Acquisition Corp closed its merger with eye care biotech Oculis SA and another prolific SPAC creator — Mountain Crest Acquisition Corp, now onto blank check merger #5 — takes AUM Biosciences to Nasdaq, Sanaby Health made the call to dissolve after deciding it can’t find a company to take public within the mandated time frame.
Editas pipeline shuffle
Storied CRISPR gene editing pioneer Editas Medicine is in advanced discussions regarding the sale of the preclinical oncology lineup in its pipeline as the biotech works through a makeover of the company, its executive team and the drugs it hopes to take through the clinic, John Carroll learned. One key asset in the negotiations is an NK cell therapy.
Disease awareness daze?
Most people know that October is Breast Cancer Awareness Month. But it’s also Healthy Lung, National ADHD Awareness and Spina Bifida Awareness month. And it also includes individual weeks for Mental Health Illness Awareness and International Infection Prevention, along with separate awareness for World Sight, World Osteoporosis and World Psoriasis days. These are some of more than 200 health and disease awareness observances officially recognized by Congressional resolutions in the US. But how much is too much? Do disease awareness months — and the related unbranded marketing campaigns from pharma companies — lose their punch with people after the steady drone day after day and year after year? The answer isn’t a simple yes or no, Endpoints MarketingRx editor Beth Bulik writes.
Facing some possible macroeconomic pressures, such as inflation, Covid-19 and other global events, J&J is holding off on the volatile M&A market for now. But with $34 billion in cash on hand and the consumer health business off its back, the company feels confident that when it’s bullish or even eager to do something, it won’t act out of desperation.
Roche scrapped a mid-stage drug candidate for geographic atrophy from its pipeline. Quietly tucked into the Q3 earnings presentation, the decision to stop treatment with galegenimab in a Phase II trial was made “because the risk/benefit ratio was considered not supportive of further treatment.”
On the commercial side, Roche missed its mark on sales goals for the quarter just passed, due in part to the entrance of biosimilar competition and the decline of Covid-19 drug sales. And while the company does plan on seeing Covid-19 drug sales getting better sometime soon, outgoing CEO Severin Schwan doesn’t expect it to last into next year.
Behind Amgen and Mirati are a whole host of players lining up their own KRAS candidates. Roche, which already has its own in-house KRAS drug in Phase I studies, doubled down by licensing a preclinical KRAS program from Hookipa Pharma for $25 million upfront. It also has the option of adding a second immunotherapy candidate for $15 million.
Kenneth Galbraith wanted for Zymeworks what Daiichi Sankyo got for its HER2 breakout drug: a Big Pharma partner that could turn its bispecific into a worldwide med. While not quite the size of an AstraZeneca, Jazz Pharmaceuticals is coming in to fill that role, doling out an initial $50 million for a $1.7 billion deal around the HER2 bispecific antibody known as zanidatamab.
Takeda just added another program to its determined campaign to tackle celiac disease from all sides. The global player joined hands with Zedira and Dr. Falk Pharma GmbH on a Phase IIb celiac drug dubbed ZED1227/TAK-227, giving Takeda dibs on the big US market plus a string of countries outside Europe.
GSK has been aggressively looking at AI, and in the newest bid to beef up its capabilities, it is broadening its partnership with AI outfit Tempus, which specializes in de-identified patient data. Having worked together since 2020, GSK has paid Tempus $70 million upfront for three more years of partnership, focused on improving clinical trial design, speeding up enrollment and identifying drug targets.
Oncology-focused Advaxis is in the early stages of merging with Ayala, a penny stock biotech incorporated in Delaware that has its primary operations located in Israel. As Advaxis looks to become a later-stage company, the combined company would focus on Ayala’s lead program, a Phase II/III drug to treat desmoid tumors.
SCOOP
After running into a setback for its hepatitis B program, OrbiMed-backed Enyo Pharma is seeking partners to move that forward while it focuses on testing an FXR agonistFXR agonist among a subset of NASH patients who also have chronic kidney disease and running a Phase II study in the rare disease Alport syndrome.
DermBiont axed trials of its original lead dermatology gel after running into hurdles during Covid-19, the biotech confirmed to Endpoints, and with little investor appetite for microbiome-based therapies, the company is raising a Series B to bankroll studies of other topicals it acquired in recent years.
For years, Utah’s Thunder Biotech has flown under the CAR-M radar, being outpaced by the likes of Moderna-allied Carisma Therapeutics. The upstart worked calmly, put walls around its IP and purposefully steered clear of talks with institutional investors and pharmas before it would open its doors to a potential storm of attention. But it’s ready to break cover.
More than 60 jobs are on the chopping block at J&J’s New York City Design Center, according to a state filing. The company reported a “plant layoff” to the state’s WARN system, noting that 64 employees will be laid off from Jan. 13 to Jan. 26, 2023. J&J listed the reason behind the cuts as “economic,” noting that the staffers are not part of a union.
Geneticist and serial entrepreneur Dietrich Stephan had hoped that his antisense oligonucleotide platform at NeuBase Therapeutics could expand the field that Ionis pioneered to new areas. But in these turbulent times, he’s decided to put that on the back burner and pivot to gene editing instead. As a result, the biotech is letting 60% of its staffers go.
Invivyd, the company formerly known as Adagio before a rebranding last month, is leaning into a new round of cuts as it looks to shed its troubled past. CFO and CBO Jane Henderson will depart, as well as chief commercial officer Eric Kimble, while the biotech lays off an unspecified number of employees in a push to rebuild itself around a new Covid-19 antibody program.
Scynexis is laying off about 40% of its employees, shaking up its executive team, narrowing the pipeline and winding down promotional activities for its approved yeast infection drug, Brexafemme, which is on tap for an sNDA decision at the FDA in just six weeks. The whole commercial team is being let go as the biotech tries to find a partner.
San Diego-based biotech Ambrx announced that it is reorganizing its pipeline and cutting around 15% of its staff to extend its cash resources. At last count, Ambrx had around 81 employees. But due to a “significant shift this past year in the HER2 metastatic breast cancer competitive landscape,” it decided to shelve its lead HER2-targeting antibody-drug conjugate.
Oz Azam drew the curtain on the neuroscience startup he joined after leaving Tmunity, unveiling a $22 million round for Empyrean Neuroscience — which is focusing on psychedelics but would rather be considered “a genetic engineering company in neuroscience.” In the neuro renaissance, the CEO sees a resemblance to the very early days of CAR-T.
For patients with solid tumors, drugs are given at roughly three different stages: before surgery, after surgery and when the cancer comes back. But what about during surgery? A former Harvard professor who studied under Bob Langer closed a $26 million Series A to test this idea, with a hydrogel technology to underlie the platform at Surge Therapeutics.
An $8 billion exit from Eli Lilly and a short-lived stint heading up oncology work at the Big Pharma post-Loxo sale have seemingly persuaded Josh Bilenker to be hush-hush about his new venture. Treeline, his new startup with Jeffrey Engelman, put out word that KKR led an expansion to its pool of investors but stayed mum on pretty much everything else, saying only it’s focused on the “outer edge of scientific possibility.”
While the combo of machine learning and drug discovery has managed to rake in capital for multiple companies, Oxford spinout Nucleome Therapeutics is looking to the dark side of the genome to stand out. By decoding “dark matter” from the human genome — the parts that don’t encode any protein — the biotech, armed with $42 million, hopes to find targets no one else has gone after.
FINANCING
David Liu’s CRISPR 3.0 play officially hit Nasdaq after pricing its IPO with $175 million in new cash. Prime Medicine’s public debut comes amid a largely frozen IPO market lasting most of the year, but one that’s seen signs of thawing recently. It’s still early days with human testing not expected until at least 2024, but the gene editing player hopes to bring an approach that’s safer and more effective than early generations.
The executive crew and backers at the little startup Acrivon clearly aren’t averse to risk. They handed over a modest $5 million in cash to pharma giant Eli Lilly in order to pluck one of its mid-stage cancer failures off the shelves. And now they’re jumping into the frigid waters of Nasdaq to see if they can float a very rare IPO.
Cap-binding proteins that bind to one end of RNA sequences direct how they are spliced and translated into proteins. That’s the protein that PIC Therapeutics is targeting in hopes of treating breast cancer — and now, six years after it was spun out from the lab of Harvard physicist Gerhard Wagner, it has $35 million in funding to get that program into the clinic.
In seven years, Orionis Biosciences has built two programs in immuno-oncology and molecular glues and got itself into a partnership with Novartis. Now the transatlantic biotech is refilling its coffers with a $55 million Series C, which it says gives it flexibility to expand its R&D operations, increase its pipeline and bring assets into human trials.
The money continues to flow in the once-dormant neuroscience field — especially to the companies spearheading the renaissance. Denali Therapeutics plans to raise $250 million via a public offering, while on the opposite US coast, Biohaven also announced a public offering that could net $195 million to $224 million.
R&D
More than two years after Milestone Pharmaceuticals’ stock got hammered after its lead heart drug flopped in a crucial Phase III study, the biotech is looking for some redemption now that its redesigned Phase III pivotal has come through with positive results. Not only did the self-administered nasal spray version of a calcium channel blocker help restore heart rhythm, it also kept a significant portion of patients out of the ER.
Long-standing Merck partner NGM Bio took a stock beating in the wake of a Phase II flop. The trial had tested a monoclonal antibody for geographic atrophy, a late-stage form of dry age-related macular degeneration, and Merck still holds an option on the program. The biotech is digging further into the data before determining next steps.
Talaris Therapeutics, which set out to make organ transplants less dangerous, reported that a patient died in a Phase III study of its drug after developing symptoms associated with acute graft-versus-host disease. Though Talaris did not report whether the death was related to its experimental drug, the biotech’s data monitoring committee said the study can continue dosing and new patient enrollment.
The Dutch biotech AM-Pharma lost the Phase III battle on kidney disease, but execs are still fighting the long regulatory war. While the treatment failed an interim analysis on the primary — 28-day all-cause mortality — they’re spotlighting a significant response on a key secondary for a reduction of major adverse kidney events.
Salarius Pharmaceuticals is hitting the brakes on an early clinical trial after a patient’s death. The small Houston biotech paused new patient enrollment in its Phase I/II trial — which focused on two types of rare pediatric sarcomas — to take a pulse of the situation and adjust clinical protocols.
Four months after taking an axe to the pipeline resulting from a Phase II miss, Immunic is back with another miss. But since execs believe the Phase Ib results for a psoriasis drug were confounded by an unexpectedly high placebo response, they still plan to move forward with the drug while sifting through the data.
Bristol Myers Squibb is ready for a data showdown with Merck as they move their respective PD-1 inhibitorsPD-1 inhibitors into earlier lines of melanoma treatment. Among patients with completely resected stage IIB or IIC melanoma, Opdivo reduced the risk of recurrence or death by 58% — translating to a hazard ratio of 0.42 on the primary endpoint of recurrence-free survival.
Not six months after raising cash to take a drug for obstructive sleep apnea through Phase III trials, Apnimed is setting the stage with a positive readout. It reported promising Phase IIb data for its oral candidate AD109 for obstructive sleep apnea, a sleep disorder that has traditionally been treated or mitigated with CPAP machines, suggesting it can reduce instances where breathing completely stops.
LAW
Pfizer is taking a hotly-contested kickback case all the way to the Supreme Court, with a petition seeking to overturn a lower court decision that barred the Big Pharma from providing financial assistance to help Medicare beneficiaries access its drug for a rare and fatal cardiac condition. The court went too far in its interpretation of the anti-kickback statute, Pfizer argues.
As psychedelic therapies make their way through the clinic, one Seattle doctor is taking his fight to change psilocybin’s status as a Schedule I substance to federal court. Sunil Aggarwal petitioned the Ninth Circuit Court of Appeals to review a decision by the Drug Enforcement Administration in September to leave psilocybin, an active substance in some “magic mushrooms,” on the Schedule I list under the Controlled Substances Act.
Last March, the top court in France ordered Merck KGaA to pay out more than $3.6 million to more than 3,000 people to settle a long-running dispute over the reformulation of its hypothyroidism drug. The legal dispute was heightened as a judge in Marseille indicted the German Merck’s French affiliate.
Five years after Takeda shelled out more than $5 billion to acquire Ariad Pharmaceuticals, it’s fending off generic competitors to one of the main drugs from the deal. Filing a lawsuit in New Jersey federal court, it took issue with Canada-based Apotex’s purported plans to launch an Iclusig generic before its final patent expires in 2033.
After the spectacular collapse of Neil Woodford’s flagship fund left them reeling from big losses, thousands of investors are now suing players whom they believe are also culpable, namely Hargreaves Lansdown — the trading platform where those investors bought into Woodford’s equity income fund — and Link Fund Solutions, the fund’s authorized corporate director.
Amid speculation on how the waning demand for Covid-19 vaccinesCovid-19 vaccines, coupled with the looming end of government purchasing programs, would put pressure on prices, Pfizer came out with an expected price tag on its BioNTech-partnered shot for the US market: $110 to $130. The move comes just as the CDC recommends adding Covid-19 jabs to regular immunization schedules.
Bristol Myers Squibb lined up a date with the FDA to expand the footprint of mavacamten, the drug it obtained from the $13 billion buyout of MyoKardiaMyoKardia now marketed as Camzyos. The label expansion would cover a subset of patients with obstructive hypertrophic cardiomyopathy who have severe symptoms and are referred to septal reduction therapy.
Pharma companies may be up in arms with Democrats over recently passed legislation to allow Medicare to negotiate certain expensive drug prices, but their political action committees aren’t showing it. According to the federal campaign finance database, the PACs for eight pharma giants contributed more than $7 million to national and state political campaigns last year and so far this year.
Analyzing data that were disclosed thanks to a drug price transparency law, drug pricing watchdog ICER flagged increases that they said were unsupported by new clinical evidence: Bristol Myers Squibb’s cancer drugs Revlimid and Sprycel, and Eli Lilly’s migraine drug Emgality. But the group also called for net prices, not just WACs, to be included.
ICER also took a closer look at how TG Therapeutics’ ublituximab stacks up with existing MS treatments. At a placeholder price of $71,187 in wholesale acquisition cost, or $55,081 a year, the group finds the drug missing the bar on cost-effectiveness when compared to the market-leading oral, even if it has comparable or even better efficacy.
MARKETINGRX
SpringWorks Therapeutics is tackling the little-known and misunderstood rare desmoid tumor condition — and the message seems to be resonating with patients. The “Rethink Desmoid Tumors” campaign website shows the unpredictable and tendril-like desmoid tumors’ growth and also offers patients with educational information and guides, on top of personal stories.
Is pharma advertising recession-resistant? That’s likely to be a popular question in the industry in the coming months, but for now, it’s holding steady — and even faring better than most other categories. Pharma advertising spending rose 12% in September compared year over year to the same month in 2021 and saw the second-biggest jump among 12 categories, according to Standard Media Index’s monthly trend report.
It’s not a big secret, but pharma advertisers love TV news programming. As midterm elections and political news trend on morning and evening live news shows, pharma TV ads are seeing a correlating uptick, according to the latest data from TV ad tracker iSpot.tv. The top overall show for pharma advertising in the third quarter was “ABC World News Tonight with David Muir.”
With public health officials gearing up for a tough flu season, so is GSK consumer spinoff Haleon and its flu brand Teraflu by offering a helping hand to workers who don’t get sick leave. Now in its second year, “The Right to Rest and Recover” campaign is spreading the word, especially about the inequities around unpaid sick time for Black and Latina mothers. It’s also delivering checks to 1,200 families to make up for the lost wage.
Commercialization service provider Indegene is building out its life sciences marketing business again — this time acquiring a pharma marketing agency. CultHealth is its first ad agency buy, although Indegene has been “providing advertising and marketing services to life sciences companies for many years.”
Novartis is continuing to spotlight real patient stories in the latest social media campaign for breast cancer drug Kisqali; California turns to advertising to help with its opioid problem, taking bids for a $40 million contract for opioid overdose and misuse prevention — but not from any agencies with conflicts of interest; GSK’s spinoff Haleon embraces tech in two efforts to close gender gaps, improve label visibility; and more can be found in the MarketingRx roundup.
FDA+
Biogen will have to wait three more months to hear back from the FDA about tofersen, the antisense drug for a genetic subset of ALS. Although the drug failed the functional endpoint in a Phase III test, Biogen is gunning for accelerated approval based on a surrogate biomarker. The FDA had requested additional information, but it’s not clear what they sought.
As Congress punted the user fee riders on accelerated approval pathway reforms, FDA commissioner Rob Califf made clear at a conference that such reforms need to happen “as soon as possible.” The comment followed similar suggestions for reforms from officials at the FDA’s Oncology Center of Excellence, who took to the NEJM late last month.
The UK’s Medicines and Healthcare products Regulatory Agency is increasingly granting drugmakers access to its exclusive Innovative Licensing and Access Pathway. Its latest selection for the program is Cambridge, MA-based Avrobio’s investigational gene therapy that uses patients’ own hematopoietic stem cells to treat the rare Gaucher disease.`
In the latest roadblock for companies developing new treatments for Huntington’s disease, the FDA paused enrollment in a Phase II trial testing PTC’s RNA-targeting molecule. The biotech stopped short of saying the words “clinical hold,” but the FDA’s request for additional data is keeping it from dosing new patients in the US.
Back in 2020, despite the FDA spelling out its qualms with Minerva Neurosciences’ schizophrenia drug candidate roluperidone — explicitly asking for more data — the biotech pushed forward with meetings attempting to assuage concerns and, earlier this year, went ahead seeking approval. But regulators are still not buying it, slapping the application with a refuse-to-file letter.
How can drugmakers avoid false conclusions about a drug’s effect? The FDA released finalized guidance, first drafted in 2017, that will help sponsors better understand the FDA’s thinking about the problems posed by multiple endpoints in the analysis and interpretation of clinical trial results, and how these problems can be better managed.
With tens of billions in annual appropriations, a group of four Republican senators is publicly wondering why the NIH can’t do a better job of ensuring taxpayer-funded research is published in a timely manner. Writing in a letter to NIH acting director Lawrence Tabak, they asked for the names of grant recipients who failed to comply with reporting requirements and next steps.
With the rise of new drugs that won accelerated approvals for genetically targeted (rather than tissue-targeted) cancer therapies, the FDA released new draft guidance to help cancer drugmakers try to make similar strides in what’s known as tissue-agnostic development. The agency also highlights some of the challenges associated with running trials across different tumor locations.
MANUFACTURING
Sanofi is selling off a piece of its manufacturing operations in Japan to the German CDMO Adragos Pharma, although it will continue to have its products manufactured at the site through a new supply agreement. Adragos intends to keep the oral solids and sterile liquids production as well as packing and inspection operations for now, and will also take the employees there under its wings.
Two compliance programs — largely related to the intro to new ICH work — went into effect with new information about preapproval and other drug manufacturing inspections. According to the agency, the revision provides instructions to FDA staff for obtaining information from manufacturers, while companies can glean new information about what the FDA wants from looking into the guides.
It’s been almost a year since Roche’s blockbuster ocular implant, the wet age-related macular degeneration drug Susvimo, was cleared for twice-annual administration as opposed to monthly injections. Now the company is putting a hold on it with a voluntary recall due to a manufacturing issue.
The FDA sent a warning letter to Legacy Pharmaceutical Packaging, a contract pharma packager based in Earth City, MO, raising concerns over quality and cleaning issues at its facility. The company was noted for having product mix-ups, with equipment design flaws and improper cleaning, and its response promising to investigate was not satisfactory for regulators.
Indian drugmaker Torrent Pharmaceuticals, which has pulled in about $1 billion in 2022 revenue, is the latest Indian manufacturer to find itself under the FDA’s microscope. A 15-page Form 483 inspection report followed an inspection from September that revealed cleaning and other quality issues at its manufacturing site in the village of Indrad.
After starting construction last year on the back of an acquisition and putting the finishing touches this year, Charles River is finally ready to cut the ribbon on a new 16,000-square-foot facility built on an old AstraZeneca site. The manufacturing facility will produce plasmids to be used in cell and gene therapies.
A few days after more manufacturers of generic Adderall capsules announced upcoming shortages, the FDA chimed in with an official announcement that there is a shortage of the immediate release version of the generic, which treats ADHD and narcolepsy. Teva, one of the main manufacturers, acknowledged the potential for temporary backorders but expects an inventory recovery in the coming months.
The FDA handed down inspection reports to Hikma Injectables’ outsourcing facility in Dayton, NJ, and an Eli Lilly facility in Branchburg, NJ; The Government Accountability Office chimed in on the potential gaps and risks in the HHS’ Strategic National Stockpile; The WHO reported that a strained global supply of cholera vaccines forced the hand of the body that manages the emergency supply of vaccines; for all your manufacturing news briefs, check out the Manufacturing roundup.
DON’T MISS
As new research presents the Epstein-Barr virus as an ever greater threat — notably with a deepening link to multiple sclerosis — researchers are accelerating efforts to develop a vaccine. A look at the two candidates that have entered clinical trials in the past year, and others behind them, reflect different approaches to tackling the virus and the challenges.
The trade association ABPI is warning of a “clear and serious” and “existential” threat to the future of biotech research in the UK. In the five-year span from 2017 to 2021, the number of patients recruited for UK clinical trials has fallen dramatically, per a new report, which also urged the government to take action.
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