Inflation Reduction Act: Big Changes Lurking for Drug Development

Alex Harding, MD, senior vice president, Remix Therapeutics; internal medicine physician, MGH.

Seven years ago, Martin Shkreli raised the price on Daraprim, an old generic drug that’s vital for some HIV patients. The price rocketed up 5,000 percent – from $13.50 a tablet to $750.

Outrage ensued. The backlash was felt across the entire biopharmaceutical industry. Then-presidential candidate Hillary Clinton vowed, in a tweet, to “lay out a plan to take…on” drug price gouging. The XBI biotech stock index lost about half of its value over the next year.

Read More


Protein Degraders For Outside the Cell: Aetna Wun Trombley on The Long Run

Today’s guest on The Long Run is Aetna Wun Trombley.

Aetna is the CEO of South San Francisco-based Lycia Therapeutics.

Aetna Wun Trombley, CEO, Lycia Therapeutics

Many in biotech know about targeted protein degraders. Arvinas and Kymera Therapeutics are a couple of the well-known companies that make these drugs which work to inhibit intracellular proteins. This approach has drawn a lot of excitement because it can access previous ‘undruggable’ proteins, by essentially dragging them into the cellular garbage bin.

Fascinating as this is, there are many other proteins that can’t be targeted this way because they are secreted outside cells, or reside on the cell membrane. This requires a different approach. That’s where Lycia comes in.

The company is working on lysosomal targeting chimeras, known as “Lytacs” for short. Carolyn Bertozzi, the chemist who won the Nobel Prize earlier this month, has her fingerprints all over this one. Her team at Stanford University devised a method for binding both a cell surface lysosome targeting receptor and the extracellular domain of a target protein.

That work was posted in a preprint in 2019, and then a year later in Nature. Versant Ventures was among the many groups who read the preprint and wanted to get to work developing a potentially new class of medicines. They agreed to work together, Versant committed $50 million, Aetna was recruited as CEO, and the company was off and running. A little over a year ago, Lycia struck a multi-year research collaboration with Eli Lilly that brought in a $35 million upfront payment.

This is a big idea for drug discovery.

Program note: This conversation was recorded in early September, before Bertozzi won the Nobel Prize for her work on biorthogonal chemistry. Listeners may want to go back and listen to a Long Run podcast I did with Bertozzi in April 2019.

Now before we get started, a word from the sponsor of The Long Run.

Calgary is home to more than 120 life sciences companies, from emerging startups to established firms. With this critical mass of research, technical talent and expertise, the city is an active hub for life sciences innovation.

Technologies homegrown in Calgary are changing the face of healthcare. Syantra is revolutionizing breast cancer detection using artificial intelligence-derived algorithms. NanoTess is harnessing the power of nanotechnology to tackle chronic wounds and skin conditions. And this is only the beginning. Calgary’s life sciences sector is projected to spend $428 million on digital transformation by 2024.

If you’re a bright mind or bright company solving global health challenges, Calgary is the place for you. 

Take a closer look at why at calgarylifesciences.com

Now, please join me and Aetna Wun Trombley on The Long Run.


The Kilimanjaro Climb to Fight Cancer Is Back

Luke Timmerman, founder & editor, Timmerman Report

I’m taking another biotech team to the highest peak in Africa.

Four years after the first team expedition, the Kilimanjaro Climb to Fight Cancer is back with a new crew of 28 biotech executives and investors. Together, we’re going to push ourselves physically, and raise more than $1 million for research at the Fred Hutch Cancer Center.

The expedition is set for Feb. 9-20, 2023.

Most people on the team don’t know each other, and don’t know what it’s like to hike in the thin air of 19,000 feet. A couple are veterans of previous expeditions. Everyone is training to get ready.

They love nature. They love science. They dislike cancer. They are making a big commitment to others in need, raising at least $50,000 apiece.

Here’s who is coming together for this challenging and memorable adventure:

  • Luke Timmerman, founder & editor, Timmerman Report
  • Nathan Trinklein, co-founder and CSO, Rondo Therapeutics
  • Bharatt Chowrira, president, Puretech Health
  • Jens Eckstein, managing partner, Apollo Health Ventures
  • Mark Mendel, venture partner, Sozo Ventures 
  • Wendy Nelson, president and founder, Boston Biotech Forum; corporate development Danforth Advisors
  • Brian Gallagher Jr., biotech investor and entrepreneur
  • Soufiane Aboulhouda, PhD candidate, Church Lab, Wyss Institute, Harvard University, co-founder and president, Nucleate
  • Melinda Richter, global head of Johnson & Johnson Innovation JLABS
  • Paul Biondi, president, Pioneering Medicines at Flagship Pioneering
  • Ben Vollrath, COO, Amprion
  • Ramani Varanasi, managing director, ReVive Advisors
  • Ram Aiyar, CEO, Korro Bio
  • Greg Verdine, co-founder and CEO, LifeMine Therapeutics
  • Kamal Puri, CSO, OncoResponse
  • Nerissa Kreher, chief medical officer, Entrada Therapeutics
  • Saul Fink, SVP, pharmaceutical & nonclinical development, Normunity
  • Alex Federation, co-founder and CEO, Talus Bio
  • Amber Kaplan, consultant, Amber Lab LLC, MBA candidate USC Marshall School of Business
  • Laurie Halloran, founder and CEO, Halloran Consulting Group
  • Joanne Smith-Farrell, CEO, Be Biopharma
  • Jeff Leek, vice president, chief data officer, professor; Fred Hutch Cancer Center
  • Ginger Cooper, associate vice president, Agilent Technologies
  • Jung Choi, chief business and strategy officer, Global Blood Therapeutics
  • Joe Budman, vice president of biology, Vicinitas Therapeutics

The first Kilimanjaro trip in 2019 was a huge success, raising more than $1.5 million for cancer research at Fred Hutch. People on that trip formed lifelong friendships. They open doors for each other.

One of the people from that team summed up the experience:

“The proposition: join Luke and equal numbers of men & women in our industry to raise money for cancer research at the Fred Hutch and climb Mt. Kilimanjaro. This was the experience of a lifetime. Breathtaking scenery – yes. Challenging physically – yes. Outstanding guides & support personnel – yes. Life-long bonding experiences – yes!”  — Bill Newell, CEO, Sutro Biopharma

You can do a couple things to help.

First, go to the Kilimanjaro 2023 team fundraising page on FredHutch.org and find a member of the team you want to support. You can click on their name to open their personal fundraising page, and donate. That will help them hit their $50,000 goal. They’ll appreciate it!

Second, if your company is interested in raising awareness of your support for cancer research, ask us about team sponsorship opportunities. See me at luke@timmermanreport.com and Elizabeth ‘Za’ Martin at eamartin@fredhutch.org.

The biotech community has tremendous capacity for scientific genius and human generosity. When we exercise these muscles, we get stronger.

I’m eager to see what this next Kilimanjaro team, with your help, will do to propel science and improve the lives of cancer patients.

The Kilimanjaro 2023 team

[ngg src=”galleries” ids=”21″ display=”thumbnail” thumbnail_crop=”0″]


Eisai and Biogen Bounce Back, Amylyx Approved, & Illumina’s Big Reveal

Luke Timmerman, founder & editor, Timmerman Report

Reporting this week from foggy San Diego and the Illumina Genomics Forum.

The market leader in DNA sequencing dimmed the lights Thursday morning, and pumped up the music. It was seeking to fire up the crowd of 1,000 genomic scientists, healthcare leaders, entrepreneurs. Illumina was orchestrating a big reveal — a series of technology advancements in product lineup it calls the NovaSeq X Series.

Read More


Going Upstream Against Inflammation: Samantha Truex on The Long Run

Today’s guest on The Long Run is Samantha Truex.

Samantha is the CEO of Waltham, Mass.-based Upstream Bio.

Samantha Truex, CEO, Upstream Bio

Upstream came out of stealth mode with a $200 million Series A financing in June. It’s a big investment in an antibody aimed at the TSLP receptor. It’s a cytokine – an inflammatory protein – that sits at the top of what scientists call an inflammatory cascade.

The idea is that if you can inhibit TSLP, then it won’t trigger a whole bunch of other cytokines such as IL-4, IL-5, IL-13, IL-17 and more. If you can keep the body from overproducing a wild storm of all those inflammatory proteins, then scientists think you might make a pretty big difference against a range of inflammatory diseases – including severe asthma.

Upstream isn’t the only company working on this target. AstraZeneca won FDA approval in December 2021 for an antibody aimed at the TSLP ligand. That drug, Tezepelumab, is cleared for severe asthma. Upstream seeks to build on that success. Upstream’s lead drug candidate was in-licensed from Astellas Pharma, has already been through extensive preclinical testing, and is being assessed in a Phase 1b trial in asthma patients that’s currently enrolling.

This is a big opportunity, from a commercial perspective and for patients. About 2 million people in the US have severe asthma, and about 30 million worldwide.

Samantha comes to this moment with a wide range of experiences, much of it in business development. She worked at a couple of the early pillars of the Boston biotech community, Genzyme and Biogen. She joined a startup, Padlock Therapeutics, that was acquired by Bristol-Myers Squibb. Her first stint as a startup CEO didn’t end the way everyone hoped it would, but it was a learning experience that opened the door for what she’s doing now.

Now before we get started, a word from the sponsor of The Long Run.

Calgary is home to more than 120 life sciences companies, from emerging startups to established firms. With this critical mass of research, technical talent and expertise, the city is an active hub for life sciences innovation.

Technologies homegrown in Calgary are changing the face of healthcare. Syantra is revolutionizing breast cancer detection using artificial intelligence-derived algorithms. NanoTess is harnessing the power of nanotechnology to tackle chronic wounds and skin conditions. And this is only the beginning. Calgary’s life sciences sector is projected to spend $428 million on digital transformation by 2024.

If you’re a bright mind or bright company solving global health challenges, Calgary is the place for you. 

Take a closer look at why at calgarylifesciences.com

Now, please join me and Samantha Truex on The Long Run.


GSK Lifts Up Spero, AlphaFold Inventors Honored, and a Microbiome Milestone

Luke Timmerman, founder & editor, Timmerman Report

The Federal Reserve took aggressive action to curb inflation this week by raising interest rates. Fed chair Jerome Powell sent clear warnings that this work isn’t done, and will continue until inflation gets back down to 2 percent. That seems like a long way off when inflation is running around 8 percent. Stocks fell.

Readers will notice signs of financial distress reverberating throughout biotech. You can see it in the kinds of deals, the kinds of financings, the kinds of personnel moves that are showing up more regularly in this weekly column.

It’s a time for regrouping, and finding ways to live to fight another day.

Catch up on the news and read the tea leaves as you see fit with Frontpoints.

Read More


Rivus Raises $132M to Harness Mitochondrial Energy to Combat Obesity

Zoe Steinsnyder, correspondent, Timmerman Report

The COVID-19 pandemic isn’t over, and it’s not the only ongoing global health crisis. The obesity crisis has gotten worse the past couple years.

About one in five adults worldwide and one in three in the United States are obese, as defined by a Body Mass Index of 30 or higher, according to data compiled by the Harvard T.H. Chan School of Public Health. Obesity is associated with a variety of health problems like type II diabetes, cardiovascular disease, arthritis, depression, and cardiometabolic disorders. If nothing is done to reverse the trend, an estimated 1 billion worldwide will be considered obese by 2030.

Charlottesville, Virginia-based Rivus Pharmaceuticals is aiming to curtail the worst of the downstream consequences of obesity by developing treatments to help more people lose weight. The goal is to develop controlled metabolic accelerators (CMAs), providing better treatment to a large patient population.

Read More


Pretzel Gets $72.5M for Gene Editing & Small Molecules Against Mitochondrial Targets

A pretzel is a twisty, tasty baked bread snack. It can also be said that mitochondria, the organelles that generate most of the energy in the cell, look a little like pretzels under the microscope.

So if you’re starting a biotech company devoted to discovering drugs against dysfunctional mitochondria, why not play around with the visual shapes, have a little fun, and call your company Pretzel Therapeutics?

“It’s a great name for us,” says Jay Parrish, the startup chairman and CEO. “You’ll never forget it.”

Read More


Amylyx’s ALS Surprise, Regeneron’s Eye Franchise Win & Roche Buys Good

Luke Timmerman, founder & editor, Timmerman Report

Biotech is back in full forward motion after the Labor Day weekend. Catch up on some of the deals, data releases, and financings that mattered in your weekly Frontpoints.

On the Road

I’ll be on the road for the first part of the week, leading a 20-person team on the Timmerman Traverse for Life Science Cares. It’s a 23-mile hike of the Presidential Traverse in the White Mountains of New Hampshire. Our final fundraising total isn’t in yet, but we are going to exceed our goal of $800,000 to support community nonprofits in Boston, San Francisco, San Diego and Philadelphia who are working to help our neighbors fight the effects of poverty.

I’m proud that this initiative has raised more than $1.5 million over the past two years. It’s exciting to imagine where this biotech community drive could go next. Thanks to all of you for your support.

Read the Life Science Cares press release where you can check the list of sponsoring organizations and chip in some support for a friend you may know on the team.

Regulatory Action

An FDA advisory committee voted 7-2 to recommend approval of Cambridge, Mass.-based Amylyx Pharmaceuticals’ sodium phenylbutyrate and taurursodiol (AMX0035) for the treatment of amyotrophic lateral sclerosis (ALS). The FDA has a deadline of Sept. 29 to make its decision on whether to allow commercial sale of the drug for the progressive, and fatal, neurodegenerative disease. The company’s application is based on a Phase 2 study of 137 patients. The FDA hearing drew an unusual amount of attention when Billy Dunn, who leads the FDA’s neurology drug review office, said the agency would apply the “broadest regulatory flexibility” when considering this application. That surprised many observers who predicted a negative outcome based on a tough review by FDA staff in the panel briefing documents.

Part of the surprise around Amylyx stems from Dunn’s recent history. He’s the same FDA official who was part of the team that made the controversial decision to allow sale of Biogen’s aducanumab (Aduhelm) for Alzheimer’s. While the FDA was pilloried for allowing sales to occur for many years without confirmatory evidence of a clinical benefit, this time Dunn voiced the need for regulatory flexibility for patients in need while also challenging Amylyx to voluntarily withdraw the drug from the market if it fails to show a confirmatory clinical benefit. Amylyx co-CEO Justin Klee agreed to that condition in the open meeting, according to STAT.

If approved by the FDA, and priced at $250,000, and allowed to stay on the market because of a confirmed clinical benefit, the new drug for ALS could achieve $1 billion a year in US sales, Umer Raffat, analyst at Evercore ISI, wrote in a note to clients. Shares of Amylyx surged 51 percent to $27.03 on the news.


South San Francisco-based Arsenal Biosciences raised $220 million in a Series B financing to advance its work on programmable cell therapies. New investors included Softbank Vision Fund 2, Bristol-Myers Squibb, and Byers Capital. (TR coverage of Arsenal, Jan. 2021).

Ken Drazan, CEO, Arsenal Bio

Cambridge, Mass.-based Orbital Therapeutics said it’s getting started with undisclosed financing from Arch Venture Partners, Andreesen Horowitz Bio + Health, and Newpath Partners. The company has a license and research collaboration with Beam Therapeutics, and will focus on developing RNA medicines with non-viral delivery technology. Orbital will have exclusive rights to the technology for vaccines and certain therapeutic proteins, and Beam retains rights for gene editing and conditioning for use in cell transplantation. Former Alnylam CEO John Maraganore is board chair, and Giuseppe Ciaramella is the interim CEO.

Washington DC-based Otolith Labs raised $20 million in a Series A financing by Morningside Ventures. It’s developing wearable medical devices for vestibular disorders.

Boston-based Photys Therapeutics said it’s getting started with a $75 million Series A financing led by MPM Capital and which included Omega Funds, Longwood Fund, 8VC, Arkin Bio, Mass General Brigham Ventures, MRL Ventures Fund and others. The company is developing bifunctional molecules to induce phosphorylation in order to attack protein dysfunction not only through targeted protein degradation, but also by activating, inactivating, or stabilizing disease-related proteins.

Deerfield Management agreed to provide up to $130 million, along with scientific and operational support, to Beth Israel Deaconess Medical Center in Boston through a collaboration to develop new therapeutics.  

New York-based Harmonic Discovery, a company using computation to advance combination kinase drug discovery, raised $8 million in seed funding. Innovation Endeavors led. Joel Dudley, a partner at the firm, is joining the board.

Personnel File

Cambridge, Mass.-based Voyager Therapeutics, a gene therapy and neuroscience company, said it hired Peter Pfreundschuh as chief financial officer, Todd Carter as chief scientific officer; and Trista Morrison as senior vice president of corporate affairs. They are part of a new team brought in by CEO Al Sandrock.

Merck said George Addona was named senior vice president and head of discovery, preclinical and translational medicine at Merck Research Laboratories. He was previously vice president of quantitative biosciences.

Boston-based Alloy Therapeutics named Trier Bryant as president of 82VS, Alloy’s affiliated venture studio. Bryant previously was Chief People Officer of the aerospace startup Astra. Alloy said in a statement she “was responsible for 70% of hires at Twitter as its Global Head of Revenue, G&A (Corporate Functions), University, and Diversity Recruiting, and led Goldman Sachs’ diversity talent acquisition strategy across 15 functions as its Vice President of Global Diversity Talent Acquisition.”

David Brailer is joining Cigna, the big health insurer, as executive vice president and chief health officer.


John Mulligan, founder and CEO, Good Therapeutics

Seattle-based Good Therapeutics agreed to be acquired by Roche for $250 million upfront cash, plus additional milestone payments that weren’t disclosed. The transaction means Roche will take over a PD-1 regulated IL-2 program for the treatment of cancer. Good Therapeutics’ molecules are intended to be active only when an antibody sensor has bound its target, in order to avoid systemic immune activation that has hampered other efforts. The startup was founded and led by CEO John Mulligan since 2016. Codon Capital, RiverVest Venture Partners, 3×5 Partners, Roche Venture Fund, and Digitalis Ventures were among its early backers. Mulligan and team are now moving on to further applications of the technology at a new company called Bonum Therapeutics.

Stamford, Conn.-based SpringWorks Therapeutics secured a $75 million equity investment from GSK as part of an expanded collaboration to develop nirogacestat, a gamma secretase inhibitor to be given in combination with in belantamab mafodotin-blmf (Blenrep), GSK’s antibody-drug conjugate directed against B-cell maturation antigen (BCMA). The companies have been working together since 2019.

San Diego-based Equillium, the developer of treatments for autoimmune and inflammatory disorders, agreed to acquire Metacrine in an all-stock merger. The deal brings an additional $33 million of cash to Equillium.

Data That Mattered

Regeneron reported on a pair of Phase III clinical trials with a new dosing regimen with its vascular endothelial growth factor inhibitor for the treatment of diabetic macular edema and the wet form of age-related macular degeneration. Researchers found that an 8-milligram dose of aflibercept could successfully be given to patients on a 12-week and 16-week dosing schedule. The company currently markets a 2-milligram dose of the protein drug under the brand name Eylea, on an 8-week schedule. The higher dose yielded similar results on safety and efficacy, and allowed more than 90 percent of patients to stay on treatment without adjusting the dose or schedule. That’s important because the treatment is administered as an injection into the eye. Not only are fewer injections more convenient for patients and physicians, but a new formulation of afilbercept means that Regeneron should be able to extend the life cycle of the aging Eylea franchise. That one product generated $9.4 billion in worldwide sales in 2021 – more than 58 percent of Regeneron’s total revenue. Shares of Regeneron surged 19 percent to $708.85 on the news.

Regeneron’s other major product, besides the REGEN-COV therapeutic antibody cocktail, also produced more clinical data to potentially expand its use to a new patient population. The IL-4 and IL-13 inhibitor dupilumab (Dupixent) met the primary endpoint in the second of two Phase III studies looking at patients with uncontrolled prurigo nodularis – a disease of intense itching and skin lesions. About 60 percent of patients on the Regeneron drug achieved a clinically meaningful reduction in itching from baseline, compared with 18 percent on placebo, according to data presented at the European Academy of Dermatology and Venereology meeting. About half of patients achieved clear or near clear skin on the new drug. Dupixent generated $6.2 billion in worldwide sales in 2021, and some analysts estimate it could exceed $15 billion a year in peak sales.

San Diego-based Mirati Therapeutics reported on an abstract of data to be presented for its KRASG12C inhibitor next week at the European Society for Medical Oncology (ESMO) meeting. Mirati enrolled 44 patients with colorectal cancer, and who have KRASG12C mutations, on twice-daily adagrasib monotherapy. Researchers found that 8 of 43 evaluable patients (19 percent) had their tumors shrink by half or more. Median duration of response was 4.3 months. Response rates improved in the cohort that received the Mirati treatment in combo with cetuximab. Almost half of patients – 13 of 28 – on the combo treatment had an objective tumor response, and the median duration of response was 7.6 months. Shares of Mirati gained 5 percent on the news.

Researchers working with Cambridge, Mass.-based Relay Therapeutics reported some striking results with an FGFR2 inhibitor for patients newly diagnosed with cholangiocarcinoma who have FGFR gene fusions or rearrangements. Researchers said 15 of 17 patients (88 percent) with this rare cancer who got Relay’s RLY-4008 in the recommended Phase 2 dose had their tumors shrink by half or more. Previous treatments have achieved that level of efficacy for about 20 to 40 percent of patients, with a duration of response of 5-9 months, researchers said. (Late-Breaking Abstract 12, ESMO). Relay’s stock shot up 24 percent on the news to close at $31.37. The company plans to discuss the results in a conference call at 8 am ET Sept. 12.

Gilead Sciences reported that its Trop-2 directed antibody-drug conjugate for hormone-positive, HER2-negative metastatic breast cancer was able to show a survival benefit. Patients on the Gilead drug a median time of 14.4 months, compared with 11.2 months for patients on physician’s choice of chemotherapy in the Tropics-2 study. The data on the key secondary endpoint are being presented at ESMO on Friday. Gilead already presented positive results on progression-free survival, the primary endpoint, at ASCO earlier this year – but they were overshadowed by the Daiichi Sankyo antibody-drug conjugate directed against HER2, marketed as Enhertu.

Cambridge, Mass.-based Alnylam Pharmaceuticals released details from the Apollo-B study of patisiran (Onpattro) for the treatment of transthyretin-mediated amyloidosis (ATTR) with cardiomyopathy. The positive top-line result was previously announced in August. It is the big new indication that builds on Alnylam’s initial market of ATTR patients with polyneuropathy. Researchers said ATTR patients with cardiomyopathy on the Alnylam drug were able to walk a median of 14.7 meters farther than those on placebo after 12 months of study. The overall decline in walking distance was about 8 meters for patients on the Alnylam drug after one year, compared with a 21-meter decline for patients on the placebo. Patients on the Alnylam drug also scored higher on a quality of life questionnaire. The company said it plans to submit an application to the FDA in late 2022 to start marketing to the additional patient population. Shares of Alnylam gained 8 percent on the news, closing at $226.30.

Carlsbad, Calif.-based Ionis Pharmaceuticals, a competitor to Alnylam in the ATTR field, reported on interim Phase III results with eplontersen, a next-generation self-administered antisense oligonucleotide drug. The drug was able to reduce TTR in the bloodstream by 81 percent, and patients reported an improvement in neuropathy symptoms, and quality of life, at the 35-week analysis. The study enrolled patients with hereditary transthyretin-mediated amyloidosis, and with polyneuropathy. A separate study is evaluating patients with cardiomyopathy.


Designing Gene Circuits For Cell Therapies: Tim Lu on The Long Run

Today’s guest on The Long Run is Tim Lu.

Tim is the co-founder and CEO of South San Francisco-based Senti Biosciences.

Tim Lu, co-founder and CEO, Senti Biosciences

Senti is working to develop gene circuits for cell therapies. This is about reprogramming cell therapies with precise genetic instructions on what to do in certain circumstances. The code essentially can tell the cell to kill tumor cells with a certain molecular marker on them, while sparing other cells that carry a particular molecular signature.

The first-generation cell therapies have delivered some extraordinary results for patients with cancer, but they also have some limitations. If Senti and others in the cell reprogramming world are successful, they could take cell therapies to a new level of safety and efficacy.

Tim and his colleagues have been working on gene circuits for a long time, dating back to his time on the faculty at MIT. He left that esteemed academic institution to go to work full-time on turning this research work into cell therapies that will someday hopefully help patients with cancer.

Senti’s work is still very early stage. It’s all preclinical. But it plans to seek clearance from the FDA to begin its first clinical trial, for patients with acute myeloid leukemia, in 2023.

Tim, like many biotech entrepreneurs, is the son of immigrants. His story starts there and takes a few interesting turns before getting to his current chapter, running a startup company. I think you’ll enjoy hearing about the person and the science.

Now before we get started, a word from the sponsor of The Long Run.

Calgary is home to more than 120 life sciences companies, from emerging startups to established firms. With this critical mass of research, technical talent and expertise, the city is an active hub for life sciences innovation.

Technologies homegrown in Calgary are changing the face of healthcare. Syantra is revolutionizing breast cancer detection using artificial intelligence-derived algorithms. NanoTess is harnessing the power of nanotechnology to tackle chronic wounds and skin conditions. And this is only the beginning. Calgary’s life sciences sector is projected to spend $428 million on digital transformation by 2024.

If you’re a bright mind or bright company solving global health challenges, Calgary is the place for you. 

Take a closer look at why at calgarylifesciences.com

Now, please join me and Tim Lu on The Long Run.


Seek Truth and Kill Quickly: Learning the Hard Way About Biotech Startups

Alex Harding, MD, senior vice president, Remix Therapeutics; internal medicine physician, MGH.

Consider this: A department chair at a major academic medical center presents compelling data, implicating the role of a new pathway in sleep apnea.

You immediately see an opportunity to make an impact on a prevalent disease. It’s a therapeutic arena with no pharmacologic therapy; the best we can do is advise patients to strap a CPAP machine to their face that blows air down their throat all night. Unsurprisingly, compliance rates with this standard of care are abysmal. A Nobel Prize winner has collaborated with the professor on several of the foundational papers.

This is an opportunity to help a substantial population of patients with major morbidities from their disease, to explore a novel target with well-characterized physiology, and to build an exciting biotech startup.

What do you do? Join part-time or full-time? Raise a $2 million seed financing or $50 million?

Read More


Arch Bets $50M on Vilya, Baker Lab Spinout to Make Targeted, Oral Peptide Drugs

David Baker and colleagues at the University of Washington are wizards of designing proteins on computers, from scratch, with optimized properties that often aren’t found in nature. They have been dazzling fellow scientists with a series of achievements over the past decade, and they’ve spun out a handful of companies with applications in mind for therapeutics, diagnostics, and vaccines.

Arch Venture Partners has watched from a cool distance. The top VC firm in Seattle hasn’t invested in any Baker Lab spinouts until now.

Read More


A Leap for Open Science, Fauci Exits NIH, & Bivalent Boosters on Deck

Luke Timmerman, founder & editor, Timmerman Report

Free the Data

The White House Office of Science and Technology Policy outlined a new policy that federally funded agencies update their public access policies ASAP to make sure that publications, and data, based on research conducted with federal grants is made freely available to the public. The deadline to get in compliance is Dec. 31, 2025. This will make it easier for people to learn and share, even if they don’t have access to expensive subscription journals or well-financed university libraries. It’s a win for science, a win for the public, and a loss for private sector publishers like Elsevier. This will remove some sand from the gears of knowledge generators.

This policy is about 10 years in the making. As someone who attended the Biden Cancer Summit in 2017 and 2018, I’m aware this is a longstanding interest of President Biden. After his son Beau died of cancer, he convened top scientists, businesspeople and policymakers to ask about the roadblocks that slow down and interfere with progress. A lack of open access to scientific papers and a lack of data sharing ranked high on the list. I’m glad to see this come to fruition after so many years. It’s an obscure policy that won’t resonate on the Interwebs or woo new voters, but it will have broadly positive implications for biomedical research, climate research, and the development of renewable energy sources. I expect it will also make it easier for young people to engage with science, especially young people from traditionally underrepresented backgrounds. (Full memo by Alondra Nelson, director of the White House Office of Science and Technology Policy).

A Legend Moves On

Anthony Fauci, the tireless physician-scientist who has led the National Institute for Allergy and Infectious Disease since 1984, announced he’s stepping down from that job at the end of the year. Fauci, 81, is retiring with a legacy of accomplishment in science and government. By his early 40s, he was a successful B-cell immunologist, publishing widely cited papers when immunology was still in very early days. He then moved up to NIAID leadership, guiding public health strategy on all kinds of infectious bugs, starting with HIV in the 1980s and extending all the way through SARS-CoV-2 and Monkeypox in the 2020s. He has earned the respect of the scientific community for his brilliance, the trust of many millions of citizens for his clear, calm science communication style on TV, and worrisome threats and demonization from people who have been radicalized against him. It’s one thing to have honest disagreements about policy, and it’s another to let those disagreements descend into threats of violence. Read the story about his retirement in the Washington Post, and a gracious tribute from HIV/AIDS activist Gregg Gonsalves, a professor of public health at Yale University, in the New York Times.

While Fauci may technically be leaving government service, when I saw him at an event at the Fred Hutch Cancer Center in Seattle earlier this month, he had plenty of vim and vigor left in the tank. He was clearly energized by meeting with summer interns who are part of a generation of young people inspired to pursue careers in science and medicine because of the leadership role he played in the COVID-19 public health crisis. He’ll continue doing that work, and is certainly capable of commenting about the infectious disease issues of the day in an intelligent way – and perhaps without the political constraints that have required him to adhere to a certain level of diplomacy.

If you read one scientific thing this weekend, I urge you to read an article Fauci co-authored with NIH colleague David Morens in August 2020 for Cell. This piece should have gotten much more public discussion than it did at the time of publication. It’s about all of the converging, long-term forces that gave rise to SARS-CoV-2 and which likely will seed more pandemics of the future. Think about the mass migration of people from rural areas to cities, the globally interconnected economy that now includes China (home to one-fifth of all people on Earth). Think about the rise of mass industrial livestock agriculture, and its proximity to people — which creates ample opportunity for spillover events. The question after reading this article ought to be – what are we doing to better prepare ourselves to live in an Age of Pandemics? Fauci is, and will remain, one of the world’s leading thinkers on this important topic, whether he remains a visible public figure or not. We would be wise to listen carefully to what he and Morens and other serious thinkers on the subject have to say.

Science Policy

FDA commissioner Robert Califf outlined a few concerns and policy priorities in a piece aimed at clinicians who read JAMA. The gist: the US is doing poorly against COVID. We need to continue pushing for more equitable distribution of vaccines and other resources. Misinformation is dangerous and alarming in its spread. Drug overdoses are a serious and growing problem. Tobacco remains a stubborn thorn in the side of public health. We need to keep doing more to speed up advances against cancer, genetic diseases. We need to do a lot more to improve pandemic surveillance systems. A lot of heavy lifting is required, inside the FDA and in the clinical and industrial communities. (Full article in JAMA, Aug. 22).


Cambridge, Mass.-based Orna Therapeutics struck a huge partnership with Merck. The startup, developing circular RNA therapies that build on the success with linear mRNA, secured $150 million in upfront cash, plus a $100 million investment from Merck in the company’s Series B financing. Total deal value is more than $3.5 billion when counting all potential milestones for the developing of vaccines and therapies.

Wayne, Penn.-based Teleflex agreed to acquire Standard Bariatrics, the developer of a stapling technology for bariatric surgery, for $170 million at closing and $130 million in milestones.

Switzerland-based Alcon, the eye care company, said it agreed to acquire Durham, NC-based Aerie Pharmaceuticals for $15.25 a share, or $770 million. Aerie is a developer of treatments for ophthalmic diseases, and has a couple of marketed once-daily eye drops for lowering elevated intraocular pressure.

Novartis said it’s spinning out its Sandoz generic drug unit.

Regulatory Action

The FDA signaled that it’s preparing to authorize bivalent booster vaccinations against the Omicron BA.5 variant shortly after Labor Day. Peter Marks, the head of the FDA’s Center for Biologics Evaluation and Research, told the New York Times he’s “extremely confident” in the data supporting the updated vaccines. Both of the mRNA vaccine makers, Moderna and Pfizer/BioNTech, have turned in applications for the vaccines designed to match the viral variants currently in circulation.

Gilead Sciences secured European approval for lenacapavir (Sunlenca), the first HIV treatment that can be given twice a year. It’s for patients with multi-drug resistant HIV. The FDA is reviewing an application for the drug, and has a Dec. 27, 2022 legal deadline to complete the review.

Philadelphia-based Century Therapeutics received the green light from the FDA to start a clinical trial with its CAR-NK cell therapy that targets CD19 for B-cell malignancies, which was derived from induced pluripotent stem cells, and which contains six gene edits.

The FDA granted clearance to J&J and AbbVie’s ibrutinib (Imbruvica) for pediatric patients with graft-versus-host disease. It’s the 12th approval for the BTK inhibitor across seven indications.

Cambridge, Mass.-based Foghorn Therapeutics, the developer of drugs that target the chromatin regulatory system, said its Phase I study of a drug candidate for acute myeloid leukemia and myelodysplastic syndrome has gone on full clinical hold. It had previously been on partial clinical hold, but that changed after the company submitted more data that pointed to “additional suspected cases of fatal differentiation syndrome believed to be associated with FHD-286.”

Takeda Pharmaceuticals secured regulatory clearance in Indonesia, the fourth most-populated country in the world behind China, India and the United States, for its dengue vaccine Qdenga. In the first half of 2022, Indonesia reported 63,000 cases and nearly 600 deaths from dengue, a mosquito-borne virus

Data That Mattered

Pfizer and BioNTech released follow up data on their COVID vaccine for little kids ages 6 months through age 4. It showed that a three-dose regimen delivered consistent vaccine efficacy of more than 70 percent, even though testing was conducted during a period when Omicron BA.2 was largely the variant in circulation. This is from a tiny 3 microgram dose for the smallest kids – far less than the 10 microgram used in kids 5-11 and 30 micrograms used for adults. Adverse events were mild to moderate, and the safety profile was similar to placebo, the companies said. This ought to provide more assurance of a positive risk-benefit profile for parents of kids in this age group.

Pfizer said it hit the primary endpoint of a Phase III clinical trial with a vaccine for respiratory syncytial virus (RSV). Data will be presented at an upcoming medical meeting. If it passes muster with the FDA, it would be the first vaccine for this respiratory viral scourge.

A research team from Israel pored over data from adults over 40 who got Pfizer’s nirmatrelvir (Paxlovid) for treatment of COVID-19, and found a benefit for people over age 65, but no benefit for the younger adults. New England Journal of Medicine. Aug. 24. (Ronen Arbel et al).

Novartis said it’s halting dosing in a Phase II trial with branaplam for Huntington’s disease after seeing signs that it might be causing peripheral neuropathy.


Cambridge, Mass.-based Aktis Oncology secured an $84 million Series A extension, which included new investors Cowen Healthcare Investements and MRL Ventures, among others. The company is developing targeted radiopharmaceuticals for cancer. (TR coverage).

South San Francisco-based 3T Biosciences secured a $40 million Series A financing led by Westlake Village BioPartners. It’s working to discover new targets for bispecific T-cell engaging cancer drugs.

Seattle-based Monod Bio raised $25 million in seed financing. It’s a spinout from the Institute for Protein Design at the University of Washington working on diagnostic biosensors that emit light in the presence specific molecules. Matrix Capital led.

New York-based Ordaos raised $5 million in seed financing to discover mini-protein drugs.

San Mateo, Calif.-based Bluejay Therapeutics, the developer of a treatment for chronic hepatitis B, raised $41 million in a Series B financing led by Arkin Bio Ventures.

Belgium-based eTheRNA, an mRNA therapeutics company, raised 39 million Euros in a Series B2 financing. Novalis LifeSciences led. It’s an investment vehicle led by Marijn Dekkers, former CEO of Bayer and Thermo Fisher Scientific.

Watertown, Mass.-based PanTher Therapeutics said it received a $14.2 million grant from the Cancer Prevention and Research Institute of Texas (CPRIT) to advance work on an implantable treatment for pancreatic cancer.

Investing in US Manufacturing

The University of Pittsburgh and ElevateBio entered into a 30-year agreement to bring biomanufacturing to Pittsburgh. The deal is enabled in part by a $100 million grant from the Richard King Mellon Foundation. The agreement calls for ElevateBio to bring one of its BaseCamp facilities to Pittsburgh, where it will have the people and technology to do process development and GMP manufacturing, and which includes ElevateBio’s enabling technology for gene editing, induced pluripotent stem cell (iPSC) and cell, vector, and protein engineering. The project is expected to generate 170 permanent full-time jobs, 900 construction jobs, and 360 off-site support jobs.

South San Francisco-based Sonoma Biotherapeutics, the developer of T-regulatory cell therapies for autoimmunity, said it’s investing in an 83,000-square-foot cell therapy R&D and manufacturing center in Seattle. It’s along the Elliott Bay waterfront.

Thermo Fisher Scientific said it’s opening up a 300,000 square foot facility in Plainville, Mass. to manufacture viral vectors for gene therapies. Separately, Thermo Fisher said it opened a $105 million, 400,000 square foot facility in Nashville, Tennessee to make materials for vaccines and new therapies for cancer and other diseases.

The California Institute for Regenerative Medicine (CIRM), the state agency that has funded 81 clinical trials and has 161 active regenerative medicine projects, said it’s adding Resilience and ElevateBio to its network of industry partners. Each firm has expertise in advanced manufacturing that scientists in the CIRM network can lean on.

Personnel File

Sarah Grant joined Cambridge, Mass.-based Celsius Therapeutics as chief medical officer. The company uses single-cell RNA sequencing to develop treatments for cancer and autoimmunity. Grant previously worked for Novartis.

Sarah Grant, chief medical officer, Celsius Therapeutics

Cambridge, Mass.-based Alnylam Pharmaceuticals named Elliott Sigal to its board of directors. He’s the former chief scientific officer of Bristol Myers Squibb, and currently co-chairs the SAB at Amgen.

Fort Myers, Florida-based Neogenomics hired Chris Smith as CEO. He previously was CEO of Ortho Clinical Diagnostics.

Charlottesville, Virginia-based HemoShear Therapeutics named Brian Wamhoff as interim CEO and Robert Gould as board chair. Wamhoff is a founder of the company.

St. Louis and San Diego-based Wugen named Natalie Mount as its board chair. She was CEO of Adaptate Biotherapeutics until its acquisition by Takeda.

Austin, Tex.-based Aeglea Biotherapeutics said CEO Anthony Quinn has stepped down, effective immediately, and is being replaced by general counsel Jim Kastenmayer on an interim basis. The company is also cutting 25 percent of its workforce to extend its cash runway into the fourth quarter of 2023.

South San Francisco-based Atara Biotherapeutics, a developer of T-cell immunotherapies, said it’s cutting 20 percent of its workforce.

Frazier Life Sciences added Annette Doherty as a senior advisor. She was previously an SVP of product development and supply at GSK.

Singapore-based Tessa Therapeutics, a cell therapy company, hired Thomas Willemsen as CEO. He was previously senior vice president of Asia/Pacific for Takeda Pharmaceuticals.

Emeryville, Calif.-based Kyverna Therapeutics, the developer of engineered T cells for autoimmunity, hired Tom Van Blarcom as senior vice president of research. He comes from Allogene Therapeutics.


A Life in Autoimmune Drug Discovery: Jo Viney on The Long Run

Today’s guest on The Long Run is Jo Viney.

She is the CEO of Watertown, Mass.-based Seismic Therapeutic. Seismic is working to discover biologic drugs for autoimmune disease. It aims to speed up the process by using machine learning on key aspects – starting with structural biology and including engineering of the protein drugs themselves.

Jo Viney, co-founder, president and CEO, Seismic Therapeutic

Jo has a long track record in this field. She was previously chief scientific officer of Pandion Therapeutics, a startup acquired by Merck for $1.85 billion in February 2021. Before that, she worked at Biogen, Amgen, Immunex and Genentech.

In this conversation, Jo talks about immigrating from the UK, how she found a career path in industry, and some key insights on how she thinks about building a startup with a creative culture.

Now before we get started, a word from the sponsors of The Long Run.

Calgary is home to more than 120 life sciences companies, from emerging startups to established firms. With this critical mass of research, technical talent and expertise, the city is an active hub for life sciences innovation.

Technologies homegrown in Calgary are changing the face of healthcare. Syantra is revolutionizing breast cancer detection using artificial intelligence-derived algorithms. NanoTess is harnessing the power of nanotechnology to tackle chronic wounds and skin conditions. And this is only the beginning. Calgary’s life sciences sector is projected to spend $428 million on digital transformation by 2024.

If you’re a bright mind or bright company solving global health challenges, Calgary is the place for you. 

Take a closer look at why at calgarylifesciences.com

For Bensalem Township in Pennsylvania, going a step beyond meant taking the word ‘serial’ out of crime, thanks to DNA analysis technology. Before the introduction of this technology, processing the sample of a suspect took 18 months. But with the dedicated efforts of Director Fred Harran and Thermo Fisher Scientific’s RapidHIT ID analysis system, it now takes only 90 minutes – meaning offenders can be caught and put behind bars before they have a chance to become repeat offenders. It’s also helped prove the innocence of 16 people in the last five years.

To watch Director Harran’s story, visit www.thermofisher.com/bensalem-DNA-analysis

Now, please join me and Jo Viney on The Long Run.