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The Pacific Northwest is home to beautiful Seattle, Washington.  Anchored by companies such as Starbuck’s Coffee, Pike’s Place Market and the iconic Space Needle, it is a must-see destination in the U.S.  It is also a growing biotech hub.  With the prestigious Fred Hutchinson Cancer Center, Seattle Children’s and the University of WA incubating technologies, the list of innovative biotech companies continues to grow.

The Seattle-Tacoma-Bellevue triangle is the fastest growing area for life sciences employment, closely followed by the Atlanta area and the Orlando-Kissimmee-Sanford triangle, according to a new report from JLL. (EndPoint News, March, 2021).

Here are a few interesting and innovative companies to watch:

Chinook Therapeutics, Seattle, is a clinical-stage biopharmaceutical company discovering, developing and commercializing precision medicines for rare, severe chronic kidney diseases.  This includes IgA nephropathy (IgAN), other proteinuric glomerular diseases and hyperoxalurias.

Blaze Biosicence, Seattle, has developed the first Tumor Paint product candidate, BLZ-100, for cancer surgery in multiple solid tumor types. BLZ-100 is a drug administered by IV injection that circulates within the body and “lights up” cancer cells. It consists of a peptide, which binds and internalizes into cancer cells, and a fluorescent dye, which emits light in the near-infrared range.

Sana Biotechnology, Seattle, imagines a world in which it is possible to cure genetically inherited diseases and modify genes to create better therapeutics for acquired diseases. Sana’s in vivo Cell Engineering platform aims to provide solutions for patients that current gene therapies cannot address. Success will require creative science, experienced product and clinical development strategies, and aggregating important technologies.

Icosovax, Seattle, is using novel Virus-like Particles (VLPs) to enable high-density, multivalent display of antigens in a manner that closely resembles viruses. This induces stronger and more durable immunological responses compared to traditional soluble antigens. VLPs contain no genetic material, so they are non-infectious and can provide a safer alternative to live-attenuated or inactivated vaccines.

Mozart Therapeutics, Seattle, is developing CD8 T cell immune modulators for the treatment of autoimmune diseases. It specifically targets a subset of T lymphocytes, delaying the onset and ameliorate targeted autoimmune diseases, enabling patients to get modern ways for treatment.

Athira Pharma, Bothell, is focused on improving neuronal health by promoting natural repair systems for Neurodegenerative diseases – like Alzheimer’s and Parkinson’s.  Their therapeutic approach involves small molecule treatments designed to work by targeting specific, naturally occurring repair mechanisms. One of our initial targets is HGF/MET, a critical repair pathway we are seeking to promote with our lead therapeutic candidate, ATH-1017.  Boosting this pathway can help improve brain health and function.

Southern California is known for warm weather, sunny beaches and as a desirable residence for the rich and famous.  Those in the scientific community also know it as a hub for biotechnology.  Centers of excellence in higher education, well equipped biotech incubators and an experienced workforce come together to build a framework for success.

According to a Biocom 2020 Impact Report, the life science industry in California generated $372 billion annually and provided 1.4M Jobs. In addition, California also received the most research funding from National Institutes of Health (NIH) of any state in the country, a total of $4.59 billion in the 2019 fiscal year.

I have highlighted a few interesting companies in Southern California below.  Narrowing the list down to 5 companies was not easy, and I may have to write a version 2. 

Viracta Therapeutics, San Diego, CA, is advancing new medicines for the treatment of virus-associated malignancies, such as Epstein-Barr virus (EBV)-positive lymphoid malignancies.

Aadi Biosciences, Pacific Palisades, CA, is focused on development and commercialization of precision medicines targeted to rare genomic alteration-driven cancers. 

NanoSmart Pharmaceuticals, Laguna Hills, CA, has multiple platform technologies to enable the next generation of biopharmaceuticals for treating all solid-tumor cancers, including: breast, prostate, lung, and rare childhood cancers.

Bionaut Laboratories, Los Angeles, CA, created a Bionaut™, a new treatment modality that uses microscale robots to deliver biologics or small molecule therapies locally to targeted disease areas to help patients suffering from severe brain disorders, such as gliomas and Parkinson’s Disease.

Onchilles Pharma, San Diego, CA, is developing a pipeline of first-in-class biologic and small molecule drug candidates that have the potential to be broadly effective against a wide range of cancers. 

This impressive list demonstrates the exciting developments in biotechnology which will ultimately result in living longer and healthier lives.

In the event you are moving to Phase III but have not engaged Commercial expertise, make sure you have done these top three initiatives before finalizing the Phase III design.

1. Get Payor and MD/KOL input on endpoints
2. Get Health Economics and Outcomes Research (HEOR) input on endpoints.
3. Establish a commercialization vision & roadmap for planning purposes and to impress investors.

If you need help assessing your commercial strategy, we can help.

Learn more on our website, www.bridge1.net.

A recurring problem at Smallco emerging biotech companies is that the Executives lack commercialization expertise and they don’t have the foresight to seek it early and /or don’t place the right value on it.  In essence, they don’t know what they don’t know. Frankly, many don’t care – they believe “cool science” will sell it in the marketplace.  That’s a mistake.

A modest but essential number of commercial actions need to be taken in Phase I, more so in Phase II and full-blown commercialization should be early in Phase III, (stage gated). Note, with gene therapy companies today, moving from IND to pivotal trial readout in five years, the need for early commercialization has accelerated.

There are highly effective and cost-efficient alternatives to hiring full time Commercial and Medial Affairs leadership too early.  Interim executives can identify, prioritize and execute Phase-specific commercial activities critical for launch.

Investing in this knowledge early on in the process helps ensure a smooth and organized commercialization process. 

For more about those alternatives, see our website at www.bridge1.net.

When we think of Florida, we think beautiful sandy beaches, dream trips to Walt Disney World and scrumptious seafood dinners.  However, there is an industry we are overlooking.  Looking at the vast array of biotech companies emerging throughout the state, we may need to add “Biotech Leader” to describe this boom. 

Florida has an active and growing biotechnology trade association, BioFlorida.  BioFlorida’s member driven initiatives provide a strong business climate for the advancement of innovative products and technology that improve lives and promote economic benefits to the state. (Bioflorida.com).

Thanks to thebiopharmguy.com, I counted 260 small biotech companies that call the Sunshine State home.  I have chosen the top companies to watch in 2021.

Cyclo Therapeutics in Gainesville, FL has developed Trappsol® Cyclo™, an orphan drug designated product in the United States and Europe, in ongoing formal clinical trials for Niemann-Pick Disease Type C, and an expected IND filing in H2 2021 for Alzheimer’s Disease.

Scion BioMedical in Miami, FLhas developed cost-effective solutions designed to support advanced healing and wound care using a proprietary bioactive compound. This naturally occurring product base is proven to effectively adhere and absorb, while also acting as a natural hemostatic agent.

Jupiter Orphan Therapeutics in Jupiter, FL has developed a unique platform product, JOTROL™​, a patented reformulation of the well-known “wonder” medication resveratrol. JOTROL™​ is the first and only product that delivers resveratrol in a highly bioavailable form which can effectively cross the blood brain barrier allowing for the treatment of the central nervous system. 

REGENTYS™ in Miami Lakes, FLis a development stage, regenerative medicine company focused on developing treatments for patients with gastrointestinal (GI) disorders.  Their ExtraCellular Matrix Hydrogel (ECMH), is a first-in-class, non-pharmacologic, non-surgical treatment option for patients suffering from ulcerative colitis.

Allodynic Therapeutics in North Miami, FL is a clinical-stage specialty biopharma company developing a naltrexone and acetaminophen combination for migraine and neuropathic pain treatment. Their product is potentially a non-addictive alternative to opioid medications, helping to end the opioid crisis. 

INHIBITOR Therapeutics in Tampa, FL, developed SUBA-Itraconazole, the company’s lead product candidate, which is a patented, oral formulation of the currently FDA approved and marketed, well-understood, anti-fungal drug, itraconazole, which, when studied to treat patients with cancer, has demonstrated activity against multiple tumor types. INHIBITOR is developing SUBA-Itraconazole for late-stage, metastatic castrate-resistant prostate cancer (mCRPC) and late-stage non-squamous non-small cell lung cancer (NSCLC).

Do you have any additions?   What is your top biotech to watch?

Continuing my series on cool cities for biotech, I take you to Scandinavia.  Copenhagen, the capital city of Denmark, has emerged as a growing biotech hub.  In fact, Nordic Life Science News named Denmark the best country in Europe for biotech research and development in 2019.

According to https://investindk.com/, there’s a reason why Denmark is the home of major international pharmaceutical companies such as Novo Nordisk, Lundbeck, LEO Pharma and ALK. It all started back in the early 1900s, when the first Danish pharma companies built the foundations for the thriving life science sector.

These strong pillars have planted the seeds for new, innovative companies to grow.  Some of the standouts include:

IO Biotech has a unique and novel technology platform, T-win®, for developing immune modulating anti-cancer therapies.  The technology is unique in that the compounds directly target tumor cells and tumor micro environment and initiate a dynamic process of activating the host immune system, which can then be exploited by concurrent or subsequent therapies.

Pokeacell is working on a new type of personalized T cell immunotherapy, which boosts the patient’s own immune system so that it can defeat cancer attacks itself.

Meabco A/S has developed a low toxicity novel cancer drug for pancreatic and metastatic breast cancer called BP-C1.

Kariya Pharma has developed a first-in-class, dual incretion receptor co-agonist to slow the progression of Parkinson’s Disease.

Avilex Pharm is producing novel inhibitors of PDZ domain proteins to treat ischemic stroke.

Keep an eye on Denmark.  It is fast becoming a worldwide leader in clinical trials due to its hospital network and ample patient population.  Biotech incubators and generous funding bodies will keep Copenhagen’s biotech community thriving in the future.

I recently had a dear friend discover that she had ovarian cancer.  Not long ago, that diagnosis would have had a very defined outcome.  However, the doctor talked about surgery and asked her to get genetic testing to see if the cancer was dictated by BRCA1.  Why did that matter?  Because sequencing her genome would guide the physicians towards her best and most beneficial care.

She had the test performed by INVATE.  They offer a variety of genetic testing for inherited cancers, heart disease and carrier screening.  The power of genetic sequencing is having a real and positive effect on patients.

After her second surgery, she learned that the tumors had been sequenced.  Why?  To learn their individual genetic makeup to predict the efficacy of different treatments such as chemotherapies and immuno-therapies.

The power of DNA sequencing has definitely made its way to doctor-patient decision making.  It was not long ago when there were standard treatments, one size fits most with a check in down the road to see if the therapy was working.  With the democratization of DNA sequencing via benchtop devices such as Thermo Fisher’s ION Torrent, sequencing has become more accessible and more affordable.

Enter CRISPR – Clustered regularly interspaced short palindromic repeats.  Scientists now have the ability to edit genomic information by excising the damaged DNA and repairing it.  By “editing” damaged DNA, a new healthy protein can potentially be expressed.  Some of the interesting biotech companies putting CRISPR to work for patients are:

Intellia Therapeutics with a pipeline for Hemophelia A and B, Transthyretin Amyloidosis and Hereditary Angioedema.

Editas Medicine focusing on Leber CongenitalAmaurosis 10 (LCA10), UsherSyndrome 2a (USH2A) and Autosomal DominantRetinitis Pigmentosa 4(RP4).

CRISPR Therapeutics with products for Hemoglobinopathies and Immuno-Oncology

Sangamo Therapeutics who have Phase 2/3 clinical trials for hemophelia, solid organ transplant, Sickle Cell and Fabry diseases. Watson and Crick knew they had discovered something powerful with DNA, but even they could have never imagined the powerful tools to come and the potential positive impact on patients and their quality of life. 

Recent IPO’s from the following list of biotech companies have provided blockbuster capital returns so one might ask, what do these A-list companies have in common?

A provocative & differentiated technology.
Experienced entrepreneurial leadership.

Recent examples include:

IPOs

• Silverback / Laura Shawler / $277m
• SEER / Omid Farokhzad / $250m
• Sigilon / Rogerio Vivaldi / $145m
• Kronos /Norbert Bischofberger / $288m IBO / $157m private placement
• Kinnate Bio / Nima Farzan / $276m
• Olema / Sean Bohen / $382m (B,C, IPO)
• Rocket Pharma / Gaurav Shah / $299m
• Ikena Oncology / Mark Manfredi / $144m

Mega A/B Rounds

• EQRx / Alexis Borisey / $700m (A, B)
• Scorpion / Adam Friedman / $270m (since Q1, 2020)
• Werewolf / Dan Hicklin / $72m (B)

The third thing in common is their X-Factor:  

They bring in commercial leadership early that execute the Phase specific commercial imperatives and develop defensible commercial valuations – those that stand up to diligence with the board and other industry experts in licensing discussions.  Their Commercial leadership also teach and align the Executive Team and Board on commercialization, (e.g. Market Access, Pricing & Reimbursement, Medical Education, KOL development etc.).

Take a closer look at all these companies – they have the science, the leadership and the Commercial X-factor in place.

Wanna be an A-lister too – contact us.

Anyone who has ever led a biotech product launch knows the importance of an accurate and defensible revenue forecast.  Having spent many years in the industry, I have seen the downsides of poor forecasting.  Whether the numbers are too high or too low, they can have a devastating impact especially on 1^st product to market-emerging biotech companies.  Some of the fallout of poor forecasting include:

• Missed expectations on Wall Street
• Revenue gaps to fund company expansion and new clinical development
• Changing the sales plan (especially revised upwards) with the Sales Force (ouch!)
• Over or under inventory of drug supply available for sale
• Manufacturing team facing expired stock or shortages

When developing a revenue forecast for a drug, it is important to build it from the ground up with defensible assumptions. And by “defensible”, we mean sourced information assembled in a logical format that other industry experts would deem credible and find difficult to refute.

Gather or develop these top three foundational elements of the forecast:

1. Market sizing based on expert epidemiology for each key geography that nets out “treatable patient populations”. Note, at Bridge, we rely on Tessellon inc. for our defensible epidemiology, (www.Tessellon.com, 636-299-0487).

2. Foundational health care professional (HCP) and payor market research.

3. Analyze the competitive landscape through the lens of today’s standard of care (SOC) and changes in the SOC that may occur by the time of your drug launch.

A thorough understanding of the data ensures you are making the right assumptions and if don’t have the commercial assessment / revenue forecasters in house – “rent” some experts.

Lastly, but as importantly, the launch forecast and a 5 year post launch forecast are used as an anchor for how large the Commercial & Medical Affairs organizations are (e.g. FTEs) and the “dollar spend” for all the commercial imperatives. What you do and how much you spend for a 5-year to peak revenue of $250m is very very different than what you do for a 5-year peak revenue of $1B. If your company is challenged by revenue forecasting and/or defining the size of the Commercial/Medical Affairs organization contact us.

Prior to COVID-19, we worked with a small European biotech company in need of key commercial and medical affairs leadership and a vision of what commercialization looked like. We mapped out all of the costs associated with commercial & medical affairs imperatives and headcount and worked closely with the Chief Financial Officer to complete a 3-year commercial budget. Due to COVID-19, the company was able to leverage our adaptive commercialization model to first pause all commercial work for six months and later continue commercial support through early 2021 when they onboarded the CCO.

To properly commercialize Phase II/Phase III drugs or therapies, one must be sure to have all the bases covered with an early vision of commercialization including the launch critical success factors. It is important to develop a roadmap with key milestones and deliverables. From pricing to reimbursement rates, there should be no stone left unturned in your precommercial planning and execution.

Using our adaptive model, a company can ensure that they are paying for the commercial deliverables that are most important to their present and future launch success. In addition, it can fill a knowledge gap at the organization. Instead of investing in a full time Chief Commercial Officer, a company can invest in areas important to their product(s) with less risk. Oftentimes, this comes with a cost savings of 50% or greater. We call this service an Interim Chief Commercial Officer, iCCO.

Looking at the activities needed for a comprehensive commercialization plan, one should cover the following:

• Defensible Commercial Assessments & Revenue Forecasting  
• Commercial & Medical Affairs Talent Acquisition & Onboarding
• Strategy Across Geographies
• Pricing and Reimbursements
• Competitive Landscape and Market Assessment
• Go-To-Market Strategies
  Epidemiology

Looking for assistance with your commercial vision & blueprint? We can show you how to get there with less risk.