Heidelberg Pharma is a clinical stage biotech company developing antibody drug conjugates (ADCs).

Its lead amanitin-based ADC product candidate, HDP-101, targets relapsed or refractory multiple myeloma. HDP-101 recently received orphan drug designation; it is currently in a phase I/IIa clinical trial, demonstrating first signs of clinical efficacy. 

The company is also expanding its therapeutic product pipeline with a further four programs across a variety of malignant hematologic and solid tumors.

To tell us about how HDP-101 works, and what Heidelberg Pharma is doing in the space, we had a conversation with the company’s CEO, Prof. Andreas Pahl.

00:55-02:05: About Heidelberg Pharma
02:05-03:41: What are antibody-drug conjugates?
03:41-05:27: Why are antibody-drug conjugates in the news currently?
05:27-06:15: What is big pharma’s involvement in the field?
06:15-07:04: Will there be more antibody-drug conjugate approvals?
07:04-08:42: What can antibody-drug conjugates be used to treat?
08:42-09:48: How do you avoid toxicity issues?
09:48-10:31: How important is the linker?
10:31-12:06: What is alpha-amanitin?
12:06-13:28: How do you make a toxin not toxic?
13:28-14:11: What tumor types can be targeted?
14:11-15:59: How do you discover a toxin can treat a disease?
15:59-17:38: How is alpha-amanitin delivered?
17:38-19:27: Is alpha-amanitin a monotherapy?
19:27-22:16: What clinical trials are you doing?
22:16-23:31: What are the next steps now you have orphan drug designation?
23:31-24:14: What are the timelines?
24:14-25:36: Where are antibody-drug conjugates headed?
25:36-27:14: Are AI and machine learning making an impact?

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Diamyd Medical develops precision medicine therapies for the prevention and treatment of Type 1 diabetes and LADA (latent autoimmune diabetes in adults). 

Diamyd is an antigen-specific immunomodulatory therapeutic for the preservation of endogenous insulin production. It has been granted Orphan Drug Designation in the U.S. as well as Fast Track Designation. 

DIAGNODE-3, a confirmatory phase 3 trial, uses injections into a superficial lymph node can to optimize the treatment response. 

This week on the podcast we have a conversation about treatments of type 1 diabetes, including the company’s development of Diamyd. Our guest is Anders Essen-Möller, founder and chairman of Diamyd Medical.

00:51-19:16: Diamyd Medical’s approach to Type 1 diabetes
19:16-21:03: Other approaches to tackling Type 1 diabetes
21:03-22:08: Keeping costs of Type 1 diabetes treatment low
22:08-22:53: Can Type 1 diabetes be cured?

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Rare diseases affect around 1 in 2,000 people. Many of the thousands of rare diseases are difficult to diagnose. Of the known rare diseases, about 80% have genetic causes.  

Leukodystrophies are a group of rare genetic disorders for which there is currently no curative therapy.

This week on the podcast we are discussing novel treatments of rare pediatric diseases in a conversation with Dan Williams, CEO, executive director and co-founder of Synaptix Bio. Synaptix Bio is working on developing treatments for severe leukodystrophies. 

The company has partnered with the Children’s Hospital of Philadelphia to take a TUBB4a leukodystrophy therapy to the clinic and beyond. 

01:41-05:08: About Synaptix Bio
05:08-07:07: What are rare diseases?
07:07-09:36: How do you develop treatments for rare diseases?
09:36-11:15: How many rare diseases have treatments?
11:15-13:32: What is the space looking like for companies looking to develop rare disease treatments?
13:32-14:10: How does the fact that many rare diseases are genetic affect drug development?
14:10-16:21: Do the low numbers of people with rare diseases affect funding and research? 
16:21-17:55: Are the drugs being developed useful in treating other diseases?
17:55-22:44: What is H-ABC?
22:44-23:25: Are there any treatments for H-ABC? 
23:25-25:15: Where is Synaptix Bio with clinical trials?
25-15-27:40: How do you design clinical trials for H-ABC with the number of patients being low?

This week’s podcast was sponsored by KBI Biopharma. Visit their facility in Geneva, Switzerland, on June 27, to join the discussion on solving biotherapeutic challenges. Register at kbibiopharma.com/conferences.

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Hearing loss is a huge – and growing – global challenge.

Using its OSPREY platform, UK-based Rinri Therapeutics is developing a portfolio of off-the-shelf regenerative cell therapy products to address sensorineural hearing loss, a significant global medical challenge. 

The company’s lead asset, Rincell-1, is designed to regenerate auditory neurons and provide a novel treatment for patients with severe-to-profound age-related hearing loss or auditory neuropathy. The company is looking to start first-in-human trials in 2025.

Today, on the podcast we have a conversation with Rinri CEO Simon Chandler about the different kinds of hearing loss, what’s being done to tackle them, and Rinri’s novel approach to treatments.

01:08-03:19: About Rinri Therapeutics
03:19-05:16: About hearing loss
05:16-07:31: Mental health and hearing loss
07:31-10:22: The different kinds of hearing loss
10:22-11:37: The importance of early intervention
11:37-13:46: What is Rinri doing to tackle hearing loss?
13:46-15:58: The difficulty of accessing the cochlea
15:58-19:12: Is your treatment a cure for hearing loss?
19:12-20:45: Would repeat treatment be necessary?
20:45-23:30: Are other companies working on hearing loss?
23:30-25:27: The cost of treating hearing loss
25:27-27:13: The economic costs of hearing loss
27:13-28:33: Rinri treatment timelines
28:33-30:15: The rest of Rinri’s pipeline

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June is myasthenia gravis awareness month. Myasthenia gravis is a chronic autoimmune disorder where antibodies destroy the communication between nerves and muscle, and this results in weakness of the skeletal muscles. 

It especially affects the voluntary muscles of the eyes, mouth, throat and/or limbs. It is most frequent in women from 20 and 30 and men aged 50 and older. 

To talk about the disease, how it is being treated, and what is in the pipeline for future treatments, we had a conversation with Dana Vigier, head of Central Eastern and Northern Europe at Alexion.

01:07-04:26: Background on Dr Dana Vigier
04:26-07:07: What is myasthenia gravis?
07:07-09:43: What is the prognosis for someone diagnosed with myasthenia gravis?
09:43-11:33: How effective are current treatments for myasthenia gravis?
11:33-12:56: Do all the myasthenia gravis drugs work in the same way? 
12:56-18:35: What R&D is taking place in myasthenia gravis?
18:35-21:14: Alexion and its work on myasthenia gravis
21:14-22:01: Myasthenia gravis clinical trials
22:01-23:58: The importance of myasthenia gravis awareness month
23:58-25:31: Would early diagnosis make a difference?
25:31-27:54: Transformation through new treatments

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German-headquartered Isarna Therapeutics is developing a portfolio of antisense therapies targeting an emerging therapeutic field in human biology – transforming growth factor beta (TGF-B) signaling.

Precise modulation of TGF-B pathways using antisense therapy may result in safer and more effective treatment options for a broad range of indications.

The company’s lead compound, ISTH0036, is in clinical development in ophthalmology.

On the podcast this week we chat with René Rückert, chief operating officer at Isarna Therapeutics, who explains TGF-B and antisense oligonucleotides’ role in tackling disease.

00:59-02:45: About Isarna Therapeutics
02:45-05:05: What is transforming growth factor beta?
05:05-06:16: Why is TGF a good target?
06:16-09:00: What are antisense oligonucleotides?
09:00-10:02: What are antisense oligonucleotides useful for treating?
10:02-11:21: What advantages do antisense oligonucleotides have?
11:21-13:00: How can antisense oligonucleotides be delivered?
13:00-14:58: Are antisense oligonucleotides still being researched?
14:58-16:31: Are there challenges with using antisense oligonucleotides?
16:31-16:56: Are many companies working on antisense oligonucleotides?
16:56-18:06: Are antisense oligonucleotides best used as a monotherapy or with other treatments?
18:06-20:42: What is Isarna’s pipeline?
20:42-22:47: How does your AMD treatment work?
22:47-23:50: What is the timeline for your treatment?
23:50-26:34: What can patients expect?

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Cytokinetics is a late-stage, specialty cardiovascular biopharmaceutical company focused on discovering, developing and commercializing first-in-class muscle activators and next-in-class muscle inhibitors as potential treatments for debilitating diseases in which cardiac muscle performance is compromised.

This week, we have a conversation with the CEO of Cytokinetics, Robert Blum, about the company’s aficamten, its next-in-class cardiac myosin inhibitor, prior to results from a phase 3 clinical trial in obstructive hypertrophic cardiomyopathy. 

00:54-02:32: About Cytokinetics
02:32-04:11: What are the current treatments for cardiovascular conditions?
04:11-05:44: About cardiovascular disease
05:44-08:59: What is aficamten?
08:59-09:12: How is it administered?
09:12-10:04: Where is aficamten on the path to commercialization?
10:04-12:06: What does this mean for patients?
12:06-12:42: What will you be presenting in Portugal?
12:42-16:06: Could aficamten have other applications?
16:06-16:50: What is CK-136?
16:50-18:02: What is omecamtiv mecarbil?
18:02-20:26: Cytokinetics’ business model

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Duchenne muscular dystrophy (DMD) is a genetic disorder with progressive muscle degeneration and weakness due to changes in the protein dystrophin, which helps keep muscle cells intact. 

There are few treatments for DMD, although recent advances mean that males – who are primarily affected by DMD – can now sometimes survive into their 30s.

More drugs are being developed in the fight against DMD, and this week we had a conversation with Frank Gleeson, CEO of Satellos Bioscience, about recent work on addressing DMD, including the company’s own work in the field.

00:47-02:01: About Satellos Bioscience
02:01-04:33: What is Duchenne muscular dystrophy?
04:33-05:43: What are the challenges in treating Duchenne muscular dystrophy?
05:43-09:08: What are the new treatments for Duchenne muscular dystrophy?  
09:08-11:20: What is your treatment for Duchenne muscular dystrophy?
11:20-13:42: Are there different approaches to treating Duchenne muscular dystrophy?
13:42-14:06: How is your treatment delivered?
14:06-17:32: How important is early intervention?
17:32-18:56: Where is Satellos at with clinical trials?
18:56-20:41: Preclinical trial results
20:41-21:44: Outreach to the Duchenne muscular dystrophy community
21:44-23:12: Is a cure for Duchenne muscular dystrophy possible?

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This week, we have a conversation about clinical trial design with Gen Li, the president and founder of Phesi, a global provider of patient-centric data analysis. 

Earlier this year, Phesi’s global analysis of all clinical trials conducted in 2023 revealed that more than a quarter (28%) of trials were cancelled during phase II – above the average attrition rate of 20% before 2020. 

00:39-01:31: About Phesi
01:31-01:49: Is your database global?
01:49-03:08: How successful are the different phases of clinical trials?
03:08-04:29: What are the biggest challenges for clinical trials currently?
04:29-06:23: Are clinical trials improving?
06:23-08:14: How can data improve clinical trials?
08:14-10:47: How does artificial intelligence affect clinical trial design?
10:47-12:53: Can clinical trial costs be reduced?
12:53-15:15: Can clinical trial times be shortened? 
15:15-18:21: Can data help with clinical trial diversity?
18:21-19:46: How can you fill knowledge gaps?
19:46-22:01: Do you have less data for rare diseases?
22:01-23:28: How does your company help with clinical trial design?
23:28-25:12: What kind of solutions can you provide?

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RTW is a global multi-strategy investment firm focused on supporting innovative biotech and biopharma companies across the US, Europe, and China. 

The company invests in both public and private companies across their entire life cycle, with a focus on those addressing next-generation gene and RNA therapies, rare diseases, targeted oncological, cardiovascular, and neurological disorders, and more.

To get an overview of what’s happening in financing for biotech companies currently, we had a conversation with Stephanie Sirota, chief business officer of RTW Investments.

01:11-04:18: About RTW Investments
04:18-06:45: What kind of biotech companies do you invest in?
06:45-08:31: How do you evaluate biotech companies?
08:31-11:36: How hands-on are you with companies you invest in?
11:36-14:08: How can companies raise funds currently?
14:08-16:05: Are there any undervalued sectors in biotech?
16:05-17:52: How important are M&As?
17:52-18:30: Is there a trend with M&As?
18:30-19:46: What is the state of the market with IPOs?
19:46-21:21: What are the major trends in biotech in 2024?
21:21-22:17: How could the US elections affect biotech funding?
22:17-24:41: What is the royalties market?
24:41-26:12: Are there regional differences?
26:12-27:52: Getting good biotech deals today

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