Keeping tabs on pharma: Big pharma makes progress on ‘difficult-to-treat’ diseases and GHD patients receive new drug

The past week saw significant advances in the field of drug development, with Eli Lilly teaming up with Lycia Therapeutics to tackle ‘difficult-to-treat’ diseases and the FDA giving approval for a new growth hormone developed by Ascedis Pharma. Read on to uncover the latest from the world of Pharma.

• Eli Lilly forms strategic collaboration with Lycia Terapeutics

  Eli Lilly has teamed up with Lycia Therapeutics, in order to gain access to its lysosomal targeting chimera (LYTAC) protein degradation technology. The collaboration has been formed with the intent of targeting and tackling ‘difficult-to-treat’ diseases.

  “This collaboration with Lycia furthers Lilly's strategy to utilize innovative new technology to treat challenging disease areas, such as immunology and pain,” said Ajay Nirula, VP of immunology at Eli Lilly. “We believe Lycia's technology may allow us to develop targeted therapeutics that were not previously feasible and make advances for patients in areas of high unmet need.”

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  According to Eli Lilly, the LYTAC platform has the potential to enable the development of several therapeutic modalities including the development of new antibodies and compounds that can inhibit targets previously considered intractable.

  According to Contract Pharma, this next-generation degradation approach, developed by Lycia, promises the ability to target the untapped extracellular proteome, including cell surface receptors and secreted proteins, utilizing the cell’s lysosomal trafficking and degradation pathway to target both soluble and membrane-bound extracellular proteins.

  Aetna Wun Trombley, PhD, president and CEO of Lycia, remarked: “We look forward to collaborating with Lilly to advance novel therapies against challenging targets in underserved disease areas while we simultaneously advance our in-house pipeline of first-in-class LYTAC-based therapeutics.”

• Ascendis gains FDA approval for once-weekly growth hormone

  Ascendis Pharma has taken steps to enhance convenience for growth hormone recipients with the development of a new drug that requires far fewer treatments than those currently in use, attaining FDA approval for their once-weekly growth hormone.

  Pfizer has also been developing its own once-weekly growth hormone, which will be up for FDA approval in October 2021.

  The Ascendis drug, lonapegsomatropin-tcgd, also known as Skytrofa, has been approved in children ages one and older who suffer from growth hormone deficiency (GHD) and weigh less than 25.4 pounds. The new GHD drug could be transformative for the lives of many children who suffer from the deficiency, promising them 86 per cent fewer injection days per year than they would have when using the current industry standard, Somatropin.

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  “This once-weekly treatment could reduce treatment burden and potentially replace the daily Somatropin therapies, which have been the standard of care for more than 30 years,” noted Paul Thornton, phase three investigator at Ascendis.

  Ascendis has seized its opportunity in what is a very lucrative market, while potentially providing convenience for millions in the United States, with plans to continue expanding into new markets outside of the US.

  “Considering the growth hormone market is fragmented with multiple undifferentiated products that must be treated daily, [Ascendis] believes that the market is primed for disruption,” noted Joseph Schwartz, senior research analyst covering rare diseases at SVB Leerink . Schwatz added that that he expected the drug to be approved in Europe by the end of the year.

  With the global human growth hormone market valued at more than $4bn, Pfizer and Opko Health have made progress with their own once-weekly hormone for GHD, with their version of the drug, Somatrogon, up for FDA decision in December 2021.

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