RARE Daily

Scholar Rock Raises $85 Million in Public Offering

October 12, 2023

Rare Daily Staff

Scholar Rock raised $85 million in an upsized underwritten public offering to advance a pipeline targeting neuromuscular disorders, cancer, and fibrosis.

The company priced 12.4 million shares of its common stock at $6.85 per share. The aggregate gross proceeds to Scholar Rock from this offering are expected to be approximately $85.0 million, before deducting underwriting discounts and commissions and other offering expenses. In addition, Scholar Rock granted the underwriters a 30-day option to purchase up to an additional 1.9 million shares of common stock at the public offering price, less the underwriting discounts and commissions.

Scholar Rock intends to use the net proceeds from the offering to advance lead therapeutic candidate apitegromab, develop its clinical and preclinical pipeline, as well as for working capital and other general corporate purposes.

Scholar Rock’s approach is to target the molecular mechanisms of growth factor activation with monoclonal antibodies that locally and selectively target these signaling proteins at the cellular level. By developing product candidates that act in the disease microenvironment, the company intends to avoid the historical challenges associated with inhibiting growth factors for therapeutic effect.

Apitegromab is a selective anti-latent myostatin in phase 3 development to improve motor function in patients with spinal muscular atrophy (SMA), a rare, and often fatal, neuromuscular disorder that typically manifests in young children and is characterized by the loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk and progressive muscle weakness. The underlying pathology of SMA is caused by insufficient production of the SMN (survival of motor neuron) protein, essential for the survival of motor neurons, and is encoded by two genes, SMN1 and SMN2. While there has been progress in the development of therapeutics that address the underlying SMA genetic defect, via SMN-dependent pathways, there continues to be a high unmet need for therapeutics that directly address muscle function.

Myostatin is a member of the TGFβ superfamily of growth factors that is expressed primarily by skeletal muscle cells to inhibit muscle growth and works in concert with other growth factors and hormones to maintain appropriate muscle mass. Vertebrates that lack the myostatin gene display increased muscle mass and strength but are otherwise healthy.

Scholar Rock is developing apitegromab with the aim of improving patients’ motor function. Rather than the traditional approach of blocking already activated, mature myostatin or the receptor, apitegromab selectively targets the precursor, or inactive, form of myostatin to block its activation in the muscle. By targeting the precursor form, apitegromab has demonstrated in in vitro studies that it offers potent and highly selective inhibition of myostatin while avoiding blocking the activity of other closely related members of the TGFβ superfamily that may lead to undesirable side effects.

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